Last updated on February 2020

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

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: SICKLE CELL ANEMIA | Sickle Cell Disease
  • Age: Between 1 - 17 Years
  • Gender: Male or Female

Inclusion Criteria:

  1. Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS 0thal)
  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:
    • 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.
  8. Hemoglobin (HB):
  9. Part A - No restriction
  10. Parts B, C, & D - Hb 10.5 g/dL
  11. 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:

  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
  2. Requires chronic transfusion therapy
  3. 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).
  4. Transfusion within 30 days prior to signing the ICF

Exclusion Criteria for Part D Only:

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

Recruitment Status: Open

Brief Description Eligibility Contact Research Team

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