Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease (HOPE Kids 2)
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|ClinicalTrials.gov Identifier: NCT04218084|
Recruitment Status : Not yet recruiting
First Posted : January 6, 2020
Last Update Posted : January 6, 2020
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Voxelotor Drug: Placebos||Phase 3|
Expanded Access : Global Blood Therapeutics has indicated that access to an investigational treatment associated with this study is available outside the clinical trial.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||224 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||Double-Blind, Placebo-Controlled|
|Official Title:||A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants With Sickle Cell Disease|
|Estimated Study Start Date :||February 2020|
|Estimated Primary Completion Date :||March 2026|
|Estimated Study Completion Date :||March 2026|
Voxelotor 1500mg or equivalent daily as a tablet or as powder for oral suspension.
Participants are randomized 1:1 to receive voxelotor or placebo.
Placebo Comparator: Placebo
- TCD Measurement [ Time Frame: 24 weeks ]The change in time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow, as measured by TCD.
- Change in TCD Flow Velocity [ Time Frame: 48 and 96 weeks ]
- Conversion to Abnormal TCD Flow Velocity [ Time Frame: 24, 48, and 96 weeks ]Incidence of conversion to abnormal TCD flow velocity (≥ 200 cm/sec)
- Reversion to Normal TCD Flow Velocity [ Time Frame: 24, 48, and 96 weeks ]Incidence of reversion to normal TCD flow velocity (<170 cm/sec)
- TCD Flow Velocity Reduction [ Time Frame: 24, 48, and 96 weeks ]Proportion of participants with TCD flow velocity reduction ≥ 15 cm/sec
- Number of Participants with Change in Hemoglobin (Hb) [ Time Frame: 24, 48, and 96 weeks ]
- Change in Unconjugated Bilirubin [ Time Frame: 24, 48 and 96 weeks ]
- Change in Percent of Reticulocyte [ Time Frame: 24, 48, and 96 weeks ]
- Change in Absolute Reticulocyte [ Time Frame: 24, 48, and 96 weeks ]
- Change in Lactate Dehydrogenase (LDH) [ Time Frame: 24, 48, and 96 weeks ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04218084
|Contact: Dan Rudin, MD||(650) email@example.com|
|Contact: Aimee Enriquez||(650) firstname.lastname@example.org|