Extended Access of Momelotinib in Myelofibrosis
Research type
Research Study
Full title
Extended Access of Momelotinib for Subjects with Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)
IRAS ID
242700
Contact name
Adam Mead
Contact email
Sponsor organisation
GlaxoSmithKline Research & Development Limited
Eudract number
2017-004350-42
Clinicaltrials.gov Identifier
Duration of Study in the UK
8 years, 5 months, 14 days
Research summary
Myelofibrosis (MF) is a bone marrow disorder that disrupts the body’s normal production of blood cells. The result is extensive scarring in the bone marrow, leading to severe anaemia, weakness, fatigue and often an enlarged spleen.
Treatments for MF are predominantly symptom controlling such as blood transfusions (for low blood counts). Other treatments are chemotherapy (for spleen and liver size reduction), splenectomy (removal of the spleen), radiotherapy (to reduce the size of the spleen if removal isn't possible) and the use of drugs such as biological therapies, steroids, JAK inhibitors and drugs which treat a low red blood count.
The genes JAK1 and JAK2 are important in the regulation of blood cells. Mutations in these genes have been implicated in MF. JAK1 and JAK2 inhibitors stop the JAK1 and JAK2 genes from signalling to stem cells (in the bone marrow) to make blood cells.Studies of Momelotinib (MMB) in adults have demonstrated that MMB may possess beneficial activity in patients with MF. Administration of MMB has resulted in clinical improvement in participants with MF with spleen and anaemia responses, as well as improvements in constitutional symptoms.
Patients who have been treated for a Secondary MF disorder (where MF is combined with another disorder) and patients with untreated Primary MF (where no other blood disorder was present) have taken part in 3 parent studies being treated with Momelotinib.
This extended access study will provide continued treatment with MMB for participants being treated with MMB in one of three parent studies, who have not experienced progression of disease whilst assessing the long-term safety of MMB.
This study is for 96 weeks and involves the participants self-administering MMB tablets daily, with study visits every 12 weeks for safety assessments. This study will enrol approximately 200 participants at around 100 sites globally.
REC name
London - Fulham Research Ethics Committee
REC reference
18/LO/0666
Date of REC Opinion
15 May 2018
REC opinion
Further Information Favourable Opinion