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

Sponsor
Global Blood Therapeutics (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03573882
Collaborator
(none)
179
Enrollment
48
Locations
1
Arm
75.9
Anticipated Duration (Months)
3.7
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

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
Phase 3

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).

Study Design

Study Type:
Interventional
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 :
Jun 6, 2018
Anticipated Primary Completion Date :
Oct 1, 2024
Anticipated Study Completion Date :
Oct 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Other: 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 Names:
  • GBT440

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [Five Years]

      Safety based on Adverse Events

    2. Frequency of sickle cell-related complications [Five Years]

      Frequency of SCD-related complications with long-term dosing with voxelotor.

    Secondary Outcome Measures

    1. Response in Hemolytic Anemia [Five Years]

      Measured by Hemoglobin, Bilirubin, and Reticulocyte counts

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Arkansas Primary Care Clinic, PA Little Rock Arkansas United States 72204
    2 UCSF Benioff Children's Hospital Oakland Oakland California United States 94609
    3 University of Miami Medical Center Miami Florida United States 33136
    4 Emory University School of Medicine Atlanta Georgia United States 30342
    5 University of Illinois College of Medicine Chicago Illinois United States 60612
    6 Indiana Hemophilia and Thrombosis Center Indianapolis Indiana United States 46260
    7 Our Lady of the Lake Hospital, Inc. Baton Rouge Louisiana United States 70809
    8 University Medical Center New Orleans New Orleans Louisiana United States 70112
    9 The Johns Hopkins Hospital Baltimore Maryland United States 21287
    10 Brigham and Women's Hospital Department of Neurology Boston Massachusetts United States 02115
    11 Boston University Medical Center Boston Massachusetts United States 02118
    12 Wayne State University Detroit Michigan United States 48201
    13 Newark Beth Israel Medical Center Newark New Jersey United States 07112
    14 Montefiore Medical Center Bronx New York United States 10467
    15 Columbia University Medical Center New York New York United States 10032
    16 University of North Carolina at Chapel Hill Chapel Hill North Carolina United States 27599
    17 Duke University Durham North Carolina United States 27710
    18 Lynn Health Science Institute Oklahoma City Oklahoma United States 73112
    19 Thomas Jefferson University Hospital Philadelphia Pennsylvania United States 19107
    20 University of Pittsburgh Medical Center Health System Pittsburgh Pennsylvania United States 15232
    21 Medical University of South Carolina Charleston South Carolina United States 29425
    22 Methodist Healthcare Foundation Memphis Tennessee United States 38104
    23 Texas Children's Hospital Houston Texas United States 77030
    24 VCU Health - Research Parent Richmond Virginia United States 23298
    25 University Health Network Toronto Ontario Canada M5G 2C4
    26 Zagazig University Hospital Al Sharkeya Egypt 44519
    27 Alexandria University Hospital Alexandria Egypt 21131
    28 Cairo University Hospital- Abou El Rish Cairo Egypt 00000
    29 Ain Shams University Hospital Cairo Egypt 11566
    30 Hôpital Européen Georges Pompidou Paris France 75015
    31 Azienda Ospedaliera di Padova Padova Italy 35128
    32 Gertrude's Children's Hospital Nairobi Kenya 00200
    33 KEMRI - Centre for Respiratory Disease Research (CRDR) Nairobi Kenya 00200
    34 KEMRI (Kenya Medical Research Institute) Kisumu Siaya Kenya 144 - 40600
    35 American University of Beirut Medical Center Beirut Lebanon 1107 2020
    36 Nini Hospital s a l Tripoli Lebanon 1434
    37 Amsterdam UMC, Locatie AMC Amsterdam Netherlands 1105 AZ
    38 Erasmus Medisch Centrum Rotterdam Netherlands 3015 CE
    39 Sultan َQaboos University Hospital Muscat Oman 123
    40 Acibadem Adana Hospital Adana Turkey 01130
    41 Erciyes University Medical Faculty Kayseri Turkey 38039
    42 Mersin University Medical Faculty Mersin Turkey 33079
    43 Royal London Hospital London Greater London United Kingdom E1 1BB
    44 Homerton University Hospital London Greater London United Kingdom E9 6SR
    45 St Thomas' Hospital London Greater London United Kingdom SE1 7EH
    46 King's College Hospital London Greater London United Kingdom SE5 9NU
    47 University College London Hospitals London Greater London United Kingdom WC1N 3BG
    48 Hammersmith Hospital London United Kingdom W12 0HS

    Sponsors and Collaborators

    • Global Blood Therapeutics

    Investigators

    • Study Director: Margaret Tonda, PharmD, Global Blood Therapeutics

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Global Blood Therapeutics
    ClinicalTrials.gov Identifier:
    NCT03573882
    Other Study ID Numbers:
    • GBT440-034
    First Posted:
    Jun 29, 2018
    Last Update Posted:
    May 26, 2021
    Last Verified:
    May 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    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:
    Anemia, Sickle Cell

    Study Results

    No Results Posted as of May 26, 2021