Rollover study to evaluate participants involved with luspatercept
Research type
Research Study
Full title
A Phase 3b, open-label, single-arm, rollover study to evaluate long-term safety in subjects who have participated in other luspatercept (ACE-536) clinical trials
IRAS ID
261663
Contact name
Ghulam Mufti
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2018-002915-93
Duration of Study in the UK
7 years, 8 months, 5 days
Research summary
Myelodysplastic syndrome (MDS) is a bone marrow disorder which causes anaemia. The standard treatment for the loss of red blood cells is supportive treatment with erythropoiesis-stimulating agents (ESAs) and administration of red blood cell (RBC) transfusions.
Beta-thalassemia is an inherited blood disorder that reduces the production of haemoglobin. People with betathalassemia have a shortage of red blood cells (anaemia) and low levels of haemoglobin leading to a lack of oxygen in many parts of the body causing weakness, fatigue and serious complications. Current treatment options for beta-thalassemia include blood transfusion, iron chelation therapy, folic acid supplements, blood and marrow stem cell transplant and splenectomy.
Myeloproliferative neoplasm (MPN)-associated myelofibrosis is a disease found in participants bone marrow (soft tissue inside participants bones that makes blood cells), causing significant scarring, thereby causing the participants body to produce too few or too many cells that make blood, including red and white blood cells and platelets.
Luspatercept (pronounced lus pat’ er sept), works by increasing the production of red blood cells. Parental studies preliminary data suggests that Luspatercept is likely to lower ineffective erythropoiesis, correct the anaemia that characterises MDS and could provide significant clinical benefit to participants by improving haemoglobin levels and reducing regular RBC transfusions.
Participants will continue the same treatment from their parent studies. There are 3 parts for participants rolling over on treatment: Transition Phase, Treatment Phase and Follow-up Phase. Long-term follow-up participants will enter the Follow-up Phase only.
The purpose of this study is to ensure continued supply of the investigational drug for participants rolling over on treatment or for Long-term follow-up at the time their parent protocol is closed; continued supply of the investigational drug and to evaluate the long-term effect of the investigational drug.
742 participants will be enrolled worldwide. The study is sponsored by Celgene Corporation.
REC name
London - Westminster Research Ethics Committee
REC reference
19/LO/1215
Date of REC Opinion
4 Nov 2019
REC opinion
Further Information Favourable Opinion