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Pediatric 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 : April 29, 2021
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Tracking Information
First Submitted Date  ICMJE December 3, 2019
First Posted Date  ICMJE December 6, 2019
Last Update Posted Date April 29, 2021
Actual Study Start Date  ICMJE November 18, 2019
Estimated Primary Completion Date January 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 3, 2019)
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pediatric Open-Label Extension of Voxelotor
Official Title  ICMJE An Open-Label Extension Study of Voxelotor Administered Orally to Pediatric Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
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.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE 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
Study Arms  ICMJE 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.

Intervention: Drug: Voxelotor
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: December 3, 2019)
50
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2026
Estimated Primary Completion Date January 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Lebanon,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04188509
Other Study ID Numbers  ICMJE GBT440-038
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Global Blood Therapeutics
Study Sponsor  ICMJE Global Blood Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Margaret Tonda, PharmD Global Blood Therapeutics, Inc.
PRS Account Global Blood Therapeutics
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP