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Open-Label Extension of Voxelotor

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
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Study Description
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Brief Summary:
Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Voxelotor Phase 3

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

Detailed Description:
Open-label extension (OLE) study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials. Up to approximately 600 participants with sickle cell disease (SCD), will be enrolled at approximately 70 clinical sites globally. All participants will receive voxelotor once daily, administered orally as tablets, dispersible tablets, or powder for oral suspension formulation. The objective of this OLE is to assess the safety of, and SCD-related complications of, long-term treatment with voxelotor, in participants who have completed treatment in a Global Blood Therapeutics (GBT)-sponsored voxelotor clinical study.
Study Design
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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

Resource links provided by the National Library of Medicine

Drug Information available for: Voxelotor

Arms and Interventions
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Arm Intervention/treatment
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.
 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


Outcome Measures
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Primary Outcome Measures :
  1. TEAEs and SAEs [ Time Frame: Throughout entire study ]
    Treatment Emergent Adverse Events and Serious Adverse Events

  2. Sickle Cell Disease-Related Complications [ Time Frame: Throughout entire study ]
    Frequency of SCD-related complications


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Mark Davis, MS Global Blood Therapeutics, Inc.
More Information
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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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn