MEDALIST Trial | Aplastic Anemia and MDS International Foundation

ClinicalTrials.gov Identifier:

NCT02631070

Company

Celgene Acceleron

Contact Info

Daniel Matlosz, Clinical Trial Manager

Call (888) 260-1599

Dates

Start: December 2015
End: June 2019

Official Title

MEDALIST Trial

Purpose

The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in subjects with anemia due to International Prognostic Scoring System-Revised (IPSS-R) very low, low, or intermediate Myelodysplastic syndrome (MDS) with ring sideroblasts (≥ 15%) who require Red blood cell (RBC) transfusions.

Instructions

If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.

Status: 
Recruiting
Associated Drug(s): 
Phase: 
Phase 3
Gender: 
Female
Male
Age Group: 
18 and older
Accepts Healthy Volunteers: 
No
Inclusion Criteria: 

Subjects must satisfy the following criteria to be enrolled in the study:

  1. Subject is ≥ 18 years of age the time of signing the informed consent form (ICF).
  2. Documented diagnosis of MDS according to World Health Organization (WHO)/French American British (FAB) classification that meets IPSS R classification of very low, low, or intermediate risk disease, and:
    - Ring sideroblast ≥ 15% of erythroid precursors in bone marrow or ≥ 5% (but < 15%) if SF3B1 mutation is present.
    - < 5% blasts in bone marrow
    - Peripheral blood WBC count < 13,000/μL
  3. Requires RBC transfusions, as documented by the following criteria:
  • average transfusion requirement of ≥ 2 units/8 weeks of packed red blood cells (pRBCs) confirmed for a minimum of 16 weeks immediately preceding randomization.
  • Hemoglobin levels at the time of or within 7 days prior to administration of a RBC transfusion must have been ≤ 10.0 g/dL in order for the transfusion to be counted towards meeting eligibility criteria. Red blood cell transfusions administered when Hgb levels were > 10.0 g/dL and/or RBC transfusions administered for elective surgery will not qualify as a required transfusion for the purpose of meeting eligibility criteria.  
  • no consecutive 56-day period that was RBC transfusion-free during the 16 weeks immediately preceding randomization

4.    Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2

5.    Subjects who ar refractory/intolerant/ineligible to prior ESA treatment, defined as:

  • Refractory to prior Erythropoiesis- stimulating agents(ESA) treatment: documentation of non-response or response that is no longer maintained to prior ESA-containing regimen, either as single agent or combination (eg, with G-CSF); ESA regimen must have been either recombinant human erythropoietin (rHu EPO) > 40,000 IU/wk for at least 8 doses or equivalement OR darbepoetin alpha > 500 μg Q3W for at least 4 doses or equivalent 
  • Intolerant to prior ESA treatment: documentation of discontinuation of prior ESA-containing regimen, either as single agent or combination (eg, with G-CSF), at any time after introduction due to intolerance or an adverse event
  • ESA ineligible: low chance of response to ESA base on endogenous serum erythropoietin level > 200 U/L for subjects not previously treated with ESAs
Exclusion Criteria: 

The presence of any of the following will exclude a subject from enrollment:

  1. Prior therapy with disease modifying agents for underlying MDS disease (eg, immune-modulatory drug [IMiDs such as lenalidomide], hypomethylating agents, or immunosuppressive therapy [IST]).     
  2. Previously treated with either luspatercept (ACE-536) or sotatercept (ACE-011)
  3. MDS associated with del 5q cytogenetic abnormality
  4. Secondary MDS, ie, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases.
  5. Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
    - iron deficiency to be determined by a bone marrow aspirate stain for iron, calculated transferrin saturation (iron/total iron   binding capacity) ≤ 20%, or serum ferritin ≤ 15 μg/L 
  6. Prior allogeneic or autologous stem cell transplant
  7. Known history of diagnosis of Acute myeloid leukemia (AML)
  8. Use of any of the following within 5 weeks prior to randomization:
    • anticancer cytotoxic chemotherapeutic agent or treatment
    • corticosteroid, except for subjects on a stable or decreasing dose for ≥ 1 week prior to randomization for medical conditions other than MDS
    • iron-chelating agents, except for subjects on a stable or decreasing dose for at least 8 weeks prior to randomization
    • other RBC hematopoietic growth factors (eg, Interleukin-3)
    • investigational drug or device, or approved therapy for investigational use.  If the half-life of the previous investigational product is known, use within 5 times the half-life prior to randomization or within 5 weeks, whichever is longer is excluded. 
  9. Major surgery within 8 weeks prior to randomization. Subjects must have completely recovered from any previous surgery prior to randomization.
  10. Prior history of malignancies, other than MDS, unless the subject has been free of the disease (including completion of any active or adjuvant treatment for prior malignancy) for ≥ 5 years. However, subjects with the following history/concurrent conditions are allowed:
    • Basal or squamous cell carcinoma of the skin
    • Carcinoma in situ of the cervix
    • Carcinoma in situ of the breast
    • Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system)

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