Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||18 Years and Over|
Inclusion Criteria:Subjects must satisfy the following criteria to be randomized in the study: 1. Subject is ≥18 years of age at the time of signing the ICF. 2. Subject has a diagnosis of PMF according to the 2016 World Health Organization (WHO) criteria or diagnosis of post-ET or post-PV MF according to the IWG-MRT 2007 criteria , confirmed by the most recent local pathology report. 3. Subject is requiring RBC transfusions as defined as: a. Average RBC-transfusion frequency: 4 to 12 RBC units/12 weeks immediately up to randomization. There must be no interval > 6 weeks (42 days) without ≥ 1 RBC transfusion. b. RBC transfusions are scored in determining eligibility when given for treatment of:
- - Symptomatic (ie, fatigue or shortness of breath) anemia with a pretransfusion Hgb ≤ 9.5 g/dL or.
- - Asymptomatic anemia with a pretransfusion Hgb ≤ 7 g/dL c.
Exclusion Criteria:The presence of any of the following will exclude a subject from randomization: 1. Subject with anemia from cause other than MPN-associated MForJAK2 inhibitor therapy (eg, iron deficiency, vitamin B12 and/or folate deficiencies, autoimmune or hemolytic anemia, infection, or any type of known clinically significant bleeding or sequestration). 2. Subject use of hydroxyurea, immunomodulatory compounds such as pomalidomide, thalidomide, ESAs, androgenic steroids or other drugs with potential effects on hematopoiesis ≤ 8 weeks immediately up to the date of randomization. 1. Systemic corticosteroids are permitted for nonhematological conditions providing the subject is receiving a constant dose equivalent to ≤ 10 mg prednisone for the 4 weeks immediately up to randomization. 2. Iron chelation therapy (ICT) is permitted providing the subject is receiving a stable dose for the 8 weeks immediately up to randomization. 3. Subject with any of the following laboratory abnormalities at screening: 1. Neutrophils: < 1 x 109/L. 2. White blood count (WBC): > 100 x 109/L. 3. Platelets: the lowest allowable level as approved for the concomitant JAK2 inhibitor but not < 25 x 109/L or > 1000 x 109/L. 4. Peripheral blood myeloblasts:> 5% 5. Estimated glomerular filtration rate:< 40 mL/min/1.73 m2 (via the 4-variable modification of diet in renal disease [MDRD] formula) or nephrotic subjects (eg, urine albumin-to-creatinine ratio > 3500 mg/g) 6. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT):> 3.0 x upper limit of normal (ULN) 7. Direct bilirubin: ≥ 2 x ULN.
- - Higher levels are acceptable if these can be attributed to active red blood cell precursor destruction within the bone marrow (eg, ineffective erythropoiesis) 4.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Principal Investigator Affiliation||Bristol-Myers Squibb|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
|Countries||Australia, Austria, Belgium, Canada, China, Czechia, France, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Japan, Korea, Republic of, Poland, Russian Federation, Spain, United Kingdom, United States|
The disease, disorder, syndrome, illness, or injury that is being studied.
|Myeloproliferative Disorders, Myelofibrosis, Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Anemia|
Permitted Concomitant Medications and Procedures.
- - Subjects are receiving a JAK2 inhibitor for the treatment of MPN-associated MF that is approved in the country where the study is being conducted.
- - Best supportive care (BSC) includes, but is not limited to, treatment with transfusions (eg, RBC, platelet, whole blood), ICTs, antibiotic, antiviral and/or antifungal therapy, and nutritional support as needed.
- - Granulocyte colony-stimulating factors (ie, G-CSF, granulocyte macrophage colony-stimulating factor [GM-CSF]) are allowed only in cases of neutropenic fever or as clinically indicated per product label.
- - Prophylactic antithrombotic therapy is permitted.
- - Thrombopoietin and platelet transfusions are permitted.
- - Treatment with systemic corticosteroids is permitted for nonhematological conditions providing the subject is receiving a constant dose equivalent to ≤ 10 mg prednisone during the study.
- - Administration of attenuated vaccines (eg, influenza vaccine) is allowed if clinically indicated per Investigator discretion.
- - Iron chelation therapy (ICT) is to be used according to the product label.
- - Cytotoxic, chemotherapeutic, targeted, or investigational agents/therapies (excluding JAK2 inhibitor therapy) - Azacitidine, decitabine, or other hypomethylating agents.
- - Lenalidomide, thalidomide, and pomalidomide.
- - Erythropoietin stimulating agents (ESAs) and other RBC hematopoietic growth factors (eg, IL-3) - Hydroxyurea or other alkylating agents.
- - Androgens (unless given to treat hypogonadism) - Oral retinoids (topical retinoids are permitted) - Arsenic trioxide.
- - Interferon.
- - Anagrelide.
- - Systemic corticosteroids at a dose equivalent to > 10 mg prednisone.
Experimental: Experimental Arm: Luspatercept (ACE-536)
Luspatercept will be given to participants via subcutaneous injection (administered on Day 1 of each 21-day treatment cycle)
Placebo Comparator: Control Arm: Placebo
Placebo starting dose with volume equivalent to experimental arm subcutaneous injection every 3 weeks (administered on Day 1 of each 21-day treatment cycle)
Drug: - ACE-536
Other: - Placebo
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.