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

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ClinicalTrials.gov Identifier: NCT03573882
Recruitment Status : Enrolling by invitation
First Posted : June 29, 2018
Last Update Posted : July 11, 2019
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Brief Summary:
Open Label Extension Study of GBT440 Clinical Trial Patients with Sickle Cell Disease Who Participated in GBT440 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:

This international open-label extension (OLE) study will be available to eligible participants from Study GBT440-031. The study will be conducted at approximately 100 clinical sites.

All participants in GBT440-031 who completed 72 weeks of treatment are eligible for this study. Depending on their Group assignment in GBT440-031, participants may be eligible for this study prior to the completion of 72 weeks of treatment; see the GBT440-031 protocol for additional information.

All participants (approximately 435) who have received treatment in Study GBT440-031 and who meet the eligibility criteria for this study may be enrolled.

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

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

All participants will receive daily GBT440 treatment.

If both the 900 and 1500 mg dose are maintained for the GBT440-031 study's Main Population, then participants will continue receiving the GBT440 dose they were randomized to in the GBT440-031 study. Participants who received placebo in GBT440-031 will be re-randomized 1:1 to receive GBT440 900 mg or 1500 mg in this study. If one dose is selected from the Main Population analysis, this dose may be used for all participants.

Duration of 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 GBT440 from an alternative source (i.e., commercialization or through a managed access program).


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 435 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Extension Study of GBT440 Administered Orally to Patients With Sickle Cell Disease Who Have Participated in GBT440 Clinical Trials
Actual Study Start Date : June 6, 2018
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Active Comparator: 900 mg
Voxelotor (GBT440) 900 mg
Drug: Voxelotor
300 mg Tablet, Oral, With or Without Food
Other Name: GBT440

Active Comparator: 1500 mg
Voxelotor (GBT440) 1500 mg
Drug: Voxelotor
300 mg Tablet, Oral, With or Without Food
Other Name: GBT440




Primary Outcome Measures :
  1. Hemolysis Markers [ Time Frame: Five Years ]
    Measured by total bilirubin, LDH and reticulocyte lab values

  2. Frequency of sickle cell-related complications [ Time Frame: Five Years ]
    Frequency of SCD-related complications with long-term dosing with GBT440.


Secondary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: Five Years ]
    Safety based on Adverse Events



Information from the National Library of Medicine

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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 GBT440.

  • 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 continue to 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


  Show 25 Study Locations
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Dan Rudin, MD Global Blood Therapeutics

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Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT03573882     History of Changes
Other Study ID Numbers: GBT440-034
First Posted: June 29, 2018    Key Record Dates
Last Update Posted: July 11, 2019
Last Verified: July 2019
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