Trial record 12 of 509 for:
Open Studies | "Anemia"
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia (ACE-011-DBA)
This study is currently recruiting participants.
Verified October 2012 by North Shore Long Island Jewish Health System
Sponsor:
North Shore Long Island Jewish Health System
Information provided by (Responsible Party):
North Shore Long Island Jewish Health System
ClinicalTrials.gov Identifier:
NCT01464164
First received: October 31, 2011
Last updated: October 8, 2012
Last verified: October 2012
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Diamond Blackfan Anemia |
Drug: Sotatercept |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
| Official 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 |
Resource links provided by NLM:
Further study details as provided by North Shore Long Island Jewish Health System:
Primary Outcome Measures:
- Determine a safe and effective dose of sotatercept in adults with DBA and RBC transfusion dependence [ Time Frame: 9 months ] [ Designated as safety issue: No ]Achieve a complete response transfusion- independent and Hemoglobin >9 gm/dl or a partial response- increase in transfusion interval from baseline and hemoglobin < 9gm/dl iith an increase in reticulocyte count
Secondary Outcome Measures:
- Safety [ Time Frame: 9 months ] [ Designated as safety issue: Yes ]Assess type, frequency, and 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
- Time to response [ Time Frame: 9 months ] [ Designated as safety issue: No ]Length of time required to achieve a response
- Response duration [ Time Frame: 9 months ] [ Designated as safety issue: No ]Length of time between transfusions
- Exploratory outcome improvement of bone density [ Time Frame: 9 months ] [ Designated as safety issue: No ]Improvement as measured by bone densitometry (DEXA scan)
| Estimated Enrollment: | 20 |
| Study Start Date: | January 2012 |
| Estimated Study Completion Date: | December 2013 |
| Estimated Primary Completion Date: | February 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Sotatercept
Sotatercept to be given as a subcutaneous injection once a month for 4 consecutive months, using a dose escalation scale among 3 cohorts.
|
Drug: Sotatercept
Dose escalation study of the drug sotatercept to be given as a subcutaneous injection: Cohorts of 3 subjects will receive sotatercept every 28 days for up to 4 doses. Starting dose of 0.1 mg/kg (dose level 1). The second and subsequent cohorts may begin after and only after all 3 subjects in the first cohort have completed 28 days following their 4th dose of sotatercept (or last dose if sotatercept is not discontinued for safety reasons) and safety has been determined to proceed. Subsequent dose levels are 0.3 mg/kg (dose level 2) and 0.5 mg/kg (dose level 3). Other Name: ACE-011
|
Show Detailed Description Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01464164
Contacts
| Contact: Ellen Muir, MSN, RN, CNS | 516-562-1505 | emuir@nshs.edu |
| Contact: Eva Atsidaftos, MA | 516-562-1504 | eatsidaf@nshs.edu |
Locations
| United States, New York | |
| North Shore- LIJ campus of The Feinstein Institute for Medical Research | Recruiting |
| Manhasset, New York, United States, 11030 | |
| Contact: Ellen Muir, MSN, RN, CNS 516-562-1505 emuir@nshs.edu | |
| Contact: Eva Atsidaftos, MA 516-562-1504 eatsidaf@nshs.edu | |
| Principal Investigator: Adrianna Vlachos, MD | |
| Principal Investigator: Johnson Liu, MD | |
| Sub-Investigator: Jeffrey M Lipton, MD, PhD | |
Sponsors and Collaborators
North Shore Long Island Jewish Health System
Investigators
| Principal Investigator: | Adrianna Vlachos, MD | North Shore- Long Island Jewish Medical Center; Cohen Children's Medical Center of NY |
| Principal Investigator: | Johnson Liu, MD | North Shore- Long Island Jewish Medical Center |
More Information
Publications:
| Responsible Party: | North Shore Long Island Jewish Health System |
| ClinicalTrials.gov Identifier: | NCT01464164 History of Changes |
| Other Study ID Numbers: | 11-02-199 - 06A |
| Study First Received: | October 31, 2011 |
| Last Updated: | October 8, 2012 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by North Shore Long Island Jewish Health System:
|
Diamond Blackfan anemia Blackfan Diamond anemia Anemia, Congenital Pure Red Cell Congenital Hypoplastic Anemia Hypoplastic Congenital Anemia DBA Congenital pure red cell aplasia Aase-Smith II Syndrome |
Aase Syndrome Aplasia, Congenital Pure Red Cell BDA Erythrogenesis Imperfecta Inherited Erythroblastopenia pure red cell aplasia bone marrow failure syndrome |
Additional relevant MeSH terms:
|
Anemia Anemia, Diamond-Blackfan Hematologic Diseases Anemia, Hypoplastic, Congenital |
Anemia, Aplastic Red-Cell Aplasia, Pure Bone Marrow Diseases Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on May 19, 2013