An extension to the phase 2 study A536-03 for the treatment of anemia in patients with myelodysplastic syndrome of low and intermediate risk groups
- Conditions
- Myelodysplastic Syndromes (MDS)MedDRA version: 20.0Level: SOCClassification code 10029104Term: Neoplasms benign, malignant and unspecified (incl cysts and polyps)System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 20.0Level: HLTClassification code 10028536Term: Myelodysplastic syndromesSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2014-001280-13-DE
- Lead Sponsor
- Acceleron Pharma Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 153
All patients must meet the following criteria:
1. Completion of the treatment period in the base study A536-03.
2. Females of child bearing potential (defined as sexually mature women who have not undergone hysterectomy or bilateral oophorectomy, or are not naturally postmenopausal = 24 consecutive months) must have negative urine or blood pregnancy test prior to enrollment and use adequate birth control methods (abstinence, oral contraceptives, barrier method with spermicide, or surgical sterilization) during study participation and for 12 weeks following the last dose of ACE-536. Males must agree to use a latex condom during any sexual contact with females of child-bearing potential while participating in the study and for 12 weeks following the last dose of ACE-536, even if he has undergone a successful vasectomy. Patients must be counseled concerning measures to be used to prevent pregnancy and potential toxicities prior to dosing with ACE-536.
3. Patient is able to adhere to the study visit schedule, understand and comply with all protocol requirements.
4. Patient understands and is able to provide written informed consent.
Patients with treatment interruption (defined as patients who complete their A536-03 EOS visit) must also meet the following criteria:
1. Documented diagnosis of idiopathic/de novo MDS or non-proliferative chronic myelomonocytic leukemia (CMML) according to the World Health Organization (WHO) criteria2 (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;
2. 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 NTD patients (defined as having received < 4 units of 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.
3. Platelet count = 30 x 10^9/L.
4. ECOG performance status of 0, 1, or 2 (if related to anemia).
5. 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 76
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 77
1. 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 AE, disease progression, persistent increase in white blood cell count (WBC), presence of = 1% blasts in peripheral blood, hypersensitivity reaction to the study drug, at the discretion of the sponsor, or loss to follow-up) prior to completion of the treatment period.
2. Prior treatment with azacitidine (injectable or oral) or decitabine.
3. Treatment within 28 days prior to Cycle 1 Day 1 with:
- ESA,
- Granulocyte colony-stimulating factor (G-CSF) and granulocyte-macrophage colony stimulating factor (GM-CSF),
- Lenalidomide.
4. For patients with treatment interruption only: Iron chelation therapy if initiated within 56 days prior to Cycle 1 Day 1.
5. 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.
6. 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.
7. Known positive for human immunodeficiency virus (HIV), active infectious hepatitis B (HBV) or active infectious hepatitis C (HCV).
8. Uncontrolled hypertension defined as systolic blood pressure (SBP) = 150 mm Hg or diastolic blood pressure (DBP) = 100 mm Hg.
9. Pregnant or lactating females.
10. History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational drug.
11. Any other condition not specifically noted above which, in the judgment of the investigator, would preclude the patient from participating in the study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method