Trial Outcomes & Findings for A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA) (NCT NCT03075878)
NCT ID: NCT03075878
Last Updated: 2020-05-13
Results Overview
A TEAE was defined as any adverse event that starts on or after the first dose of study drug or occurs prior to the first dose and worsens in severity on or after the first dose of study drug, during the Treatment Period and Follow-up Period. A TEAE was considered "serious" if, in the view of either the investigator or sponsor, it resulted in any of the following outcomes: death, life-threatening adverse drug event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an event that may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously listed outcomes. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. All serious TEAEs were not considered related to the study drug.
TERMINATED
PHASE1/PHASE2
8 participants
Day 0 (after first dose) through Day 112
2020-05-13
Participant Flow
This study was terminated after the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy were characterized in Cohort 1 (SYNT001 Dose 1), before any participants were enrolled in Cohort 2 (SYNT001 Dose 2). This results disclosure is for Cohort 1 only.
Participant milestones
| Measure |
Cohort 1: ALXN1830
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
Overall Study
STARTED
|
8
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
8
|
|
Overall Study
COMPLETED
|
7
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Cohort 1: ALXN1830
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
Overall Study
Physician Decision
|
1
|
Baseline Characteristics
A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA)
Baseline characteristics by cohort
| Measure |
Cohort 1: ALXN1830
n=8 Participants
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
Age, Continuous
|
54.9 years
STANDARD_DEVIATION 16.82 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 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
|
1 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Age at Warm Autoimmune Hemolytic Anemia Diagnosis
|
48.4 years
STANDARD_DEVIATION 16.85 • n=5 Participants
|
PRIMARY outcome
Timeframe: Day 0 (after first dose) through Day 112Population: Safety Population: All participants who received at least 1 dose of study drug.
A TEAE was defined as any adverse event that starts on or after the first dose of study drug or occurs prior to the first dose and worsens in severity on or after the first dose of study drug, during the Treatment Period and Follow-up Period. A TEAE was considered "serious" if, in the view of either the investigator or sponsor, it resulted in any of the following outcomes: death, life-threatening adverse drug event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an event that may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously listed outcomes. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. All serious TEAEs were not considered related to the study drug.
Outcome measures
| Measure |
Cohort 1: ALXN1830
n=8 Participants
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
TEAEs
|
8 Participants
|
|
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
Deaths
|
0 Participants
|
|
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
Serious TEAEs
|
2 Participants
|
|
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
Discontinuations due to TEAEs
|
0 Participants
|
SECONDARY outcome
Timeframe: Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdosePopulation: PK Population: All participants who received at least 1 dose of study drug and had postdose PK data available at the specified timepoint.
The Cmax was determined directly from the concentration-time profile. Starting on Days 0 and 28, serum samples were collected just prior to the start of study drug infusion (predose), at 5 minutes, at 2, 4, 6, 24, and 48 hours, and at 5 days postdose after the end-of-study drug infusion. Results are reported in micrograms/milliliter (ug/mL).
Outcome measures
| Measure |
Cohort 1: ALXN1830
n=8 Participants
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
Maximum Serum Concentration (Cmax) On Day 0 And Day 28
Day 0
|
249.6 ug/mL
Standard Deviation 35.76
|
|
Maximum Serum Concentration (Cmax) On Day 0 And Day 28
Day 28
|
245.8 ug/mL
Standard Deviation 41.55
|
SECONDARY outcome
Timeframe: Baseline, Day 33Population: PD Population: All participants who received at least 1 dose of study drug and had postdose PD data available at the specified timepoint.
Reticulocyte count was measured as a PD biomarker. Pharmacodynamic samples were collected for analyses throughout the study prior to infusion of study drug for Cohort 1. Measurements for PD biomarkers were derived from the laboratory results. Results are reported in cells/liter (cells\*10\^12/L). A decrease in reticulocyte count indicated a potential improvement in condition.
Outcome measures
| Measure |
Cohort 1: ALXN1830
n=7 Participants
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
Change From Baseline In Reticulocyte Count At Day 33
|
-0.044 cells*10^12/L
Standard Deviation 0.0948
|
SECONDARY outcome
Timeframe: Baseline, Day 33Population: PD Population: All participants who received at least 1dose of study drug and had postdose PD data available at the specified timepoint.
Hemoglobin was measured as a PD biomarker. Pharmacodynamic samples were collected for analyses throughout the study prior to infusion of study drug for Cohort 1. Measurements for PD biomarkers were derived from the laboratory results. Results are reported in grams/deciliter (g/dL). An increase in hemoglobin indicated a potential improvement in condition.
Outcome measures
| Measure |
Cohort 1: ALXN1830
n=8 Participants
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
Change From Baseline In Hemoglobin At Day 33
|
0.66 g/dL
Standard Deviation 1.581
|
SECONDARY outcome
Timeframe: Day 112Population: Safety Population: All participants who received at least 1 dose of study drug.
