Trial Outcomes & Findings for A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD) (NCT NCT04983264)

Results Overview

An adverse event (AE) was any untoward medical occurrence in a study participant administered a product or medical device; the event need not necessarily have a causal relationship with the treatment or usage. A treatment-emergent AE (TEAE) was an AE that occurs or worsens during the on-treatment period defined as the time from the first dose of study drug through minimum of 56 days after last dose of study treatment. A Serious Adverse Event (SAE) was any untoward medical occurrence at any dose that: resulted in death; was life-threatening experience (immediate risk of death); required inpatient hospitalization or prolongation if existing hospitalization; resulted in persistent or significant disability/ incapacity; resulted in congenital anomaly/birth defect; was considered an important medical event. TEAEs and SAEs were reported for both Sickle Cell Disease (SCD) and non-SCD related events, in this outcome measure.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

6 participants

Primary outcome timeframe

From first dose of study drug (Day 1) to at least 56 days after last dose of study drug (up to a maximum of 316 days)

Results posted on

2024-06-10

Participant Flow

Participant milestones

Participant milestones
Measure	Part A: Single Dose Period Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Part A: Single Dose Period STARTED	6	0	0
Part A: Single Dose Period COMPLETED	6	0	0
Part A: Single Dose Period NOT COMPLETED	0	0	0
Part B: Multiple Dose Period STARTED	0	6	0
Part B: Multiple Dose Period COMPLETED	0	5	0
Part B: Multiple Dose Period NOT COMPLETED	0	1	0
Part C: Extended Treatment Period STARTED	0	0	4
Part C: Extended Treatment Period COMPLETED	0	0	3
Part C: Extended Treatment Period NOT COMPLETED	0	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Part A: Single Dose Period Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Part B: Multiple Dose Period Physician Decision	0	1	0
Part C: Extended Treatment Period Physician Decision	0	0	1

Baseline Characteristics

A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Overall Study n=6 Participants All participants who received GBT021601 as single dose, multiple dose or extended treatment in Part A, Part B and Part C, respectively were included.
Age, Continuous	20.2 Years STANDARD_DEVIATION 2.23 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: From first dose of study drug (Day 1) to at least 56 days after last dose of study drug (up to a maximum of 316 days)

Population: Safety population was defined as all participants who received any amount of study drug (GBT021601).

An adverse event (AE) was any untoward medical occurrence in a study participant administered a product or medical device; the event need not necessarily have a causal relationship with the treatment or usage. A treatment-emergent AE (TEAE) was an AE that occurs or worsens during the on-treatment period defined as the time from the first dose of study drug through minimum of 56 days after last dose of study treatment. A Serious Adverse Event (SAE) was any untoward medical occurrence at any dose that: resulted in death; was life-threatening experience (immediate risk of death); required inpatient hospitalization or prolongation if existing hospitalization; resulted in persistent or significant disability/ incapacity; resulted in congenital anomaly/birth defect; was considered an important medical event. TEAEs and SAEs were reported for both Sickle Cell Disease (SCD) and non-SCD related events, in this outcome measure.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period n=6 Participants Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period n=4 Participants Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) TEAEs: SCD Related	1 Participants	3 Participants	1 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) SAEs: SCD Related	0 Participants	2 Participants	0 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) TEAEs: Non-SCD Related	5 Participants	5 Participants	3 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) SAEs: Non-SCD Related	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days)

Population: Safety population was defined as all participants who received any amount of study drug (GBT021601).

Physical examination included were general appearance, head, ears, eyes, nose, throat, neck, skin, cardiovascular system, respiratory system, gastrointestinal system, musculoskeletal system, lymph nodes, and nervous system. Clinically significant physical examination abnormalities were considered as adverse events based on investigator's discretion.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period n=6 Participants Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period n=4 Participants Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Number of Participants With Clinically Significant Physical Examination Findings	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days)

Population: Safety population was defined as all participants who received any amount of study drug (GBT021601).

Laboratory parameters included hematology(hemoglobin,hematocrit,red blood cell count,platelet count,white blood cell count,total neutrophils,eosinophils,monocytes,basophils,lymphocytes);blood chemistry(blood urea,nitrogen, creatinine,glucose,calcium,sodium,potassium,chloride,total bicarbonate,aspartate aminotransferase,alanine aminotransferase,total bilirubin,alkaline phosphatase,uric acid albumin,total protein);urinalysis(decimal logarithm of reciprocal of hydrogen ion activity \[pH\], glucose, protein, blood,ketones, microscopy\[urine tested positive for blood or protein\]),urine drug screening:cannabinoids,amphetamines,methamphetamines,opiates,methadone,cocaine,benzodiazepines,phencyclidine,barbiturates,alcohol breath test. Hemoximetry RBC deformability,dense cells test; erythropoietin,follicle stimulating hormone,pregnancy test,Serology panel for HIV 1/2 antibody,Hepatitis A, B and C,SARS CoV-2.Clinical significance of any parameter was determined at the investigator's discretion.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period n=6 Participants Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period n=4 Participants Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Number of Participants With Clinically Significant Change From Baseline in Laboratory Parameters	1 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days)

Population: Safety population was defined as all participants who received any amount of study drug (GBT021601).

