Phase 3 study of ACE-536 in MPN-associated myelofibrosis & anaemia
Research type
Research Study
Full title
A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Subjects with Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions The “INDEPENDENCE” Trial
IRAS ID
289604
Contact name
Claire Harrison
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2020-000607-36
Duration of Study in the UK
7 years, 10 months, 23 days
Research summary
This is a clinical research study of a potential new drug, Luspatercept (ACE-536) in participants with anaemia due to myeloproliferative neoplasm associated myelofibrosis, which is a disease that causes significant scarring of your bone marrow [soft tissue inside your bones that make blood cells] and disrupts the body’s ability to produce normal production of blood cells, (including red blood cells).
Luspatercept is a first in class erythroid (red blood cell) maturation agent being developed to treat patients who have serious blood disorders associated with ineffective erythropoiesis (production of red blood cells), also known as anaemia, and has the potential to reduce or eliminate the need for regular red blood cell transfusions.
This study will include approximately 28 participants from 6 sites in the UK. Approximately 309 participants will take part in the study worldwide and participants may be in the study for a maximum of 5 years.
Following a 28-day screening period, participants will be assigned to one of two treatment groups at random. Neither the participant, nor the study doctor will know which treatment group they will be assigned. Participants have a 2 in 3 chance of receiving luspatercept and a 1 in 3 chance of receiving placebo. Study treatment will be given on Day 1 of every 21-day treatment cycle as a subcutaneous injection (under the skin).
After randomization, participants will complete a 24-week core treatment period. Participants who benefit from the study drug will enter the extension phase treatment period. After stopping study treatment, they will continue in a follow-up period of at least 3 years with assessments every 3 months.The study involves procedures including physical examinations, vital signs, blood, saliva and urine sample testing, Electrocardiogram (ECG), and completion of questionnaires.
The study is sponsored by Celgene Corporation, a wholly owned subsidiary of Bristol-Myers Squibb.
REC name
South Central - Oxford C Research Ethics Committee
REC reference
20/SC/0434
Date of REC Opinion
19 Jan 2021
REC opinion
Further Information Favourable Opinion