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.
Recruiting
Phase 2
| Drug | Synonyms | Arms |
|---|---|---|
| Sotatercept | ACE-011, Decoy Activin Receptor ACE-011 | Treatment (sotatercept) |
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.
| Name | Type | Description | Interventions |
|---|---|---|---|
| Treatment (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. |
|
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)
| Maximum Eligible Age: | N/A |
| Minimum Eligible Age: | 18 Years |
| Eligible Gender: | All |
| Healthy Volunteers: | No |
| Measure: | Incidence of toxicities |
| Time Frame: | Up to 28 days after the last dose of study drug |
| Safety Issue: | |
| Description: | 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. |
| Phase: | Phase 2 |
| Primary Purpose: | Interventional |
| Overall Status: | Recruiting |
| Lead Sponsor: | M.D. Anderson Cancer Center |
August 5, 2020
