for people ages 12–17 (full criteria)
at Oakland, California and other locations
study started
estimated completion:



This study consists of two parts, Part A and Part B. Part A is a single dose 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.

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


Sickle Cell Disease Anemia, Sickle Cell


You can join if…

Open to people ages 12–17

  1. Male or female age 12 to 17 years of age (inclusive) with homozygous hemoglobin SS(HbSS) or hemoglobin S beta0 thalassemia (HbS β0 thal).
  2. A subject taking hydroxyurea may be enrolled if the dose has been stable for at least 3 months with no anticipated need for dose adjustments during the study and no sign of hematological toxicity.
  3. Written informed parental/ guardian consent and subject assent has been obtained per institutional review board (IRB)/Ethics Committee (EC) policy and requirements,consistent with International Council on Harmonisation (ICH) guidelines.

You CAN'T join if...

  1. Any one of the following requiring medical attention within 14 days prior to signing the informed consent form (ICF):
  2. Vaso-occlusive crisis (VOC)
  3. Acute chest syndrome (ACS)
  4. Stroke
  5. Splenic sequestration crisis
  6. Dactylitis
  7. Requires chronic transfusion therapy
  8. History of stroke or meeting criteria for primary stroke prophylaxis (history of two Transcranial Doppler (TCD) measurements ≥ 200 cm/sec)
  9. Transfusion within 30 days prior to signing the Informed Consent Form.
  10. Renal dysfunction requiring chronic dialysis or creatinine ≥ 1.5 milligrams per deciliter (mg/dL)
  11. History of hepatic compromise or severe liver disease
  12. Clinically relevant cardiac abnormality


  • Oakland, California, 94609, United States
  • Kansas City, Missouri, 64108, United States
  • Cleveland, Ohio, 38105, United States
  • Memphis, Tennessee, 38105, United States
  • Chicago, Illinois, 60612, United States
  • Chicago, Illinois, 60611, United States
  • Cincinnati, Ohio, 45229, United States
  • Atlanta, Georgia, 30342, United States
  • New Brunswick, New Jersey, 08901, United States


accepting new patients
Start Date
Completion Date
Global Blood Therapeutics
Phase 2
Study Type
Last Updated
April 2017