Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
NCT ID: NCT02850406
Last Updated: 2025-08-15
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2
147 participants
INTERVENTIONAL
2016-07-05
2023-10-02
Brief Summary
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* Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
* Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent participants with Sickle Cell Disease ages 12 to 17 years.
* Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of voxelotor in pediatric participants with Sickle Cell Disease ages 4 to 17 years.
* Part D is a multiple dose, safety, tolerability, and PK study, which examines the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease ages 6 months to \< 4 years.
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Detailed Description
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Conditions
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Voxelotor
Subjects to receive daily oral dosing of voxelotor according to which Part (A, B, C, or D), the subject is participating in:
* Part A: Subjects to receive daily oral dosing of voxelotor for 1 day (single dose)
* Part B: Subjects to receive daily oral dosing of voxelotor for up to 24 weeks (multiple dose)
* Part C: Subjects to receive daily oral dosing of voxelotor for up to 48 weeks (1500mg or 1500mg equivalent dose)
* Part D: Subjects to receive daily oral dosing of voxelotor for up to 48 weeks (1500mg equivalent dose)
Voxelotor
* Part A: Voxelotor will be administered as oral capsules or tablets
* Part B: Voxelotor will be administered as oral capsules or tablets
* Part C: Voxelotor will be administered as oral dispersible tablets or powder for oral suspension
* Part D: Voxelotor will be administered as oral dispersible tablets or powder for oral suspension
Interventions
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Voxelotor
* Part A: Voxelotor will be administered as oral capsules or tablets
* Part B: Voxelotor will be administered as oral capsules or tablets
* Part C: Voxelotor will be administered as oral dispersible tablets or powder for oral suspension
* Part D: Voxelotor will be administered as oral dispersible tablets or powder for oral suspension
Eligibility Criteria
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Inclusion Criteria
* Age:
* Part A - 6 to 17 years of age
* Part B - 12 to 17 years of age
* Part C - 4 to 17 years of age
* Part D - 6 months to \<4 years of age
* Hydroxyurea (HU) therapy:
* Parts A, B, and C: A participant taking hydroxyurea (HU) may be enrolled if the dose has been stable for at least 3 months with no anticipated need for dose adjustment during the study and no sign of hematological toxicity.
* Part D: A participant taking HU may be enrolled if the dose has been stable for at least 1 month. Titration to the maximum tolerated dose (MTD) is allowed during the study.
* Hemoglobin (HB):
* Part A - No restriction
* Parts B, C, \& D - Hb ≤ 10.5 g/dL
* For Part C only: Participants 12 to 17 years of age must have a TCD velocity of ≥ 140 cm/sec measured anytime during screening.
Exclusion Criteria
* Vaso-occlusive crisis (VOC)
* Acute chest syndrome (ACS)
* Splenic sequestration crisis
* Dactylitis
* Requires chronic transfusion therapy
* History of stroke or meeting criteria for primary stroke prophylaxis (history of two TCD measurements ≥ 200 cm/sec by non-imaging TCD or ≥185 cm/sec by TCDi).
* Transfusion within 30 days prior to signing the ICF
* Body weight \<5 kg for 1 month prior to the screening visit and at the screening visit.
6 Months
17 Years
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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Brentwood Clinic UCSF Benioff Children's Hospital Oakland
Brentwood, California, United States
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
University of Illinois at Chicago Clinical Research Center
Chicago, Illinois, United States
Our Lady of the Lake Children's Hospital (IP Address)
Baton Rouge, Louisiana, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States
Brody School of Medicine at East Carolina University
Greenville, North Carolina, United States
University Hospitals Cleveland Medical Center, Rainbow Babies & Children's Hospital
Cleveland, Ohio, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
American University of Beirut - Medical Center
Beirut, , Lebanon
Rafik Hariri University Hospital
Beirut, , Lebanon
Nini Hospital
Tripoli, , Lebanon
University College London Hospital, NHS Foundation Trust
London, Greater London, United Kingdom
Barts Health NHS Trust, The Royal London Hospital
London, , United Kingdom
Guy's and St Thoma's NHS Foundation Trust, Evelina London Children's Hospital
London, , United Kingdom
Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital
Manchester, , United Kingdom
Countries
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References
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Estepp JH, Kalpatthi R, Woods G, Trompeter S, Liem RI, Sims K, Inati A, Inusa BPD, Campbell A, Piccone C, Abboud MR, Smith-Whitley K, Dixon S, Tonda M, Washington C, Griffin NM, Brown C. Safety and efficacy of voxelotor in pediatric patients with sickle cell disease aged 4 to 11 years. Pediatr Blood Cancer. 2022 Aug;69(8):e29716. doi: 10.1002/pbc.29716. Epub 2022 Apr 21.
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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C5341020
Identifier Type: OTHER
Identifier Source: secondary_id
GBT440-007
Identifier Type: -
Identifier Source: org_study_id
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