Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia (ACE-011-DBA)
Diamond Blackfan Anemia
About this trial
This is an interventional treatment trial for Diamond Blackfan Anemia focused on measuring Diamond Blackfan anemia, DBA, Blackfan Diamond anemia, Congenital pure red cell aplasia, Aase-Smith II Syndrome, Aase Syndrome, Anemia, Congenital Pure Red Cell, Aplasia, Congenital Pure Red Cell, BDA, Congenital Hypoplastic Anemia, Erythrogenesis Imperfecta, Hypoplastic Congenital Anemia, Inherited Erythroblastopenia, pure red cell aplasia, bone marrow failure syndrome
Eligibility Criteria
Inclusion Criteria:
- >/= 18 years of age
- DBA diagnosed
- RBC transfusion- dependence (defined as >/= 10 cc/kg of RBC per 28 days average)
- Karnofsky performance scale >/= 70
- Females of childbearing potential are to use birth control during study participation and for 112 days following the last dose of sotatercept
- Males must agree to use a latex condom during any sexual contact with females of childbearing potential while participating in the study and for 112 days following the last dose of sotatercept
- Agreement to adhere to the study visit schedule, understand and comply with all protocol requirements
- Understand and sign a written informed consent
Exclusion Criteria:
- Creatinine clearance < 30 ml/min
- SGOT > 3x upper limit of normal, SGPT > 3x upper limit normal, or bilirubin >3x upper limit normal
- Heart disease (NY Heart Association classification of >/= 3
- History of hypertension
- Subjects currently responsive to corticosteroids for treatment of Diamond Blackfan anemia
- Treatment with another investigational drug or device <56 days pre-study entry
- Pregnant or lactating females
- Cancer
Sites / Locations
- North Shore- LIJ campus of The Feinstein Institute for Medical Research
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Sotatercept
Sotatercept with prednisone boost
Sotatercept to be given as a subcutaneous injection once a month for 4 consecutive months, using a dose escalation scale among 3 cohorts. *Protocol Amendment: Two additional cohorts will be given Sotatercept 0.75mg/kg and 1 mg/kg as a subcutaneous injection once every 3 weeks.
Protocol Amendment: Two additional cohorts will be given Sotatercept 0.75mg/kg and 1 mg/kg as a subcutaneous injection once every 3 weeks along with a prednisone boost of 1 mg/kg daily for 3 weeks (max of 60 mg).