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Sotatercept in Treating Patients With Myeloproliferative Neoplasm-Associated Myelofibrosis or Anemia

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ClinicalTrials.gov Identifier: NCT01712308
Recruitment Status : Recruiting
First Posted : October 23, 2012
Last Update Posted : August 12, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:
This phase II trial studies the side effects of and how well sotatercept works in treating patients with myeloproliferative neoplasm-associated myelofibrosis or anemia. Sotatercept may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Condition or disease Intervention/treatment Phase
Anemia Myelodysplastic/Myeloproliferative Neoplasm Myelofibrosis Other: Laboratory Biomarker Analysis Biological: Sotatercept Phase 2

Detailed Description:

PRIMARY OBJECTIVES:

I. Determine safety and efficacy of sotatercept as therapy for persons with myeloproliferative neoplasm (MPN)-associated myelofibrosis and anemia.

OUTLINE: This is a dose-escalation study.

Patients receive sotatercept subcutaneously (SC) once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.

After completion of study treatment, patients are followed up at 1 month.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Intervention Model: Single Group Assignment
Masking: None (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
Actual Study Start Date : February 21, 2013
Estimated Primary Completion Date : February 28, 2020
Estimated Study Completion Date : February 28, 2021


Arm Intervention/treatment
Experimental: Treatment (sotatercept)
Patients receive sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Other: Laboratory Biomarker Analysis
Correlative studies

Biological: Sotatercept
Given SC
Other Names:
  • ACE-011
  • Decoy Activin Receptor ACE-011




Primary Outcome Measures :
  1. Incidence of toxicities [ Time Frame: Up to 28 days after the last dose of study drug ]
    Severity graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0. Toxicity is defined as an adverse event and classified as possibly, probably, or definitely related to study drug. Such adverse events will be recorded by the principal investigator in a database. The maximum grade for each type of toxicity will be recorded for each patient, including start/stop dates, and frequency tables for each group will be reviewed to determine toxicity patterns.

  2. Anemia-response [ Time Frame: Up to 84 days ]
    Defined as an increase in hemoglobin level equal to or greater than 1.5 g/L without red blood cell-transfusion OR becoming red blood cell-transfusion-independent in a patient who is red blood cell-transfusion-dependent. Will be based on the intent-to-treat principle.



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:

  • MPN-associated myelofibrosis
  • Anemic patient OR red blood cell (RBC)-transfusion-dependent patient
  • Alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) and aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) equal to or less than 2.5 x upper limit of normal (ULN), or equal to or less than 4 x ULN (if upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis [EMH] related to myelofibrosis [MF])
  • Direct bilirubin equal to or less than 1.5 x ULN; or equal to or less than 2 x ULN (if upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis related to MF)
  • Creatinine clearance equal to or more than 50 mL/min
  • Treatment-related toxicities from prior therapies must have resolved to grade equal to or less than 1
  • 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
  • For cohort of patients that are already on ruxolitinib therapy: on therapy with ruxolitinib for at least for 6 months, and on stable dose for last 2 months, before starting therapy with sotatercept

Exclusion Criteria:

  • 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
  • Pregnant or lactating female
  • Known positive for human immunodeficiency virus-1 (HIV-1), or active infection with hepatitis-B or -C
  • Use of any MPN-associated myelofibrosis-directed therapy within 2 weeks prior to study day 1 (other than ruxolitinib at a stable dose for patients in the combination cohort as stated in inclusion criteria)
  • Symptomatic congestive heart failure, unstable angina, or unstable cardiac arrhythmia
  • Prior sotatercept
  • Major surgery within 4 weeks prior to day 1
  • Severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product
  • Uncontrolled hypertension (systolic blood pressure [SBP] equal to or more than 140 or diastolic blood pressure [DBP] equal to or more than 90)

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): NCT01712308


Contacts
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Contact: Prithviraj Bose, MD 713-792-7305 pbose@mdanderson.org

Locations
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United States, Texas
M D Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Prithviraj Bose    713-792-7305      
Principal Investigator: Prithviraj Bose         
Sponsors and Collaborators
M.D. Anderson Cancer Center
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Prithviraj Bose M.D. Anderson Cancer Center

Additional Information:
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Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01712308     History of Changes
Other Study ID Numbers: 2012-0534
NCI-2012-03139 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2012-0534 ( Other Identifier: M D Anderson Cancer Center )
P30CA016672 ( U.S. NIH Grant/Contract )
First Posted: October 23, 2012    Key Record Dates
Last Update Posted: August 12, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia
Primary Myelofibrosis
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms
Hematologic Diseases
Bone Marrow Diseases