Vital signs assessments included were systolic and diastolic blood pressure, heart rate, respiratory rate and body temperature. These measurements were taken after the participants had rested for at least 5 minutes in the supine position. Any clinically significant abnormal vital sign assessment required at least one repeat measurement. Clinical significance of any parameter was determined based on investigator's discretion.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period n=6 Participants Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period n=4 Participants Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Number of Participants With Clinically Significant Change From Baseline in Vital Signs	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days)

Population: Safety population was defined as all participants who received any amount of study drug (GBT021601).

ECG values included here were Heart rate (HR), PR, QRS, QT, and QTcF intervals, interpretation of the tracings (eg, rhythm, presence of arrhythmia or conduction defects, any evidence of myocardial ischemia/infarction, or ST segment, T-wave, and U-wave abnormalities). Abnormal and clinically significant 12-lead ECG included QT interval corrected for heart rate according to Fridericia's formula (QTcF) \> 450 millisecond (ms), QRS interval \>= 120 ms, PR interval \> 220 ms, based on the average of triplicated ECG, assessed at Screening and Day-1. If any of these test results were out of range, then the test could be repeated once (in triplicate).

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period n=6 Participants Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period n=4 Participants Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG)	1 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

Cmax was defined as the peak concentration observed directly from the experimental data without any interpolation.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Whole Blood	16.2 Microgram per milliliter (mcg/mL) Geometric Coefficient of Variation 25.90	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A RBC concentration	69.2 Microgram per milliliter (mcg/mL) Geometric Coefficient of Variation 28.63	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Plasma	1.01 Microgram per milliliter (mcg/mL) Geometric Coefficient of Variation 20.81	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

Cmax was defined as the peak concentration observed directly from the experimental data without any interpolation.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 98	2.77 mcg/mL Geometric Coefficient of Variation 16.41	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 105	2.96 mcg/mL Geometric Coefficient of Variation 19.79	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 56	2.46 mcg/mL Geometric Coefficient of Variation 15.27	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 63	1.28 mcg/mL Geometric Coefficient of Variation 18.54	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 70	1.54 mcg/mL Geometric Coefficient of Variation 32.36	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 77	1.17 mcg/mL Geometric Coefficient of Variation 22.44	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 84	1.17 mcg/mL Geometric Coefficient of Variation 19.75	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 91	5.15 mcg/mL Geometric Coefficient of Variation 19.32	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 112	3.22 mcg/mL Geometric Coefficient of Variation 15.50	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 56	40.9 mcg/mL Geometric Coefficient of Variation 25.31	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 63	85.5 mcg/mL Geometric Coefficient of Variation 40.91	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 70	80.3 mcg/mL Geometric Coefficient of Variation 37.07	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 77	87.1 mcg/mL Geometric Coefficient of Variation 41.84	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 84	89.1 mcg/mL Geometric Coefficient of Variation 52.79	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 91	136 mcg/mL Geometric Coefficient of Variation 41.37	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 98	201 mcg/mL Geometric Coefficient of Variation 33.19	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 105	194 mcg/mL Geometric Coefficient of Variation 44.14	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 112	200 mcg/mL Geometric Coefficient of Variation 31.58	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 56	169 mcg/mL Geometric Coefficient of Variation 25.28	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 63	366 mcg/mL Geometric Coefficient of Variation 38.19	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 70	309 mcg/mL Geometric Coefficient of Variation 38.95	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 77	337 mcg/mL Geometric Coefficient of Variation 43.45	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 84	345 mcg/mL Geometric Coefficient of Variation 50.80	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 91	522 mcg/mL Geometric Coefficient of Variation 35.93	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 98	788 mcg/mL Geometric Coefficient of Variation 25.66	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 105	748 mcg/mL Geometric Coefficient of Variation 27.21	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 112	670 mcg/mL Geometric Coefficient of Variation 33.48	—	—

SECONDARY outcome

Timeframe: Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28 and 42

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

Cmax was defined as the peak concentration observed directly from the experimental data without any interpolation.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=4 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C Plasma - Day 28	2.66 mcg/mL Geometric Coefficient of Variation 78.79	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C Plasma - Day 42	4.25 mcg/mL Geometric Coefficient of Variation 81.86	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C Whole Blood - Day 28	166 mcg/mL Geometric Coefficient of Variation 126.31	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C Whole Blood - Day 42	175 mcg/mL Geometric Coefficient of Variation 141.04	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C RBC concentration - Day 28	531 mcg/mL Geometric Coefficient of Variation 105.76	—	—
Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C RBC concentration - Day 42	540 mcg/mL Geometric Coefficient of Variation 125.43	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

