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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Adrianna Vlachos, North Shore Long Island Jewish Health System Identifier:
First received: October 31, 2011
Last updated: August 31, 2015
Last verified: August 2015
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 Northwell Health:

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 2016
Estimated Primary Completion Date: April 2016 (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

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Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01464164

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
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


Responsible Party: Adrianna Vlachos, Principal Investigator, North Shore Long Island Jewish Health System Identifier: NCT01464164     History of Changes
Other Study ID Numbers: 11-02-199 - 06A 
Study First Received: October 31, 2011
Last Updated: August 31, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Northwell Health:
Diamond Blackfan anemia
Blackfan Diamond anemia
Congenital pure red cell aplasia
Aase-Smith II Syndrome
Aase Syndrome
Anemia, Congenital Pure Red Cell
Aplasia, Congenital Pure Red Cell
Congenital Hypoplastic Anemia
Erythrogenesis Imperfecta
Hypoplastic Congenital Anemia
Inherited Erythroblastopenia
pure red cell aplasia
bone marrow failure syndrome

Additional relevant MeSH terms:
Anemia, Diamond-Blackfan
Anemia, Aplastic
Anemia, Hypoplastic, Congenital
Bone Marrow Diseases
Genetic Diseases, Inborn
Hematologic Diseases
Red-Cell Aplasia, Pure processed this record on May 22, 2016