Phase 2 proof of concept study evaluating efficacy, safety of daxdilimab in adults with DM or ASIM
Research type
Research Study
Full title
A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLEDEFFICACY AND SAFETY STUDY OF DAXDILIMAB SUBCUTANEOUS INJECTION IN ADULT PARTICIPANTS WITH INADEQUATELY CONTROLLED DERMATOMYOSITIS OR ANTI-SYNTHETASE INFLAMMATORY MYOSITIS
IRAS ID
1006956
Contact name
Stephanie Bekker-Njehia
Contact email
Sponsor organisation
Horizon Therapeutics Ireland DAC
Clinicaltrials.gov Identifier
Research summary
This is a randomised double-blind placebo-controlled study in participants with inadequately controlled dermatomyositis (DM) or anti synthetase inflammatory myositis (ASIM).
Myositis is a group of rare conditions affecting the muscles and, in some cases, other body parts like skin, lungs, or heart. It is caused by the body’s own defense system attacking the muscle, which causes them to be weak, tired, and swollen. Patients are often in pain, feel unwell, and may have skin or lung complications.
This study is being done to know how well a drug called daxdilimab works and how safe it is in participants with DM or ASIM for whom the usual treatment does not seem to work. This study also aims to describe over time how the body reacts to daxdilimab and how the drug is taken up, broken down, and removed from the body.
Daxdilimab is a protein designed to recognise and attach to a cell involved in the origin and development of conditions, like myositis, where the immune system attacks normal tissue. It is believed that by attaching to these cells, daxdilimab could reduce their number, and in this way reduce the activity of the immune system and help in the potential treatment of these conditions.
This study consists of a 4 week screening period, 48 week treatment period and a 8 week safety follow up period. Maximum duration 60 weeks.
Up to 96 participants will be randomised in a 1:1 ratio to receive daxdilimab 300 mg or placebo. At Week 24, participants who until then received placebo will receive 300 mg of the study drug, while those who received the study drug will continue with the same dose. Therefore, from Week 24 to Week 44, all participants will receive daxdilimab.
Study drug or placebo will be administered once every 4 weeks by 2 injections under the skin. Participants will receive 12 doses in total from Day 1 to Week 44.
Study procedures include vital signs, questionnaires, ECGs, blood, and urine samples, and optional MRI.REC name
South Central - Hampshire B Research Ethics Committee
REC reference
23/SC/0138
Date of REC Opinion
3 Oct 2023
REC opinion
Further Information Favourable Opinion