Cmin was defined as the lowest concentration observed directly from the experimental data without any interpolation.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 105	1.84 mcg/mL Geometric Coefficient of Variation 27.55	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 112	2.03 mcg/mL Geometric Coefficient of Variation 24.43	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 91	76.5 mcg/mL Geometric Coefficient of Variation 49.02	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 63	0.97 mcg/mL Geometric Coefficient of Variation 27.41	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 70	0.98 mcg/mL Geometric Coefficient of Variation 22.18	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 77	0.90 mcg/mL Geometric Coefficient of Variation 20.38	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 84	0.884 mcg/mL Geometric Coefficient of Variation 21.58	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 91	0.90 mcg/mL Geometric Coefficient of Variation 30.51	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Plasma- Day 98	1.94 mcg/mL Geometric Coefficient of Variation 24.77	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 63	62.6 mcg/mL Geometric Coefficient of Variation 42.01	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 70	72.8 mcg/mL Geometric Coefficient of Variation 36.26	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 77	77.4 mcg/mL Geometric Coefficient of Variation 45.27	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 84	70.8 mcg/mL Geometric Coefficient of Variation 67.54	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 98	172 mcg/mL Geometric Coefficient of Variation 43.11	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 105	161 mcg/mL Geometric Coefficient of Variation 45.00	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B Whole Blood- Day 112	182 mcg/mL Geometric Coefficient of Variation 31.55	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 63	267 mcg/mL Geometric Coefficient of Variation 41.68	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 70	280 mcg/mL Geometric Coefficient of Variation 38.56	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 77	299 mcg/mL Geometric Coefficient of Variation 46.55	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 84	274 mcg/mL Geometric Coefficient of Variation 65.32	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 91	299 mcg/mL Geometric Coefficient of Variation 43.50	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 98	674 mcg/mL Geometric Coefficient of Variation 34.28	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 105	621 mcg/mL Geometric Coefficient of Variation 26.23	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B RBC concentration- Day 112	620 mcg/mL Geometric Coefficient of Variation 34.72	—	—

SECONDARY outcome

Timeframe: Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28 and 42

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

Cmin was defined as the lowest concentration observed directly from the experimental data without any interpolation.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=4 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C Plasma - Day 28	1.54 mcg/mL Geometric Coefficient of Variation 220.43	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C Plasma - Day 42	1.99 mcg/mL Geometric Coefficient of Variation 264.95	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C Whole Blood - Day 28	122 mcg/mL Geometric Coefficient of Variation 211.47	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C Whole Blood - Day 42	140 mcg/mL Geometric Coefficient of Variation 217.11	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C RBC concentration - Day 28	392 mcg/mL Geometric Coefficient of Variation 174.41	—	—
Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C RBC concentration - Day 42	433 mcg/mL Geometric Coefficient of Variation 187.77	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

Maximum observed concentration was defined as the peak concentration observed directly from the experimental data without any interpolation. The time to the maximum observed concentration was defined as the time corresponding to Cmax.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Time to Attain Maximum Serum Concentration (Tmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Plasma	1.50 Hours Interval 1.0 to 2.02	—	—
Time to Attain Maximum Serum Concentration (Tmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Whole Blood	8.06 Hours Interval 6.0 to 35.92	—	—
Time to Attain Maximum Serum Concentration (Tmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A RBC concentration	8.06 Hours Interval 6.0 to 35.92	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

AUC0-tau was defined from time 0 to time of the last quantifiable concentration and was calculated using the linear or logarithmic trapezoid rule. AUCtau was calculated by using hours\*microgram per milliliter (hr\*mcg)/mL.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Area Under the Concentration Time Curve From Time Zero to the Next Dose (AUC0-tau) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Plasma	67.102 (hr*mcg)/mL Geometric Coefficient of Variation 13.91	—	—
Area Under the Concentration Time Curve From Time Zero to the Next Dose (AUC0-tau) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Whole Blood	4597.8 (hr*mcg)/mL Geometric Coefficient of Variation 27.07	—	—
Area Under the Concentration Time Curve From Time Zero to the Next Dose (AUC0-tau) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A RBC concentration	19627.6 (hr*mcg)/mL Geometric Coefficient of Variation 30.93	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

AUCinf was defined as the area calculated by linear/log trapezoid rule from time 0 to infinity with the area extrapolated from the last quantifiable concentration to infinity.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Area Under the Serum Concentration Time Curve From Time Zero Extrapolated to Infinite Time (AUCinf) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Plasma	72.03 (hr*mcg)/mL Geometric Coefficient of Variation 16.43	—	—
Area Under the Serum Concentration Time Curve From Time Zero Extrapolated to Infinite Time (AUCinf) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Whole Blood	4914.50 (hr*mcg)/mL Geometric Coefficient of Variation 29.55	—	—
Area Under the Serum Concentration Time Curve From Time Zero Extrapolated to Infinite Time (AUCinf) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A RBC concentration	234.08 (hr*mcg)/mL Geometric Coefficient of Variation 13.95	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

AUC0-24 was defined as the area under the free plasma concentration time curve from time 0 to 24 hours post-dose.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Area Under the Concentration Time Curve From Time Zero up to Time 24 Hours (AUC 0-24) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Plasma	7.94 (hr*mcg)/mL Geometric Coefficient of Variation 12.11	—	—
Area Under the Concentration Time Curve From Time Zero up to Time 24 Hours (AUC 0-24) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Whole Blood	322.0 (hr*mcg)/mL Geometric Coefficient of Variation 25.28	—	—
Area Under the Concentration Time Curve From Time Zero up to Time 24 Hours (AUC 0-24) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A RBC concentration	1363.3 (hr*mcg)/mL Geometric Coefficient of Variation 29.90	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

