Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease

Stopped early · Phase 2

Conditions studied: Sickle Cell Disease

In brief

This study consists of four parts, Parts A, B, C, and D. * 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.

Key facts

Study ID

NCT02850406

Run by

Pfizer

People needed

147

Starts

2016-07-05

Expected to finish

2023-10-02

Last updated by the study team

2025-08-15

Who can join

Age: 1 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS β0thal)
• 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.

You may not qualify if…

• Any one of the following requiring medical attention within 14 days of signing the Informed Consent Form (ICF):
• 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
• Exclusion Criteria for Part D Only:
• Body weight <5 kg for 1 month prior to the screening visit and at the screening visit.

Where it is running

• 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

Full record on ClinicalTrials.gov

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