Diamond Blackfan Anemia Clinical Trial
— ACE-011-DBAOfficial title:
Phase I/II, Open-Label Study to Determine Safety and Efficacy of Sotatercept (ACE-011) in Adults With Red Blood Cell Transfusion- Dependent Diamond Blackfan Anemia
Verified date | February 2023 |
Source | Northwell Health |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Terminated |
Enrollment | 19 |
Est. completion date | April 13, 2021 |
Est. primary completion date | April 13, 2021 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | North Shore- LIJ campus of The Feinstein Institute for Medical Research | Manhasset | New York |
Lead Sponsor | Collaborator |
---|---|
Northwell Health |
United States,
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* Note: There are 30 references in all — Click here to view all references
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Participants With Complete Response and Partial Response | Complete response is transfusion independence with hemoglobin >9 gm/dl; partial response is transfusion dependence with hemoglobin < 9gm/dl with an increase in reticulocyte count over baseline | 9 months | |
Secondary | Severe Adverse Events Attributable to Study Drug | Assess severity of adverse events and relationship to sotatercept according to the currently active minor version of the NCI Common Terminology for Adverse Events version 4.0 | 9 months |
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