Phase 2 study of ACE-536 in MPN-associated myelofibrosis & anaemia
Research type
Research Study
Full title
A PHASE 2, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF LUSPATERCEPT (ACE-536) IN SUBJECTS WITH MYELOPROLIFERATIVE NEOPLASM-ASSOCIATED MYELOFIBROSIS AND ANEMIA WITH AND WITHOUT RED BLOOD CELL-TRANSFUSION DEPENDENCE
IRAS ID
229845
Contact name
Claire Harrison
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2017-000322-35
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
U1111-1197-1202, WHO Universal Trial Number (UTN)
Duration of Study in the UK
5 years, 11 months, 1 days
Research summary
The goal of this study is to evaluate the efficacy and safety of luspatercept (an investigational drug) in patients with anaemia (a decrease in red blood cells or haemoglobin [a protein in red blood cells that helps to carry oxygen throughout the body] in blood) and myeloproliferative neoplasm (MPN)-associated myelofibrosis (a disease found in your bone marrow [soft tissue inside your bones that make blood cells] that causes significant scarring that disrupts the body’s ability to produce normal production of blood cells, including red blood cells) who may or may not be dependent on red blood cell (RBC) transfusions.
Overall, the study will enroll approximately 70 patients worldwide in four parallel-enrolling cohorts: 1) Cohort 1 patients will be anaemic but not dependent on RBC transfusions; 2) Cohort 2 patients will be anaemic and dependent on RBC transfusions; 3) Cohort 3A patients will match the characteristics as those patients in Cohort 1, but will be on ruxolitinib as per their local standard of care; and 4) Cohort 3B patients will match the characteristics as those patients in Cohort 2, but will be on ruxolitinib as per their local standard of care.
A patient’s participation in the study can be up to approximately 5 years, which will consist of a 28-day Screening Period (to see if they will meet the eligibility criteria), approximately 6 months up to 2-year Treatment period in which luspatercept will be administered, and at least a 3-year Posttreatment Follow-up Period (in which several safety-related assessments will be captured). Throughout the study, patients will be evaluated to see if they experience an increase in their haemoglobin or become non-dependent on RBC transfusions.
REC name
London - Harrow Research Ethics Committee
REC reference
17/LO/1320
Date of REC Opinion
22 Aug 2017
REC opinion
Favourable Opinion