A Phase 2 interventional study of GBT440 in Sickle Cell Disease, sponsored by Global Blood Therapeutics. Completed at 1 site in United Kingdom. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2019-01-02.
Sponsored by Global Blood Therapeutics · Phase 2, Interventional, and Other
This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
Dosing of study drug was 2 to 6 months, depending on subject's dose assignment in the last administration of study drug in GBT440-001 (NCT02285088).
The primary objective of the study was to evaluate the safety and tolerability of up to a total of 6 months dosing of subjects with SCD who participated in the GBT440-001 study (NCT02285088).
1,104 studies on the registry are indexed under Anemia, Sickle Cell; 236 are open to participants now.
This study's enrollment of 5 is below the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Global Blood Therapeutics is the lead sponsor of 9 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Single Arm / open label
Drug: GBT440
Oral drug
Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
Time frame: 2 - 6 months
To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia
Data presented are hemoglobin value collected at specific time points.
Time frame: 2 - 6 months
To Observed Pharmacokinetics in Plasma and Whole Blood.
Measure maximum plasma concentration (Cmax)
Time frame: 2 - 6 months
To Characterize the Effect of GBT440 on Hemolysis.
Data presented for unconjugated bilirubin at specific time point.
Time frame: 2 - 6 months
Subjects enrolled in this study participated in GBT440-001 study (NCT02285088).
| Milestone | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months |
|---|---|---|---|
| Started | 3 | 1 | 1 |
| Completed | 3 | 1 | 1 |
| Not completed | 0 | 0 | 0 |
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
| Participants | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months |
|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months. | 2 | 1 | 1 |
Data presented are hemoglobin value collected at specific time points.
| g/dL | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months |
|---|---|---|---|
| To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia | NA | NA | NA |
Measure maximum plasma concentration (Cmax)
| ug/mL | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months |
|---|---|---|---|
| To Observed Pharmacokinetics in Plasma and Whole Blood. | NA | NA | NA |
Data presented for unconjugated bilirubin at specific time point.
| umol/L | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months |
|---|---|---|---|
| To Characterize the Effect of GBT440 on Hemolysis. | NA | NA | NA |
Collected over 10 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| GBT440 - 2 Months | 0/3 (0%) | 0/3 (0%) | 2/3 (66.7%) |
| GBT440 - 6 Months | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| GBT440 - 4 Months | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| Event | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months |
|---|---|---|---|
| Sickle Cell Anemia with CrisisBlood and lymphatic system disorders | 0/3 | 1/1 | 0/1 |
| CholecystectomySurgical and medical procedures | 0/3 | 0/1 | 1/1 |
| Event | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months |
|---|---|---|---|
| DiarrheaGastrointestinal disorders | 1/3 | 0/1 | 1/1 |
| ToothacheGastrointestinal disorders | 0/3 | 1/1 | 0/1 |
| Upper Respiratory Tract InfectionInfections and infestations | 1/3 | 0/1 | 1/1 |
| HeadacheNervous system disorders | 0/3 | 0/1 | 1/1 |
| Gingival SwellingGastrointestinal disorders | 0/3 | 1/1 | 0/1 |
| Abdominal PainGastrointestinal disorders | 1/3 | 0/1 | 0/1 |
| Age, Continuous(Years) | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months | Total |
|---|---|---|---|---|
| Mean | 35 (25 to 42) | 26 | 21 | 30 (21 to 42) |
| Sex: Female, Male(Participants) | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months | Total |
|---|---|---|---|---|
| Female | 0 | 0 | 1 | 1 |
| Male | 3 | 1 | 0 | 4 |
| Race (NIH/OMB)(Participants) | GBT440 - 2 Months | GBT440 - 6 Months | GBT440 - 4 Months | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 3 | 1 | 1 | 5 |
| White | 0 | 0 | 0 | 0 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes
Supporting information: Study protocol
This study is completed, as verified in Dec 2018. You cannot join it, but the record below documents what was studied.
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Global Blood Therapeutics