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

NCT02850406 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 147

Last updated 2025-08-15

Study results available
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Summary

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.

Conditions

Interventions

DRUG

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

Sponsors & Collaborators

Principal Investigators

  • Pfizer CT.gov Call Center · Pfizer

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
6 Months
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-07-05
Primary Completion
2023-10-02
Completion
2023-10-02
FDA Drug
Yes

Countries

  • United States
  • Lebanon
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02850406 on ClinicalTrials.gov