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

• Status: Recruiting
• Trial end date: 2022-05-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to determine the safety and dosing of drug Sotatercept, as a subcutaneous injection, to stimulate production of red blood cell production. To be given every 28 days for up to four doses.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Northwell Health
• Treatments: Prednisone

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