Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan An… (NCT01464164) | Clinical Trial Compass
TerminatedPhase 1/2
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
Stopped: Supporter terminated the study due to no active patients (secondary to travel restrictions due to COVID).
United States19 participantsStarted 2012-01
Plain-language 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.
Who can participate
Age range18 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
What they're measuring
1
Number of Participants With Complete Response and Partial Response