CL/F was a quantitative measure of the rate at which a drug substance was removed from the blood. It was calculated as dose of GBT021601 by AUC from time 0 to infinity.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Apparent Oral Clearance (CL/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Plasma	1.39 Liter per hour Geometric Coefficient of Variation 16.43	—	—
Apparent Oral Clearance (CL/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Whole Blood	0.02 Liter per hour Geometric Coefficient of Variation 29.55	—	—
Apparent Oral Clearance (CL/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A RBC concentration	0.004 Liter per hour Geometric Coefficient of Variation 33.36	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

t1/2 was the time measured for the plasma concentration to decrease by one half.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Terminal Elimination Half-Life (t1/2) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Plasma	236.191 hr Interval 187.44 to 313.49	—	—
Terminal Elimination Half-Life (t1/2) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Whole Blood	230.908 hr Interval 207.8 to 303.05	—	—
Terminal Elimination Half-Life (t1/2) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A RBC concentration	231.272 hr Interval 206.58 to 302.82	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

Volume of distribution was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vz/F was determined based on the fraction absorbed.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Apparent Volume of Distribution (Vz/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Plasma	480 Liter Geometric Coefficient of Variation 13.30	—	—
Apparent Volume of Distribution (Vz/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A Whole Blood	6.87 Liter Geometric Coefficient of Variation 22.56	—	—
Apparent Volume of Distribution (Vz/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A RBC concentration	1.61 Liter Geometric Coefficient of Variation 26.23	—	—

SECONDARY outcome

Timeframe: Part A: Pre-dose,0.25,0.5,1,2,4, 6,8,12,24,36,48,72,168,336,504,672,1008 hrs on Day 1; Part B: Pre-dose,0.25 to 1,2 to 4 hrs post-dose on Day 56,63,70,77,84,91,98,105,112; Part C:Pre-dose on Day 1,14,28,42 and 0.25 to 1, 2 to 4 hrs post-dose on Day 28,42

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

Percentage hemoglobin occupancy (%Hb Occupancy) refers to the proportion of hemoglobin molecules within red blood cells that were bound to study drug (GBT021601). Cmin and Cmax values was used to calculate %Hb occupancy: %Hb Occupancy = GBT021601\*RBC per Mean Corpuscular Hemoglobin Concentration (MCHC).

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period n=6 Participants Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period n=4 Participants Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Percentage Hemoglobin Occupancy Part B: Cmin- Day 70	—	14.03 Percentage of Hemoglobin Standard Deviation 4.69	—
Percentage Hemoglobin Occupancy Part B: Cmax- Day 77	—	17.03 Percentage of Hemoglobin Standard Deviation 6.00	—
Percentage Hemoglobin Occupancy Part B: Cmin- Day 77	—	15.25 Percentage of Hemoglobin Standard Deviation 5.54	—
Percentage Hemoglobin Occupancy Part B: Cmax- Day 84	—	17.66 Percentage of Hemoglobin Standard Deviation 7.75	—
Percentage Hemoglobin Occupancy Part B: Cmin- Day 84	—	14.57 Percentage of Hemoglobin Standard Deviation 7.29	—
Percentage Hemoglobin Occupancy Part B: Cmax- Day 91	—	25.71 Percentage of Hemoglobin Standard Deviation 7.92	—
Percentage Hemoglobin Occupancy Part B: Cmin- Day 91	—	15.02 Percentage of Hemoglobin Standard Deviation 5.85	—
Percentage Hemoglobin Occupancy Part B: Cmax- Day 98	—	38.00 Percentage of Hemoglobin Standard Deviation 9.05	—
Percentage Hemoglobin Occupancy Part B: Cmin- Day 98	—	33.04 Percentage of Hemoglobin Standard Deviation 9.31	—
Percentage Hemoglobin Occupancy Part B: Cmax- Day 105	—	36.11 Percentage of Hemoglobin Standard Deviation 9.14	—
Percentage Hemoglobin Occupancy Part B: Cmin- Day 105	—	29.89 Percentage of Hemoglobin Standard Deviation 7.21	—
Percentage Hemoglobin Occupancy Part B: Cmax- Day 112	—	32.68 Percentage of Hemoglobin Standard Deviation 9.33	—
Percentage Hemoglobin Occupancy Part B: Cmin- Day 112	—	30.32 Percentage of Hemoglobin Standard Deviation 8.92	—
Percentage Hemoglobin Occupancy Part C: Cmax- Day 28	—	—	31.45 Percentage of Hemoglobin Standard Deviation 17.97
Percentage Hemoglobin Occupancy Part C: Cmin- Day 28	—	—	26.54 Percentage of Hemoglobin Standard Deviation 16.96
Percentage Hemoglobin Occupancy Part C: Cmax- Day 42	—	—	33.13 Percentage of Hemoglobin Standard Deviation 19.12
Percentage Hemoglobin Occupancy Part C: Cmin- Day 42	—	—	29.46 Percentage of Hemoglobin Standard Deviation 18.09
Percentage Hemoglobin Occupancy Part A: Cmax- Day 1	3.36 Percentage of Hemoglobin Standard Deviation 0.86	—	—
Percentage Hemoglobin Occupancy Part B: Cmax- Day 56	—	8.15 Percentage of Hemoglobin Standard Deviation 1.89	—
Percentage Hemoglobin Occupancy Part B: Cmax - Day 63	—	18.15 Percentage of Hemoglobin Standard Deviation 5.96	—
Percentage Hemoglobin Occupancy Part B: Cmin- Day 63	—	13.40 Percentage of Hemoglobin Standard Deviation 4.71	—
Percentage Hemoglobin Occupancy Part B: Cmax- Day 70	—	15.50 Percentage of Hemoglobin Standard Deviation 5.11	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

