Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia

PHASE1/PHASE2TerminatedINTERVENTIONAL
Enrollment

19

Participants

Timeline

Start Date

January 31, 2012

Primary Completion Date

April 13, 2021

Study Completion Date

April 13, 2021

Conditions
Diamond Blackfan Anemia
Interventions
DRUG

Sotatercept

"Cohort 4a: 3 patients on Sotatercept 0.75 mg/kg every 3 weeks. If the patients have no untoward events in the Patients in Dose Level 4 by the end of 3 of the 6 doses, then additional patients (Dose Level 5) will be enrolled at the next dose level:~Cohort 5a: 3 patients on Sotatercept 1 mg/kg every 3 weeks"

DRUG

Sotatercept with prednisone boost

"Cohort 4b: 3 patients on Sotatercept 0.75 mg/kg every 3 weeks with a three week prednisone boost of 1 mg/kg/day (max 60 mg). If the patients have no untoward events in the Patients in Dose Level 4 by the end of 3 of the 6 doses, then additional patients (Dose Level 5) will be enrolled at the next dose level:~Cohort 5b: 3 patients on Sotatercept 1 mg/kg every 3 weeks with a 3 week prednisone boost of 1 mg/kg/day (max 60 mg)."

Trial Locations (1)

11030

North Shore- LIJ campus of The Feinstein Institute for Medical Research, Manhasset

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Northwell Health

OTHER

NCT01464164 - Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia | Biotech Hunter | Biotech Hunter