Study of Sotatercept (ACE-011) in Subjects With Myeloproliferative Neoplasm (MPN)-Associated Myelofibrosis (MF) and Anemia

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by M.D. Anderson Cancer Center
Sponsor:
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
NCT01712308
First received: October 18, 2012
Last updated: May 9, 2014
Last verified: May 2014
  Purpose

The goal of this clinical research study is to learn if sotatercept can help to control MPN-associated myelofibrosis and anemia. The safety of this drug will also be studied.


Condition Intervention Phase
Leukemia
Drug: Sotatercept
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: A Phase-2, Prospective, Open-Label Study to Determine the Safety and Efficacy of Sotatercept (ACE-011) in Subjects With Myeloproliferative Neoplasm (MPN) -Associated Myelofibrosis and Anemia

Resource links provided by NLM:


Further study details as provided by M.D. Anderson Cancer Center:

Primary Outcome Measures:
  • Anemia Response [ Time Frame: 15 weeks ] [ Designated as safety issue: Yes ]
    Primary analysis of response for each dose cohort performed once all 20 patients have been accrued to that dose cohort and treated for at least 5 cycles. Clinical efficacy assessed as anemia response with a target rate of greater than or equal to 30% at either dose level. Anemia response is a composite endpoint defined as an increase in hemoglobin in a subject with anemia or becoming RBC-transfusion-independent in a subject who is RBC-transfusion-dependent.


Secondary Outcome Measures:
  • Duration of Response [ Time Frame: 15 weeks ] [ Designated as safety issue: No ]
    Duration of response defined as date at which subject's objective status is first noted to be a response, to date progression is documented (if one has occurred) or to date of last follow-up (for those subjects who have not progressed).


Estimated Enrollment: 50
Study Start Date: February 2013
Estimated Study Completion Date: February 2017
Estimated Primary Completion Date: February 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Sotatercept (higer dose)
Patients treated with 0.5 mg/kg dose subcutaneously on Day 1 every 3 weeks.
Drug: Sotatercept

Higher dose group: 0.5 mg/kg dose subcutaneously on Day 1 every 3 weeks.

Lower dose group: 0.3 mg/kg dose subcutaneously on Day 1 every 3 weeks.

Other Name: ACE-011
Experimental: Sotatercept (lower dose)
Patients treated with 0.3 mg/kg dose subcutaneously on Day 1 every 3 weeks.
Drug: Sotatercept

Higher dose group: 0.5 mg/kg dose subcutaneously on Day 1 every 3 weeks.

Lower dose group: 0.3 mg/kg dose subcutaneously on Day 1 every 3 weeks.

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:

  1. MPN-associated myelofibrosis
  2. Anemic patient OR RBC-transfusion-dependent patient
  3. Equal to or greater than 18 years of age
  4. ALT (SGPT) and AST (SGOT) equal to or less than 2.5x upper limit of normal (ULN), or equal to or less than 4x ULN (if upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis [EMH] related to MF)
  5. Direct bilirubin equal to or less than 1.5 x ULN; or equal to or less than 2x ULN (if upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis related to MF)
  6. Creatinine clearance equal to or more than 50 mL/min
  7. Treatment-related toxicities from prior therapies must have resolved to Grade equal to or less than 1
  8. Women of childbearing potential and men must agree to using medically approved (i.e., mechanical or pharmacological) contraceptive measure for at least 112 days following the last dose of sotatercept (ACE-011), Males must agree to use a latex condom or non-latex condom NOT made of natural (animal) membrane during any sexual contact with females of childbearing potential or a pregnant female while participating in the study and for at least 112 days following the last dose of sotatercept (ACE-011), even if he has a vasectomy.

Exclusion Criteria:

  1. Serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
  2. Pregnant or lactating female
  3. Known positive for human immunodeficiency virus-1 (HIV-1), or active infection with hepatitis-B or -C
  4. Use of any MPN-associated myelofibrosis-directed therapy within 2 weeks prior to study Day 1
  5. Symptomatic congestive heart failure, unstable angina, or unstable cardiac arrhythmia
  6. Prior sotatercept
  7. Major surgery within 4 weeks prior to Day 1
  8. Severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product
  9. Any patient who is being treated for hypertension
  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 ClinicalTrials.gov identifier: NCT01712308

Contacts
Contact: Srdan Verstovsek, MD 713-792-7305

Locations
United States, Texas
UT MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Investigators
Principal Investigator: Srdan Verstovsek, MD UT MD Anderson Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01712308     History of Changes
Other Study ID Numbers: 2012-0534, NCI-2012-03139
Study First Received: October 18, 2012
Last Updated: May 9, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by M.D. Anderson Cancer Center:
Leukemia
Myeloproliferative Neoplasm
MPN
Associated Myelofibrosis
Anemia
Sotatercept
ACE-011

Additional relevant MeSH terms:
Primary Myelofibrosis
Anemia
Neoplasms
Leukemia
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Neoplasms by Histologic Type

ClinicalTrials.gov processed this record on August 25, 2014