Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease (HOPE Kids 2)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04218084|
Recruitment Status : Recruiting
First Posted : January 6, 2020
Last Update Posted : December 22, 2021
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Voxelotor Drug: Placebos||Phase 3|
Expanded Access : An investigational treatment associated with this study is available outside the clinical trial. More info ...
|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|
|Actual Study Start Date :||April 7, 2021|
|Estimated Primary Completion Date :||March 2026|
|Estimated Study Completion Date :||March 2026|
Voxelotor 1500mg or equivalent daily as a tablet, dispersible 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: Margaret Tonda, PharmD||(650) firstname.lastname@example.org|
|Contact: Stacy Tsukayamaemail@example.com|