Status:
TERMINATED
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
Lead Sponsor:
Northwell Health
Conditions:
Diamond Blackfan Anemia
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief 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...
Detailed Description
This study is for adult patients with Diamond Blackfan Anemia who are currently being transfused every 3- 4 weeks. The drug will be given as a subcutaneous injection within one of three dose levels. ...
Eligibility Criteria
Inclusion
- \>/= 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
- 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
Key Trial Info
Start Date :
January 1 2012
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 13 2021
Estimated Enrollment :
19 Patients enrolled
Trial Details
Trial ID
NCT01464164
Start Date
January 1 2012
End Date
April 13 2021
Last Update
March 17 2023
Active Locations (1)
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1
North Shore- LIJ campus of The Feinstein Institute for Medical Research
Manhasset, New York, United States, 11030