MEDALIST (HAEM 5606)13 Aug. 2018
Research FOR Patients
-For an informed and empowered opinion-
All the trials listed on our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.
MEDALIST IS NOW CLOSED TO RECRUITMENT
Treatment with Luspatercept resulted in a significantly reduced transfusion burden compared with placebo. Regulatory submissions planned in the United States and Europe in the first half of 2019.
- SUB-TYPE OF MDS: IPSS-R and ring sideroblasts
- SEVERITY OF MDS: Low or Intermediate
- NAME OF DRUG: Luspatercept, a protein found to promote red blood cell formation by regulating the growth of red blood cells during the late-stage of their development. It works differently to erythropoietin.
- Aims and benefits: This study is a double-blind, randomized study that compares the efficacy and safety of Luspatercept (ACE-536) versus placebo in this group of patients who require red blood cell transfusions. It aims to determine the proportion of patients that will become blood transfusion independent on Luspatercept.
- Primary outcomes: Red Blood Cell Transfusion Independence for ≥ 8 weeks
- Secondary outcomes:- Red Blood Cell Transfusion Independence for ≥ 12 weeks- Reduced red blood cell units transfused over 16 weeks
- Duration of Red Blood Cell Transfusion Independence
- Haematological improvement
- Change in quality of life score
- Basic inclusion criteria:
- Documented diagnosis of MDS that meets IPSS-R classification of very low, low, or intermediate risk disease + ring sideroblast 15% of erythroid precursors in bone marrow + 5% blasts in bone marrow
- Refractory (no response or lost response) to/intolerant to/ineligible (expected to have a low chance of response)for ESA (erythropoietic stimulating agents) treatment
- Requires red blood cell transfusions (average of 2 units every 8 weeks)
- Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2
- Basic exclusion criteria:
- Prior therapy with disease modifying agents (eg, immune-modulatory drug such as lenalidomide, hypomethylating agents such as azacitadine, or immunosuppressive therapy) or experimental agents for underlying MDS disease
- Previously treated with either luspatercept or sotatercept
- MDS associated with del 5q cytogenetic abnormality
- 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.
- Known clinically significant anaemia due to other causes
- Prior allogeneic or autologous stem cell transplant.
- Known history of diagnosis of AML.
- 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)
- Absolute neutrophil count of < 0.5 x 109/L.
- Platelet count of < 50 x 109/L.
- Significant kidney or liver impairment.
- Prior history of malignancies, other than MDS, unless the subject has been free of the disease for 5 years.
- History of stroke, deep venous thrombosis (DVT), myocardial infarction, pulmonary or arterial embolism, within 6 months prior to randomization.
- Trial sites/locations and name of physician in charge of trial:
Aberdeen Royal Infirmary Aberdeen, United Kingdom, AB25 2ZN
John Radcliffe Hospital Headington, United Kingdom, OX3 9DU
St James University Hospital Leeds, United Kingdom, LS1 3EX
Guys Hospital London, United Kingdom, SE1 9RT
Kings College Hospital London, United Kingdom, SE5 9RS
Kings Mill Hospital Sutton in Ashfield, United Kingdom, NG17 4SL
- More information: https://clinicaltrials.gov/ct2/show/NCT02279654
Please read information and always discuss trial information with your own physician.