Immunogenicity analyses are reported for Day 112. Testing was carried out to detect binding antidrug antibodies by anti-ALXN1830 antibody level. Results are reported as the reciprocal of the titer where they cross the study cut point.
Outcome measures
| Measure |
Cohort 1: ALXN1830
n=4 Participants
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level
|
99.220 reciprocal of the titer
Standard Deviation 165.8149
|
Adverse Events
Cohort 1: ALXN1830
Serious adverse events
| Measure |
Cohort 1: ALXN1830
n=8 participants at risk
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
12.5%
1/8 • Baseline through Day 112
|
|
Gastrointestinal disorders
Gastritis
|
12.5%
1/8 • Baseline through Day 112
|
Other adverse events
| Measure |
Cohort 1: ALXN1830
n=8 participants at risk
SYNT001 Dose 1: Participants received ALXN1830.
|
|---|---|
|
General disorders
Fatigue
|
25.0%
2/8 • Baseline through Day 112
|
|
General disorders
Chills
|
12.5%
1/8 • Baseline through Day 112
|
|
General disorders
Face oedema
|
12.5%
1/8 • Baseline through Day 112
|
|
General disorders
Influenza like illness
|
12.5%
1/8 • Baseline through Day 112
|
|
General disorders
Oedema peripheral
|
12.5%
1/8 • Baseline through Day 112
|
|
General disorders
Pyrexia
|
12.5%
1/8 • Baseline through Day 112
|
|
Nervous system disorders
Headache
|
50.0%
4/8 • Baseline through Day 112
|
|
Nervous system disorders
Dizziness
|
25.0%
2/8 • Baseline through Day 112
|
|
Nervous system disorders
Dizziness postural
|
12.5%
1/8 • Baseline through Day 112
|
|
Nervous system disorders
Hypersomnia
|
12.5%
1/8 • Baseline through Day 112
|
|
Nervous system disorders
Syncope
|
12.5%
1/8 • Baseline through Day 112
|
|
Gastrointestinal disorders
Constipation
|
25.0%
2/8 • Baseline through Day 112
|
|
Gastrointestinal disorders
Nausea
|
25.0%
2/8 • Baseline through Day 112
|
|
Gastrointestinal disorders
Abdominal pain
|
12.5%
1/8 • Baseline through Day 112
|
|
Infections and infestations
Pneumonia
|
12.5%
1/8 • Baseline through Day 112
|
|
Infections and infestations
Rhinitis
|
12.5%
1/8 • Baseline through Day 112
|
|
Infections and infestations
Upper respiratory tract infection
|
12.5%
1/8 • Baseline through Day 112
|
|
Infections and infestations
Urinary tract infection
|
12.5%
1/8 • Baseline through Day 112
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
25.0%
2/8 • Baseline through Day 112
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.5%
1/8 • Baseline through Day 112
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
12.5%
1/8 • Baseline through Day 112
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
12.5%
1/8 • Baseline through Day 112
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
12.5%
1/8 • Baseline through Day 112
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
12.5%
1/8 • Baseline through Day 112
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
12.5%
1/8 • Baseline through Day 112
|
|
Blood and lymphatic system disorders
Anaemia
|
12.5%
1/8 • Baseline through Day 112
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
12.5%
1/8 • Baseline through Day 112
|
|
Cardiac disorders
Sinus tachycardia
|
25.0%
2/8 • Baseline through Day 112
|
|
Injury, poisoning and procedural complications
Fall
|
12.5%
1/8 • Baseline through Day 112
|
|
Injury, poisoning and procedural complications
Infusion-related reaction
|
12.5%
1/8 • Baseline through Day 112
|
|
Injury, poisoning and procedural complications
Limb injury
|
12.5%
1/8 • Baseline through Day 112
|
|
Injury, poisoning and procedural complications
Post-traumatic pain
|
12.5%
1/8 • Baseline through Day 112
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
12.5%
1/8 • Baseline through Day 112
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
12.5%
1/8 • Baseline through Day 112
|
|
Eye disorders
Blepharospasm
|
12.5%
1/8 • Baseline through Day 112
|
|
Investigations
Haematocrit decreased
|
12.5%
1/8 • Baseline through Day 112
|
|
Investigations
Haemoglobin decreased
|
12.5%
1/8 • Baseline through Day 112
|
|
Metabolism and nutrition disorders
Increased appetite
|
12.5%
1/8 • Baseline through Day 112
|
|
Psychiatric disorders
Restlessness
|
12.5%
1/8 • Baseline through Day 112
|
|
Vascular disorders
Flushing
|
12.5%
1/8 • Baseline through Day 112
|
Additional Information
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee Sponsor can review results communications at least 60 days prior to public release. Within 30 days of receipt, Sponsor shall advise of any information which is Confidential Information (other than Study Data) or which may impair the availability of patent protection for Inventions. Sponsor can require removal of specifically identified Confidential Information (other than Study Data) and/or delay the communication an additional 60 days to enable Sponsor to seek patent protection for Inventions.
- Publication restrictions are in place
Restriction type: OTHER