ACE-536 Extension Study - Myelodysplastic Syndromes

This is an interventional treatment trial for Myelodysplastic Syndromes Read More

Inclusion Criteria:

• Completion of the treatment period in the base study A536-03 (ClinicalTrials.gov Identifier:

NCT01749514)

• Adequate birth control measures
• Patient is able to adhere to the study visit schedule, understand and comply with all protocol requirements.
• Patient understands and is able to provide written informed consent.

In addition, patients with treatment interruption (defined as patients who complete their end-of-study visit in A536-03 and cannot directly roll over to A536-05) must also meet the following criteria:

• Documented diagnosis of idiopathic/de novo MDS or non-proliferative chronic myelomonocytic leukemia (CMML) according to the World Health Organization (WHO) criteria 2 (white blood count (WBC) < 13,000/μL) that meets International Prognostic Scoring System (IPSS) classification (Appendix 2) of low or intermediate-1 risk disease as determined by microscopic and standard cytogenetic analyses of the bone marrow and peripheral complete blood count (CBC) obtained during screening;
• Anemia defined as:
• Mean hemoglobin concentration < 10.0 g/dL of 2 measurements (one performed within one day prior to Cycle 1 Day 1 and the other performed 7-28 days prior to Cycle 1 Day 1), for non-transfusion dependent (NTD) patients (defined as having received ˂ 4 units of red blood cells (RBCs) within 8 weeks prior to Cycle 1 Day 1), OR
• Transfusion Dependent (TD), defined as having received ≥ 4 units of RBCs within 8 weeks prior to Cycle 1 Day 1.
• Platelet count ≥ 30 x 109/L
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia)
• Adequate renal (creatinine ≤ 2.0 x upper limit of normal [ULN]) and hepatic (total bilirubin < 2 x ULN and aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 3 x ULN) function

Exclusion Criteria:

• Discontinuation/withdrawal from the base study A536-03 (due to patient request, patient unwillingness or inability to comply with the protocol, pregnancy, use of prohibited medication [e.g. azacitidine], medical reason or adverse event (AE), hypersensitivity reaction to the study drug, at the discretion of the sponsor, or loss to follow-up) prior to completion of the treatment period
• Prior treatment with azacitidine or decitabine
• Treatment within 28 days prior to Cycle 1 Day 1 with:
• an erythropoiesis-stimulating agent (ESA),
• Granulocyte colony-stimulating factor (G-CSF) and granulocyte-macrophage colony stimulating factor (GM-CSF),
• Lenalidomide
• Iron chelation therapy if initiated within 56 days prior to Cycle 1 Day 1
• Treatment with another investigational drug (including sotatercept [ACE-011]) or device, or approved therapy for investigational use ≤ 28 days prior to Cycle 1 Day 1, or if the half-life of the previous investigational product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer
• Major surgery within 28 days prior to Cycle 1 Day 1. Patients must have completely recovered from any previous surgery prior to Cycle 1 Day 1
• Known positive for human immunodeficiency virus (HIV), active infectious hepatitis B (HBV) or active infectious hepatitis C (HCV)
• Uncontrolled hypertension defined as systolic blood pressure (SBP) ≥ 150 mm Hg or diastolic blood pressure (DBP) ≥ 100 mm Hg
• Pregnant or lactating females
• History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational drug
• Any other condition not specifically noted above which, in the judgment of the investigator, would preclude the patient from participating in the study