The plasma concentration versus time summary was measured by the amount of specific substance in the bloodstream over the specified timepoint.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Plasma Concentrations Versus Time Summary of GBT021601: Part A Pre-dose	0 mcg/mL Standard Deviation 0	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 0.25 hour	0.145 mcg/mL Standard Deviation 0.137	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 0.5 hour	0.617 mcg/mL Standard Deviation 0.281	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 1 hour	0.910 mcg/mL Standard Deviation 0.312	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 2 hours	0.821 mcg/mL Standard Deviation 0.202	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 4 hours	0.498 mcg/mL Standard Deviation 0.150	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 6 hours	0.350 mcg/mL Standard Deviation 0.100	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 8 hours	0.279 mcg/mL Standard Deviation 0.737	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 12 hours	0.228 mcg/mL Standard Deviation 0.040	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 24 hours	0.257 mcg/mL Standard Deviation 0.120	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 36 hours	0.333 mcg/mL Standard Deviation 0.286	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 48 hours	0.183 mcg/mL Standard Deviation 0.067	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 72 hours	0.138 mcg/mL Standard Deviation 0.019	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 168 hours	0.124 mcg/mL Standard Deviation 0.020	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 336 hours	0.0786 mcg/mL Standard Deviation 0.016	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 504 hours	0.047 mcg/mL Standard Deviation 0.016	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 672 hours	0.029 mcg/mL Standard Deviation 0.137	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part A 1008 hours	0.013 mcg/mL Standard Deviation 0.007	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112, 140,168, 196, 218

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints.

The plasma concentration versus time summary was measured by the amount of specific substance in the bloodstream over the specified timepoint.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 56: Pre-dose	0.143 mcg/mL Standard Deviation 0.351	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 56 :0.25-1 hour	1.163 mcg/mL Standard Deviation 1.165	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 56: 2-4 hours	2.345 mcg/mL Standard Deviation 0.3129	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 63: Pre-dose	1.015 mcg/mL Standard Deviation 0.240	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 63: 0.25-1 hour	1.148 mcg/mL Standard Deviation 0.268	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 63: 2-4 hours	1.250 mcg/mL Standard Deviation 0.210	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 70: Pre-dose	1.003 mcg/mL Standard Deviation 0.211	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 70: 0.25-1 hour	1.376 mcg/mL Standard Deviation 0.653	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 70: 2-4 hours	1.415 mcg/mL Standard Deviation 0.262	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 77: Pre-dose	0.937 mcg/mL Standard Deviation 0.151	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 77: 0.25-1 hour	1.103 mcg/mL Standard Deviation 0.318	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 77: 2-4 hours	1.173 mcg/mL Standard Deviation 0.233	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 84: Pre-dose	0.918 mcg/mL Standard Deviation 0.176	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 84 :0.25-1 hour	1.085 mcg/mL Standard Deviation 0.284	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 84: 2-4 hours	1.097 mcg/mL Standard Deviation 0.205	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 91: Pre-dose	0.941 mcg/mL Standard Deviation 0.278	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 91; 0.25-1 hour	3.019 mcg/mL Standard Deviation 2.225	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 91: 2-4 hours	4.990 mcg/mL Standard Deviation 0.635	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 98: Pre-dose	2.118 mcg/mL Standard Deviation 0.687	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 98: 0.25-1 hour	2.242 mcg/mL Standard Deviation 0.398	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 98: 2-4 hours	2.580 mcg/mL Standard Deviation 0.53933	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 105: Pre-dose	1.945 mcg/mL Standard Deviation 0.570	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 105: 0.25-1 hour	2.147 mcg/mL Standard Deviation 0.470	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 105: 2-4 hours	2.953 mcg/mL Standard Deviation 0.642	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 112 :Predose	2.095 mcg/mL Standard Deviation 0.242	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 112: 0.25-1 hour	2.748 mcg/mL Standard Deviation 0.824	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 112: 2-4 hours	2.905 mcg/mL Standard Deviation 0.688	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 140: 2-4 hours	0.225 mcg/mL Standard Deviation 0.0516	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 168: 2-4 hours	0.042 mcg/mL Standard Deviation 0.026	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 196: 2-4 hours	0.005 mcg/mL Standard Deviation 0.005	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part B Day 218: 2-4 hours	0.000 mcg/mL Standard Deviation 0.001	—	—

SECONDARY outcome

Timeframe: Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28, 42, 70, 98

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints.

The plasma concentration versus time summary was measured by the amount of specific substance in the bloodstream over the specified timepoint.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=4 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Plasma Concentrations Versus Time Summary of GBT021601: Part C Day 28: Pre-dose	2.448 mcg/mL Standard Deviation 1.786	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part C Day 28: 4 hours	2.991 mcg/mL Standard Deviation 1.476	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part C Day 42: Pre-dose	3.621 mcg/mL Standard Deviation 2.929	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part C Day 42: 0.25-1 hour	4.285 mcg/mL Standard Deviation 2.785	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part C Day 42: 2-4 hours	4.485 mcg/mL Standard Deviation 2.308	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part C Day 70: 2-4 hours	0.364 mcg/mL Standard Deviation 0.101	—	—
Plasma Concentrations Versus Time Summary of GBT021601: Part C Day 98: 2-4 hours	0.034 mcg/mL Standard Deviation 0.011	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

The whole blood concentration versus time summary was measured to find concentration of various components in whole blood that changed over a specified period. This helped to monitor the dynamics of blood components such as hemoglobin levels, hematocrit, or glucose concentration over time.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A Pre-dose	0 mcg/mL Standard Deviation 0	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 0.25 hour	1.034 mcg/mL Standard Deviation 0.876	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 0.5 hour	4.775 mcg/mL Standard Deviation 2.470	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 1 hour	8.745 mcg/mL Standard Deviation 3.833	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 2 hours	12.710 mcg/mL Standard Deviation 3.190	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 4 hours	14.07 mcg/mL Standard Deviation 2.917	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 6 hours	14.44 mcg/mL Standard Deviation 2.980	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 8 hours	14.42 mcg/mL Standard Deviation 2.861	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 12 hours	14.45 mcg/mL Standard Deviation 3.811	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 24 hours	14.42 mcg/mL Standard Deviation 3.220	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 36 hours	15.41 mcg/mL Standard Deviation 4.220	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 48 hours	13.14 mcg/mL Standard Deviation 2.895	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 72 hours	12.42 mcg/mL Standard Deviation 3.193	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 168 hours	9.847 mcg/mL Standard Deviation 2.562	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 336 hours	5.282 mcg/mL Standard Deviation 1.283	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 504 hours	3.870 mcg/mL Standard Deviation 1.071	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 672 hours	2.138 mcg/mL Standard Deviation 1.283	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part A 1008 hours	0.943 mcg/mL Standard Deviation 0.729	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs, post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112, 140,168, 196, 218

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints.

The whole blood concentration versus time summary was measured to find concentration of various components in whole blood that changed over a specified period. This helped to monitor the dynamics of blood components such as hemoglobin levels, hematocrit, or glucose concentration over time.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 56: Pre-dose	0.071 mcg/mL Standard Deviation 0.085	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 56: 0.25-1 hour	13.20 mcg/mL Standard Deviation 13.601	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 56: 2-4 hours	41.23 mcg/mL Standard Deviation 10.318	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 63: Pre-dose	74.32 mcg/mL Standard Deviation 30.266	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 63: 0.25-1 hour	88.23 mcg/mL Standard Deviation 33.335	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 63: 2- 4 hours	74.87 mcg/mL Standard Deviation 19.748	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 70: Pre-dose	78.28 mcg/mL Standard Deviation 24.403	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 70: 0.25-1 hour	81.00 mcg/mL Standard Deviation 22.709	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 70: 2-4 hours	81.25 mcg/mL Standard Deviation 25.669	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 77: Pre-dose	86.22 mcg/mL Standard Deviation 30.703	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 77: 0.25-1 hour	87.33 mcg/mL Standard Deviation 27.507	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 77: 2- 4 hours	88.68 mcg/mL Standard Deviation 29.043	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 84: Predose	87.77 mcg/mL Standard Deviation 43.076	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 84 :0.25-1 hour	88.97 mcg/mL Standard Deviation 42.685	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 84: 2-4 hours	92.05 mcg/mL Standard Deviation 36.530	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 91: Pre-dose	85.07 mcg/mL Standard Deviation 32.859	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 91; 0.25-1 hour	102.4 mcg/mL Standard Deviation 52.161	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 91: 2-4 hours	144.1 mcg/mL Standard Deviation 47.113	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 98: Pre-dose	195.8 mcg/mL Standard Deviation 61.979	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 98: 0.25-1 hour	188.8 mcg/mL Standard Deviation 61.802	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 98: 2-4 hours	198.8 mcg/mL Standard Deviation 49.301	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 105: Pre-dose	189.8 mcg/mL Standard Deviation 56.672	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 105: 0.25-1 hour	178.9 mcg/mL Standard Deviation 66.612	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 105: 2-4 hours	203.3 mcg/mL Standard Deviation 74.923	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 112: Pre-dose	195.0 mcg/mL Standard Deviation 59.117	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 112: 0.25-1 hour	200.5 mcg/mL Standard Deviation 57.263	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 112: 2-4 hours	198.5 mcg/mL Standard Deviation 59.248	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 140: 2-4 hours	29.32 mcg/mL Standard Deviation 20.149	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 168: 2-4 hours	3.082 mcg/mL Standard Deviation 2.137	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 196: 2-4 hours	0.561 mcg/mL Standard Deviation 0.560	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part B Day 218: 2-4 hours	0.108 mcg/mL Standard Deviation 0.109	—	—

SECONDARY outcome

Timeframe: Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28, 42, 70, 98

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints.

