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Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031 (034OLE)

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: NCT03573882
Recruitment Status : Active, not recruiting
First Posted : June 29, 2018
Last Update Posted : March 17, 2020
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Brief Summary:
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who 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:

This open label extension (OLE), multi-center study will be conducted at approximately 100 clinical sites globally and will be available to eligible participants from study GBT440-031.

The study will enroll participants from GBT440-031 (approximately 435) under any of the following conditions:

  • Participant has completed 72 weeks of treatment regardless of dose selection for GBT440-031
  • Dose selection has occurred for GBT440-031 and participant is on non-selected dose on GBT440-031
  • GBT440-031 study interim data analysis and/or study modifications have occurred
  • GBT440-031 study has completed

The objective of this open-label extension (OLE) study is to assess the long-term safety and treatment effect of voxelotor in participants who have completed treatment in study GBT440-031, using the following parameters:

  1. Safety based upon AEs, clinical laboratory tests, physical examinations (PE) and other clinical measures.
  2. Frequency of sickle cell disease (SCD)-related complications.
  3. Hemolytic anemia as measured by hematological laboratory parameters (e.g. hemoglobin, reticulocytes and unconjugated bilirubin).

All participants will receive daily voxelotor treatment.

Participants may receive study drug as long they continue to receive clinical benefit which outweighs risk as determined by the Investigator and/or until the participant has access to voxelotor from an alternative source (i.e., commercialization or through a managed access program).

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 179 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
Actual Study Start Date : June 6, 2018
Estimated Primary Completion Date : October 2024
Estimated Study Completion Date : October 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Voxelotor
Participants will receive voxelotor (GBT440) at the highest dose (either 900 mg or 1500 mg) deemed safe by the Data Safety Monitoring Board (DSMB).
Drug: Voxelotor
300mg or 500mg Tablet, Oral, With or Without Food
Other Name: GBT440




Primary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [ Time Frame: Five Years ]
    Safety based on Adverse Events

  2. Frequency of sickle cell-related complications [ Time Frame: Five Years ]
    Frequency of SCD-related complications with long-term dosing with voxelotor.


Secondary Outcome Measures :
  1. Response in Hemolytic Anemia [ Time Frame: Five Years ]
    Measured by Hemoglobin, Bilirubin, and Reticulocyte counts



Information from the National Library of Medicine

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.


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female study participants with SCD who participated and received study treatment in Study GBT440-031.

Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor.

  • Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
  • Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must use barrier methods of contraception to 30 days after the last dose of study drug.
  • Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant's legal representative or legal guardian, and the participant's assent must be obtained).

Exclusion Criteria:

  • Female who is breast-feeding or pregnant.
  • Participant withdrew consent from Study GBT440-031.
  • Participant was lost to follow-up from Study GBT440-031.
  • Participant requiring chronic dialysis.
  • Any medical, psychological, safety, or behavioral conditions, which, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.

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): NCT03573882


Locations
Show Show 48 study locations
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Margaret Tonda, PharmD Global Blood Therapeutics
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Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT03573882    
Other Study ID Numbers: GBT440-034
First Posted: June 29, 2018    Key Record Dates
Last Update Posted: March 17, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Global Blood Therapeutics:
Open Label Extension
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn