Open-Label Extension of Voxelotor
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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. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04188509 |
Recruitment Status :
Enrolling by invitation
First Posted : December 6, 2019
Last Update Posted : September 29, 2022
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Condition or disease | Intervention/treatment | Phase |
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Sickle Cell Disease | Drug: Voxelotor | 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 : | 600 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | Open-Label Extension study available to eligible participants from GBT-sponsored voxelotor clinical studies. |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-Label Extension Study of Voxelotor Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials |
Actual Study Start Date : | November 18, 2019 |
Estimated Primary Completion Date : | January 2026 |
Estimated Study Completion Date : | January 2026 |

Arm | Intervention/treatment |
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Experimental: Voxelotor
All participants will receive voxelotor once daily (QD), administered orally as tablets, dispersible tablets, or a stick pack formulation (powder blend formulation packaged as stick packs). Participants aged ≥ 12 years and/or ≥ 40 kgs will receive a voxelotor dose of 1500 mg QD. Participants aged < 12 years and < 40 kgs will receive weight based dosing of voxelotor. The participant's weight at study entry will be used to determine the starting voxelotor dose in this study. Participants may receive study drug as long they continue to receive clinical benefit that outweighs risk as determined by the investigator and/or until the participant has access to voxelotor from an alternative source.
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Drug: Voxelotor
All participants will receive voxelotor once daily (QD), administered orally as tablets, dispersible tablets, or a powder for oral suspension formulation (powder formulation packaged as stick packs).
Other Name: GBT440 |
- TEAEs and SAEs [ Time Frame: Throughout entire study ]Treatment Emergent Adverse Events and Serious Adverse Events
- Sickle Cell Disease-Related Complications [ Time Frame: Throughout entire study ]Frequency of SCD-related complications

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 6 Months and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male or female participant with SCD, who participated and received study drug in a GBT-sponsored voxelotor clinical study
- Participant has provided written assent (both the consent of the participant's legal representative or legal guardian and the participant's assent [where applicable] must be obtained) or consent
Exclusion Criteria:
- Female participant who is breastfeeding or pregnant
- Participant withdrew consent from a GBT-sponsored voxelotor clinical study
- Known hypersensitivity to voxelotor or any other components of the study drug
- Use of St. John's wort, sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or moderate or strong CYP3A4 inducers within 30 days of Day 1

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): NCT04188509
United States, District of Columbia | |
Children's National Medical Center | |
Washington, District of Columbia, United States, 20010 | |
United States, Georgia | |
Children's Healthcare of Atlanta | |
Atlanta, Georgia, United States, 30342 | |
United States, Illinois | |
Ann and Robert Lurie Children's Hospital of Chicago | |
Chicago, Illinois, United States, 60611 | |
United States, Missouri | |
Children's Mercy Hospital Kansas City | |
Kansas City, Missouri, United States, 64108 | |
United States, North Carolina | |
East Carolina University Brody School of Medicine | |
Greenville, North Carolina, United States, 27835 | |
United States, Pennsylvania | |
The Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 | |
Children's Hospital of Pittsburgh of UPMC - Blood and Marrow Transplantation (BMT) and Cellular Therapies | |
Pittsburgh, Pennsylvania, United States, 15224 | |
Lebanon | |
American University of Beirut Medical Center (AUBMC) | |
Beirut, Lebanon, 1107-2020 | |
Nini Hospital | |
Tripoli, Lebanon, 1300 | |
United Kingdom | |
Royal London Hospital, Barts Health NHS Trust | |
London, United Kingdom, E1 1BB | |
University College London Hospitals NHS Foundation Trust | |
London, United Kingdom, NW1 2PG |
Study Director: | Mark Davis, MS | Global Blood Therapeutics, Inc. |
Responsible Party: | Global Blood Therapeutics |
ClinicalTrials.gov Identifier: | NCT04188509 |
Other Study ID Numbers: |
GBT440-038 |
First Posted: | December 6, 2019 Key Record Dates |
Last Update Posted: | September 29, 2022 |
Last Verified: | September 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia |
Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |