Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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TERMINATED
PHASE3
162 participants
INTERVENTIONAL
2019-11-18
2024-11-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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.
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).
Interventions
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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 Intervention Names
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Eligibility Criteria
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Inclusion Criteria
Note: Participants 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.
Note: Participants who discontinued Study GBT440-032 as the result of an abnormal transcranial Doppler (TCD) flow velocity assessment (≥ 200 cm/sec) are eligible for treatment in this study.
\- Participant has provided written consent/assent (for pediatric participants, both the consent of the participant's legal representative or legal guardian and the participant's assent \[where applicable\] must be obtained).
Exclusion Criteria
* Participant withdrew consent from a GBT-sponsored voxelotor clinical study
* Known hypersensitivity to voxelotor or any other components of the study drug
* Use of St. John's wort, sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or moderate or strong CYP3A4 inducers within 30 days of Day 1
6 Months
ALL
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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Children's National Medical Center
Washington D.C., District of Columbia, United States
Children's Healthcare of Atlanta Scottish Rite
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
ECU Physicians, Brody Outpatient Clinic
Greenville, North Carolina, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Zagazig University Hospital
Zagazig, Alsharkia, Egypt
Alexandria University Hospital - Clinical Research Center
Alexandria, , Egypt
Cairo University Hospital, Abu El Rish Hospital
Cairo, , Egypt
Ain Shams University Hospital - Clinical Research Center (MASRI-CRC)
Cairo, , Egypt
American University of Beirut - Medical Center
Beirut, , Lebanon
Nini Hospital
Tripoli, , Lebanon
University of Calabar Teaching Hospital
Calabar, Cross River State, Nigeria
College of Medicine, University of Ibadan
Ibadan, Oyo State, Nigeria
Barau Dikko Teaching Hospital/Kaduna State University
Kaduna, , Nigeria
Aminu Kano Teaching Hospital
Kano, , Nigeria
Lagos University Teaching Hospital
Lagos, , Nigeria
University College Hospital NHS Foundation Trust
London, Greater London, United Kingdom
Barts Health NHS Trust , The Royal London Hospital
London, , United Kingdom
Guy'S and St Thomas' Nhs Foundation Trust
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain contact information for a study center near you, click here.
Other Identifiers
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2019-003144-76
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
C5341023
Identifier Type: OTHER
Identifier Source: secondary_id
GBT440-038
Identifier Type: -
Identifier Source: org_study_id