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Pediatric Open-Label Extension of Voxelotor

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ClinicalTrials.gov Identifier: NCT04188509
Recruitment Status : Enrolling by invitation
First Posted : December 6, 2019
Last Update Posted : December 6, 2019
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Study Description
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Brief Summary:
Open-label extension study of voxelotor for pediatric participants ages 4 to 18 years old 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 has been approved for sale to the public.   More info ...

Detailed Description:
Open-label extension (OLE) study of voxelotor for pediatric participants with Sickle Cell Disease who have participated in voxelotor clinical trials. Approximately 50 participants with sickle cell disease (SCD), aged ≥ 4 to ≤ 18 years will be enrolled at approximately 19 global clinical sites. 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 pediatric participants who have completed treatment in a Global Blood Therapeutics (GBT)-sponsored voxelotor pediatric clinical study.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Open-Label Extension study available to eligible participants from GBT-sponsored voxelotor pediatric clinical studies.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study of Voxelotor Administered Orally to Pediatric 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 will receive a voxelotor dose of 1500 mg QD, regardless of their body weight. Participants aged < 12 years will receive a voxelotor dose based on their body weight, to provide exposure corresponding to the adult dose of 1500 mg QD. The participant's weight at study entry will be used to determine the starting voxelotor dose in this study. The dose should be adjusted if the participant's weight increases or decreases over 2 consecutive clinic visits.

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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Ages Eligible for Study:   4 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female participant with SCD, aged ≥ 4 to ≤ 18 years, who participated and received study drug in a GBT-sponsored voxelotor pediatric 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)

Exclusion Criteria:

  • Female participant who is breastfeeding or pregnant
  • Participant withdrew consent from a GBT-sponsored voxelotor pediatric clinical study
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, Louisiana
Our Lady of the Lake Regional Medical Center
Baton Rouge, Louisiana, United States, 70808
United States, Missouri
Children's Mercy Hospital Kansas City
Kansas City, Missouri, United States, 64108
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC - Blood and Marrow Transplantation (BMT) and Cellular Therapies
Pittsburgh, Pennsylvania, United States, 15224
United States, Tennessee
St. Jude Children's Research Hospital (SJCRH)
Memphis, Tennessee, United States, 38105
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
Evelina London Children's Hospital - Guy's and St. Thomas' NHS Foundation Trust
London, United Kingdom, SE1 7EH
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Nicholas Vlahakis, MD 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: December 6, 2019
Last Verified: December 2019

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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