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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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16 March 2021 |
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Main ID: |
NCT01464164 |
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Date of registration:
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31/10/2011 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
ACE-011-DBA |
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Scientific title:
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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 |
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Date of first enrolment:
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January 2012 |
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Target sample size:
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20 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT01464164 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Eva Atsidaftos, MA, CCRC |
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Address:
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Telephone:
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516-562-1504 |
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Email:
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eatsidaf@northwell.edu |
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Affiliation:
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Name:
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Eva Atsidaftos, MA |
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Address:
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Telephone:
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516-562-1504 |
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Email:
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eatsidaf@northwell.edu |
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Affiliation:
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Name:
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Adrianna Vlachos, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Northwell Health/Feinstein Institute for Medical Research |
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Key inclusion & exclusion criteria
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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
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Diamond Blackfan Anemia
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Intervention(s)
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Drug: Sotatercept
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Drug: Sotatercept with prednisone boost
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Primary Outcome(s)
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Determine a safe and effective dose of sotatercept in adults with DBA and RBC transfusion dependence
[Time Frame: 9 months]
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Secondary Outcome(s)
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Time to response
[Time Frame: 9 months]
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Exploratory outcome improvement of bone density
[Time Frame: 9 months]
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Response duration
[Time Frame: 9 months]
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Safety profile of ACE-011 in DBA patients
[Time Frame: 9 months]
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Secondary ID(s)
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11-02-199 - 06A
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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