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

NCT01712308 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: 2012-0534NCI-2012-03139
• Study Type: interventional
• Target: 56
• Locations: 1 country
• Sites: 1

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.

Conditions

Anemia; Myelodysplastic/Myeloproliferative Neoplasm; Myelofibrosis

Outcome Measures

Primary Outcomes (2)

• Anemia-response

  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.

  Up to 84 days

• Duration of Response

  Response date to loss of response or last follow up.

  Up to 9 years

Study Arms (2)

Treatment Monotherapy (sotatercept)

EXPERIMENTAL

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.

Biological: Sotatercept

Treatment combination (Ruxolitinib + sotatercept)

EXPERIMENTAL

patients that are already on therapy with ruxolitinib (for at least for 6 months, and on stable dose for last 2 months) will continue ruxolitinib in addition to 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.

Biological: Sotatercept; Drug: Ruxolitinib

Interventions

Sotatercept BIOLOGICAL

Given SC

Also known as: ACE-011, Decoy Activin Receptor ACE-011

Treatment Monotherapy (sotatercept); Treatment combination (Ruxolitinib + sotatercept)

Ruxolitinib DRUG

Also known as: INCB018424

Treatment combination (Ruxolitinib + sotatercept)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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)

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• University of Texas MD Anderson Cancer Center Website

MeSH Terms

Conditions

Anemia; Myelodysplastic-Myeloproliferative Diseases; Primary Myelofibrosis

Interventions

ACE-011; ruxolitinib

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Myeloproliferative Disorders

Results Point of Contact

• Title: Prithviraj Bose MD/Associate Professor
• Organization: The University of Texas MD Anderson Cancer

PBose@mdanderson.org

713-792-7747

Study Officials

• Prithviraj Bose

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 18, 2012
• First Posted: October 23, 2012
• Study Start: February 21, 2013
• Primary Completion: May 24, 2022
• Study Completion: May 24, 2022
• Last Updated: October 10, 2023
• Results First Posted: June 6, 2023

Record last verified: 2023-10

Locations

US (1)