The whole blood concentration versus time summary was measured to find concentration of various components in whole blood that changed over a specified period. This helped to monitor the dynamics of blood components such as hemoglobin levels, hematocrit, or glucose concentration over time.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=4 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Whole Blood Concentrations Versus Time Summary of GBT021601: Part C Day 28: Pre-dose	202.2 mcg/mL Standard Deviation 136.32	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part C Day 28: 4 hours	193.6 mcg/mL Standard Deviation 106.16	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part C Day 42: Pre-dose	214.8 mcg/mL Standard Deviation 131.21	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part C Day 42: 0.25-1 hour	213.5 mcg/mL Standard Deviation 119.79	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part C Day 42: 2- 4 hours	229.9 mcg/mL Standard Deviation 132.21	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part C Day 70: 2-4 hours	33.50 mcg/mL Standard Deviation 2.553	—	—
Whole Blood Concentrations Versus Time Summary of GBT021601: Part C Day 98: 2-4 hours	3.310 mcg/mL Standard Deviation 0.826	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point.

The RBC concentration versus time summary was measured to identify quantity of RBC in a sample of blood over a specified duration. This analysis helped monitor RBC level over time.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A Pre-dose	0 mcg/mL Standard Deviation 0	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 0.25 hour	3.989 mcg/mL Standard Deviation 3.314	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 0.5 hour	18.68 mcg/mL Standard Deviation 10.413	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 1 hour	34.84 mcg/mL Standard Deviation 16.282	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 2 hours	52.51 mcg/mL Standard Deviation 14.931	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 4 hours	59.50 mcg/mL Standard Deviation 14.281	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 6 hours	61.72 mcg/mL Standard Deviation 15.356	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 8 hours	61.68 mcg/mL Standard Deviation 13.932	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 12 hours	62.24 mcg/mL Standard Deviation 18.340	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 24 hours	61.92 mcg/mL Standard Deviation 16.283	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 36 hours	65.62 mcg/mL Standard Deviation 18.780	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 48 hours	56.60 mcg/mL Standard Deviation 14.463	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 72 hours	53.68 mcg/mL Standard Deviation 15.728	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 168 hours	42.41 mcg/mL Standard Deviation 12.260	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 336 hours	22.73 mcg/mL Standard Deviation 6.316	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 504 hours	16.65 mcg/mL Standard Deviation 4.931	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 672 hours	9.207 mcg/mL Standard Deviation 5.652	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A 1008 hours	4.073 mcg/mL Standard Deviation 3.186	—	—

SECONDARY outcome

Timeframe: Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112, 140,168, 196, 218

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints.

The RBC concentration versus time summary was measured to identify quantity of RBC in a sample of blood over a specified duration. This analysis helped monitor RBC level over time.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=6 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 105: 2-4 hours	752.8 mcg/mL Standard Deviation 222.08	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 112: Predose	705.6 mcg/mL Standard Deviation 209.26	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 56: Pre-dose	0.120 mcg/mL Standard Deviation 0.134	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 56 :0.25-1 hour	52.57 mcg/mL Standard Deviation 53.388	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 56: 2-4 hours	170.8 mcg/mL Standard Deviation 45.616	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 63: Pre-dose	317.4 mcg/mL Standard Deviation 126.93	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 63: 0.25-1 hour	375.4 mcg/mL Standard Deviation 134.89	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 63: 2-4 hours	318.6 mcg/mL Standard Deviation 83.313	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 70: Pre-dose	304.7 mcg/mL Standard Deviation 105.71	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 70: 0.25-1 hour	313.9 mcg/mL Standard Deviation 97.320	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 70: 2-4 hours	314.7 mcg/mL Standard Deviation 106.95	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 77: Pre-dose	335.9 mcg/mL Standard Deviation 130.15	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 77: 0.25-1 hour	340.1 mcg/mL Standard Deviation 118.67	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 77: 2-4 hours	343.8 mcg/mL Standard Deviation 120.89	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 84: Pre-dose	338.6 mcg/mL Standard Deviation 169.41	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 84 :0.25-1 hour	344.7 mcg/mL Standard Deviation 170.26	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 84: 2-4 hours	356.0 mcg/mL Standard Deviation 144.12	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 91: Pre-dose	329.0 mcg/mL Standard Deviation 124.33	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 91; 0.25-1 hour	392.6 mcg/mL Standard Deviation 197.92	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 91: 2-4 hours	547.7 mcg/mL Standard Deviation 173.83	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 98: Pre-dose	753.5 mcg/mL Standard Deviation 207.85	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 98: 0.25-1 hour	726.2 mcg/mL Standard Deviation 215.42	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 98: 2-4 hours	768.9 mcg/mL Standard Deviation 164.84	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 105: Pre-dose	711.7 mcg/mL Standard Deviation 148.81	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 105: 0.25-1 hour	662.4 mcg/mL Standard Deviation 190.38	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 112: 0.25-1 hour	674.3 mcg/mL Standard Deviation 201.56	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 112: 2-4 hours	666.7 mcg/mL Standard Deviation 206.56	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 140: 2-4 hours	120.8 mcg/mL Standard Deviation 83.588	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 168: 2-4 hours	14.97 mcg/mL Standard Deviation 9.339	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 196: 2-4 hours	2.326 mcg/mL Standard Deviation 2.226	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B Day 218: 2-4 hours	0.850 mcg/mL Standard Deviation 0.539	—	—

SECONDARY outcome

Timeframe: Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28, 42, 70, 98

Population: Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints.

