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Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia (ACE-011-DBA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01464164
Recruitment Status : Terminated (Supporter terminated the study due to no active patients (secondary to travel restrictions due to COVID).)
First Posted : November 3, 2011
Results First Posted : December 21, 2021
Last Update Posted : December 21, 2021
Sponsor:
Information provided by (Responsible Party):
Adrianna Vlachos, Northwell Health

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 to four doses.

Condition or disease Intervention/treatment Phase
Diamond Blackfan Anemia Drug: Sotatercept Drug: Sotatercept with prednisone boost Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (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
Actual Study Start Date : January 2012
Actual Primary Completion Date : April 13, 2021
Actual Study Completion Date : April 13, 2021


Arm Intervention/treatment
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. *Protocol Amendment: Two additional cohorts will be given Sotatercept 0.75mg/kg and 1 mg/kg as a subcutaneous injection once every 3 weeks.
Drug: Sotatercept

Cohort 4a: 3 patients on Sotatercept 0.75 mg/kg every 3 weeks. If the patients have no untoward events in the Patients in Dose Level 4 by the end of 3 of the 6 doses, then additional patients (Dose Level 5) will be enrolled at the next dose level:

Cohort 5a: 3 patients on Sotatercept 1 mg/kg every 3 weeks

Other Name: ACE-011

Experimental: Sotatercept with prednisone boost
Protocol Amendment: Two additional cohorts will be given Sotatercept 0.75mg/kg and 1 mg/kg as a subcutaneous injection once every 3 weeks along with a prednisone boost of 1 mg/kg daily for 3 weeks (max of 60 mg).
Drug: Sotatercept with prednisone boost

Cohort 4b: 3 patients on Sotatercept 0.75 mg/kg every 3 weeks with a three week prednisone boost of 1 mg/kg/day (max 60 mg). If the patients have no untoward events in the Patients in Dose Level 4 by the end of 3 of the 6 doses, then additional patients (Dose Level 5) will be enrolled at the next dose level:

Cohort 5b: 3 patients on Sotatercept 1 mg/kg every 3 weeks with a 3 week prednisone boost of 1 mg/kg/day (max 60 mg).

Other Name: ACE-011 and Prednisone




Primary Outcome Measures :
  1. Number of Participants With Complete Response and Partial Response [ Time Frame: 9 months ]
    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


Secondary Outcome Measures :
  1. Severe Adverse Events Attributable to Study Drug [ Time Frame: 9 months ]
    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



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01464164


Locations
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United States, New York
North Shore- LIJ campus of The Feinstein Institute for Medical Research
Manhasset, New York, United States, 11030
Sponsors and Collaborators
Northwell Health
Investigators
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Principal Investigator: Adrianna Vlachos, MD Northwell Health/Feinstein Institute for Medical Research
  Study Documents (Full-Text)

Documents provided by Adrianna Vlachos, Northwell Health:
Publications:

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Responsible Party: Adrianna Vlachos, Principal Investigator, Northwell Health
ClinicalTrials.gov Identifier: NCT01464164    
Other Study ID Numbers: 11-02-199 - 06A
First Posted: November 3, 2011    Key Record Dates
Results First Posted: December 21, 2021
Last Update Posted: December 21, 2021
Last Verified: November 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Keywords provided by Adrianna Vlachos, Northwell Health:
Diamond Blackfan anemia
DBA
Blackfan Diamond anemia
Congenital pure red cell aplasia
Aase-Smith II Syndrome
Aase Syndrome
Anemia, Congenital Pure Red Cell
Aplasia, Congenital Pure Red Cell
BDA
Congenital Hypoplastic Anemia
Erythrogenesis Imperfecta
Hypoplastic Congenital Anemia
Inherited Erythroblastopenia
pure red cell aplasia
bone marrow failure syndrome
Additional relevant MeSH terms:
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Anemia
Anemia, Diamond-Blackfan
Hematologic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Red-Cell Aplasia, Pure
Congenital Bone Marrow Failure Syndromes
Bone Marrow Failure Disorders
Bone Marrow Diseases
Genetic Diseases, Inborn
Prednisone
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents