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Pediatric Open-Label Extension of Voxelotor

Sponsor
Global Blood Therapeutics (Industry)
Overall Status
Enrolling by invitation
CT.gov ID
NCT04188509
Collaborator
(none)
300
Enrollment
10
Locations
1
Arm
73.5
Anticipated Duration (Months)
30
Patients Per Site
0.4
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Open-label extension study of voxelotor for pediatric participants up to 18 years old with Sickle Cell Disease who have participated in voxelotor clinical trials.

Condition or Disease Intervention/Treatment Phase
Phase 3

Detailed Description

Open-label extension (OLE) study of voxelotor for pediatric participants with Sickle Cell Disease who have participated in voxelotor clinical trials. Approximately 300 participants with sickle cell disease (SCD), aged ≤ 18 years, will be enrolled at approximately 45 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 pediatric participants who have completed treatment in a Global Blood Therapeutics (GBT)-sponsored voxelotor pediatric clinical study.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
300 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.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 :
Nov 18, 2019
Anticipated Primary Completion Date :
Jan 1, 2026
Anticipated Study Completion Date :
Jan 1, 2026

Arms and Interventions

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 Names:
  • GBT440

    		•

    Outcome Measures

    Primary Outcome Measures

    1. TEAEs and SAEs [Throughout entire study]

      Treatment Emergent Adverse Events and Serious Adverse Events

    2. Sickle Cell Disease-Related Complications [Throughout entire study]

      Frequency of SCD-related complications

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Male or female participant with SCD, up 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

    • Known hypersensitivity to voxelotor or any other components of the study drug

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Children's National Medical Center Washington District of Columbia United States 20010
    2 Children's Healthcare of Atlanta Atlanta Georgia United States 30342
    3 Ann and Robert Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    4 Children's Mercy Hospital Kansas City Kansas City Missouri United States 64108
    5 East Carolina University Brody School of Medicine Greenville North Carolina United States 27835
    6 Children's Hospital of Pittsburgh of UPMC - Blood and Marrow Transplantation (BMT) and Cellular Therapies Pittsburgh Pennsylvania United States 15224
    7 American University of Beirut Medical Center (AUBMC) Beirut Lebanon 1107-2020
    8 Nini Hospital Tripoli Lebanon 1300
    9 Royal London Hospital, Barts Health NHS Trust London United Kingdom E1 1BB
    10 University College London Hospitals NHS Foundation Trust London United Kingdom NW1 2PG

    Sponsors and Collaborators

    • Global Blood Therapeutics

    Investigators

    • Study Director: Mark Davis, MS, Global Blood Therapeutics, Inc.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Global Blood Therapeutics
    ClinicalTrials.gov Identifier:
    NCT04188509
    Other Study ID Numbers:
    • GBT440-038
    First Posted:
    Dec 6, 2019
    Last Update Posted:
    Jul 12, 2022
    Last Verified:
    Jul 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Anemia, Sickle Cell

    Study Results

    No Results Posted as of Jul 12, 2022