Shots:

• The P-II portion evaluates the safety, tolerability, efficacy, PK & PD of GBT601 (100/150/200mg) in the ratio of 1:1:1 in patients (n=60) aged 18-65yrs. with SCD, planned to include sites in Africa, the EU, the Middle East, South America & the US
• The 1EP of the study will measure a change from baseline in Hb @12wks & the 2EP will evaluate the PK, PD along with the relationship b/w GBT601 & measures of anemia/hemolysis. The results from the P-I & P-II study are expected by the YE'22
• The P-III study will evaluate the safety & efficacy of the selected optimal dose of GBT601 vs PBO in patients for 48wks. The 3rd arm of the study will evaluate the PK & safety of single/multiple doses of GBT601 in pediatric patients

Ref: GBT | Image: GBT