Trial Outcomes & Findings for An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001 (NCT NCT03041909)
NCT ID: NCT03041909
Last Updated: 2019-01-02
Results Overview
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
5 participants
Primary outcome timeframe
2 - 6 months
Results posted on
2019-01-02
Participant Flow
Subjects enrolled in this study participated in GBT440-001 study (NCT02285088).
There was no screening period as subjects transitioned directly from the GBT440-001 study (NCT02285088).into this study.
Participant milestones
| Measure |
GBT440 - 2 Months
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).
|
GBT440 - 6 Months
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
|
GBT440 - 4 Months
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
|
|---|---|---|---|
|
Overall Study
STARTED
|
3
|
1
|
1
|
|
Overall Study
COMPLETED
|
3
|
1
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001
Baseline characteristics by cohort
| Measure |
GBT440 - 2 Months
n=3 Participants
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (NCT02285088) (n=3).
|
GBT440 - 6 Months
n=1 Participants
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (NCT02285088) (n=1).
|
GBT440 - 4 Months
n=1 Participants
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (NCT02285088) (n=1).
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
35 Years
n=5 Participants
|
26 Years
n=7 Participants
|
21 Years
n=5 Participants
|
30 Years
n=4 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 2 - 6 monthsPopulation: Safety
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
Outcome measures
| Measure |
GBT440 - 2 Months
n=3 Participants
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).
|
GBT440 - 6 Months
n=1 Participants
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
|
GBT440 - 4 Months
n=1 Participants
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
|
|---|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.
|
2 Participants
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 2 - 6 monthsData presented are hemoglobin value collected at specific time points.
Outcome measures
| Measure |
GBT440 - 2 Months
n=3 Participants
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).
|
GBT440 - 6 Months
n=1 Participants
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
|
GBT440 - 4 Months
n=1 Participants
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
|
|---|---|---|---|
|
To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia
|
NA g/dL
Data is not reported due to participant privacy.
|
NA g/dL
Data is not reported due to participant privacy.
|
NA g/dL
Data is not reported due to participant privacy.
|
SECONDARY outcome
Timeframe: 2 - 6 monthsMeasure maximum plasma concentration (Cmax)
Outcome measures
| Measure |
GBT440 - 2 Months
n=3 Participants
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).
|
GBT440 - 6 Months
n=1 Participants
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
|
GBT440 - 4 Months
n=1 Participants
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
|
|---|---|---|---|
|
To Observed Pharmacokinetics in Plasma and Whole Blood.
|
NA ug/mL
Data is not reported due to participant privacy.
|
NA ug/mL
Data is not reported due to participant privacy.
|
NA ug/mL
Data is not reported due to participant privacy.
|
SECONDARY outcome
Timeframe: 2 - 6 monthsData presented for unconjugated bilirubin at specific time point.
Outcome measures
| Measure |
GBT440 - 2 Months
n=3 Participants
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).
|
GBT440 - 6 Months
n=1 Participants
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
|
GBT440 - 4 Months
n=1 Participants
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
|
|---|---|---|---|
|
To Characterize the Effect of GBT440 on Hemolysis.
|
NA umol/L
Data is not reported due to participant privacy.
|
NA umol/L
Data is not reported due to participant privacy.
|
NA umol/L
Data is not reported due to participant privacy.
|
Adverse Events
GBT440 - 2 Months
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
GBT440 - 6 Months
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
GBT440 - 4 Months
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
GBT440 - 2 Months
n=3 participants at risk
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (NCT02285088) (n=3).
|
GBT440 - 6 Months
n=1 participants at risk
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (NCT02285088) (n=1).
|
GBT440 - 4 Months
n=1 participants at risk
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (NCT02285088) (n=1).
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Sickle Cell Anemia with Crisis
|
0.00%
0/3 • 10 months
|
100.0%
1/1 • Number of events 1 • 10 months
|
0.00%
0/1 • 10 months
|
|
Surgical and medical procedures
Cholecystectomy
|
0.00%
0/3 • 10 months
|
0.00%
0/1 • 10 months
|
100.0%
1/1 • Number of events 1 • 10 months
|
Other adverse events
| Measure |
GBT440 - 2 Months
n=3 participants at risk
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (NCT02285088) (n=3).
|
GBT440 - 6 Months
n=1 participants at risk
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (NCT02285088) (n=1).
|
GBT440 - 4 Months
n=1 participants at risk
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (NCT02285088) (n=1).
|
|---|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
1/3 • Number of events 1 • 10 months
|
0.00%
0/1 • 10 months
|
100.0%
1/1 • Number of events 1 • 10 months
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/3 • 10 months
|
100.0%
1/1 • Number of events 1 • 10 months
|
0.00%
0/1 • 10 months
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
33.3%
1/3 • Number of events 1 • 10 months
|
0.00%
0/1 • 10 months
|
100.0%
1/1 • Number of events 1 • 10 months
|
|
Gastrointestinal disorders
Abdominal Pain
|
33.3%
1/3 • Number of events 1 • 10 months
|
0.00%
0/1 • 10 months
|
0.00%
0/1 • 10 months
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • 10 months
|
0.00%
0/1 • 10 months
|
100.0%
1/1 • Number of events 1 • 10 months
|
|
Gastrointestinal disorders
Gingival Swelling
|
0.00%
0/3 • 10 months
|
100.0%
1/1 • Number of events 1 • 10 months
|
0.00%
0/1 • 10 months
|
Additional Information
Margaret Tonda, Sr. Director, Clinical Science
Global Blood Therapeutics
Phone: 650-741-7761
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee The sponsor and the PI will have to agree on any publications regarding the study.
- Publication restrictions are in place
Restriction type: OTHER