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

This study is currently recruiting participants.
Verified March 2014 by North Shore Long Island Jewish Health System
Sponsor:
Information provided by (Responsible Party):
North Shore Long Island Jewish Health System
ClinicalTrials.gov Identifier:
NCT01464164
First received: October 31, 2011
Last updated: March 27, 2014
Last verified: March 2014
  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 2014
Estimated Primary Completion Date: April 2014 (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: March 27, 2014
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 April 17, 2014