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

  • STATUS
    Recruiting
  • End date
    Dec 24, 2022
  • participants needed
    155
  • sponsor
    Global Blood Therapeutics
Updated on 24 May 2021
Investigator
Margaret Tonda, PharmD
Primary Contact
East Carolina University Brody School of Medicine (2.7 mi away) Contact
+27 other location
hydroxyurea
hemoglobin s
thalassemia
gbt440
sickle cell trait
sickle hemoglobin
voxelotor

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.

Details
Condition SICKLE CELL ANEMIA, Sickle Cell Disease
Treatment GBT440, Voxelotor
Clinical Study IdentifierNCT02850406
SponsorGlobal Blood Therapeutics
Last Modified on24 May 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS 0thal)
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 - 9 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

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
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