Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Pfizer
Age
0–17
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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 may not be 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
Body weight \<5 kg for 1 month prior to the screening visit and at the screening visit.
Clareo Health | Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