The RBC concentration versus time summary involved tracking a quantity of RBC in a sample of blood over a specified duration. This analysis helped to monitor RBC level over time.

Outcome measures

Outcome measures
Measure	Part A: Single Dose Period n=4 Participants Participants received a single oral dose of GBT021601 100 mg on Day 1.	Part B: Multiple Ascending- Dose Period Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants.	Part C: Extended Treatment Period Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks.
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C Day 28: Pre-dose	621.3 mcg/mL Standard Deviation 428.95	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C Day 28: 4 hours	594.4 mcg/mL Standard Deviation 319.62	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C Day 42: Pre-dose	642.4 mcg/mL Standard Deviation 396.97	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C Day 42: 0.25-1 hour	639.8 mcg/mL Standard Deviation 361.11	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C Day 42: 2-4 hours	691.1 mcg/mL Standard Deviation 404.73	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C Day 70: 2-4 hours	117.9 mcg/mL Standard Deviation 20.028	—	—
Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C Day 98: 2-4 hours	12.74 mcg/mL Standard Deviation 3.256	—	—

Adverse Events

Part A: Single Dose Period (SCD Related)

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Part B: Multiple Ascending- Dose Period (SCD Related)

Serious events: 2 serious events

Other events: 2 other events

Deaths: 0 deaths

Part C: Extended Treatment Period (SCD Related)

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Part A: Single Dose Period (Non-SCD Related)

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Part B: Multiple Ascending- Dose Period (Non-SCD Related)

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Part C: Extended Treatment Period (Non-SCD Related)

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Part A: Single Dose Period (SCD Related) n=6 participants at risk Participants received a single oral dose of GBT021601 100 mg on Day 1 and had SCD related adverse events.	Part B: Multiple Ascending- Dose Period (SCD Related) n=6 participants at risk Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants and had SCD related adverse events.	Part C: Extended Treatment Period (SCD Related) n=4 participants at risk Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks and had SCD related adverse events.	Part A: Single Dose Period (Non-SCD Related) n=6 participants at risk Participants received a single oral dose of GBT021601 100 mg on Day 1 and had non-SCD related adverse events.	Part B: Multiple Ascending- Dose Period (Non-SCD Related) n=6 participants at risk Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted percentage Hemoglobin (% Hb) occupancy of participants and had non-SCD related adverse events.	Part C: Extended Treatment Period (Non-SCD Related) n=4 participants at risk Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks and had non-SCD related adverse events.
Blood and lymphatic system disorders Sickle cell anaemia with crisis	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	33.3% 2/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.

Other adverse events

Other adverse events
Measure	Part A: Single Dose Period (SCD Related) n=6 participants at risk Participants received a single oral dose of GBT021601 100 mg on Day 1 and had SCD related adverse events.	Part B: Multiple Ascending- Dose Period (SCD Related) n=6 participants at risk Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants and had SCD related adverse events.	Part C: Extended Treatment Period (SCD Related) n=4 participants at risk Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks and had SCD related adverse events.	Part A: Single Dose Period (Non-SCD Related) n=6 participants at risk Participants received a single oral dose of GBT021601 100 mg on Day 1 and had non-SCD related adverse events.	Part B: Multiple Ascending- Dose Period (Non-SCD Related) n=6 participants at risk Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted percentage Hemoglobin (% Hb) occupancy of participants and had non-SCD related adverse events.	Part C: Extended Treatment Period (Non-SCD Related) n=4 participants at risk Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks and had non-SCD related adverse events.
Blood and lymphatic system disorders Neutropenia	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	33.3% 2/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Blood and lymphatic system disorders Anaemia	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Blood and lymphatic system disorders Thrombocytosis	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Ear and labyrinth disorders Tinnitus	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Gastrointestinal disorders Diarrhoea	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	33.3% 2/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Gastrointestinal disorders Abdominal pain	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	25.0% 1/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Gastrointestinal disorders Abdominal pain upper	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Gastrointestinal disorders Constipation	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Gastrointestinal disorders Flatulence	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Gastrointestinal disorders Vomiting	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Hepatobiliary disorders Hyperbilirubinaemia	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Infections and infestations Adenoviral upper respiratory infection	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Infections and infestations Tooth infection	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Infections and infestations Viral upper respiratory tract infection	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Injury, poisoning and procedural complications Thermal burn	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Investigations Blood creatinine increased	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Investigations Electrocardiogram QT prolonged	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Investigations Transaminases increased	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Metabolism and nutrition disorders Hyperuricaemia	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	25.0% 1/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	25.0% 1/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	25.0% 1/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Nervous system disorders Headache	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	25.0% 1/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Renal and urinary disorders Urinary retention	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Skin and subcutaneous tissue disorders Angioedema	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Vascular disorders Thrombophlebitis	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
Blood and lymphatic system disorders Sickle cell anaemia with crisis	16.7% 1/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	33.3% 2/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	25.0% 1/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/6 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.	0.00% 0/4 • From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days) Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.

Additional Information

Pfizer ClinicalTrials.gov Call Center

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Phone: 1-800-718-1021

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publication until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Publication restrictions are in place

Restriction type: OTHER