Summary

Eligibility
for people ages 6 months to 17 years (full criteria)
Location
at Brentwood, California and other locations
Dates
study started
completion around

Description

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.

Official Title

A Phase 2a, Open-label, Single and Multiple Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Treatment Effect of GBT440 in Pediatric Participants With Sickle Cell Disease

Keywords

Sickle Cell Disease, Sickle Cell Anemia, Voxelotor

Eligibility

You can join if…

Open to people ages 6 months to 17 years

  • 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 CAN'T join 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.

Locations

  • Brentwood Clinic UCSF Benioff Children's Hospital Oakland in progress, not accepting new patients
    Brentwood California 94513 United States
  • UCSF Benioff Children's Hospital Oakland in progress, not accepting new patients
    Oakland California 94609 United States
  • UCSF Benioff Children's Hospital Walnut Creek in progress, not accepting new patients
    Walnut Creek California 94598 United States
  • St. Jude Children's Research Hospital - Pharmaceutical Services accepting new patients
    Memphis Tennessee 38105 United States
  • St. Jude Children's Research Hospital in progress, not accepting new patients
    Memphis Tennessee 38105 United States
  • Brody School of Medicine at East Carolina University accepting new patients
    Greenville North Carolina 27834 United States
  • ECU Physicians accepting new patients
    Greenville North Carolina 27834 United States

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
Links
To obtain contact information for a study center near you, click here.
ID
NCT02850406
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 148 study participants
Last Updated