Summary

Eligibility
for people ages 9 months to 17 years (full criteria)
Location
at Oakland, California and other locations
Dates
study started
estimated completion
Principal Investigator
Lynne Neumayr, MD
Photo of Lynne Neumayr
Lynne Neumayr

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. Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent Sickle Cell Disease participants who were 12-17 years of age. 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 who are 4 to 17 years of age. Part D is a multiple dose, safety, tolerability, and PK study, which will examine the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease who are between 9 months to < 4 years of age.

Official Title

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

Keywords

Sickle Cell Disease Anemia, Sickle Cell Voxelotor

Eligibility

You can join if…

Open to people ages 9 months to 17 years

  1. Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS β0thal)
  2. Age:
  3. Part A - 6 to 17 years of age
  4. Part B - 12 to 17 years of age
  5. Part C - 4 to 17 years of age
  6. Part D - 9 months to <4 years of age
  7. Hydroxyurea (HU) therapy:
  8. 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.
  9. 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.
  10. Hemoglobin (HB):
  11. Part A - No restriction
  12. Parts B, C, & D - Hb ≤ 10.5 g/dL
  13. . 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...

  1. 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:

  1. . Body weight <5 kg for 1 month prior to the screening visit and at the screening visit.

Locations

  • UCSF Benioff Children's Hospital Oakland accepting new patients
    Oakland California 94609 United States
  • Children's Mercy Hospital Kansas City accepting new patients
    Kansas City Missouri 64108 United States

Lead Scientist at UCSF

  • Lynne Neumayr, MD
    Professor, Pediatrics. Authored (or co-authored) 62 research publications.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Global Blood Therapeutics
ID
NCT02850406
Phase
Phase 2
Study Type
Interventional
Last Updated