A Phase 1b study investigating ARGX-119 in adult participants with DOK7 CMS
Research type
Research Study
Full title
A Phase 1b, Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes
IRAS ID
1010165
Contact name
Sabine Coppieters
Contact email
Sponsor organisation
Argenx
ISRCTN Number
ISRCTN83274361
Clinicaltrials.gov Identifier
Research summary
Congenital myasthenic syndromes (CMS) are a group of rare neuromuscular disorders caused by genetic defects affecting the neuromuscular junction (NMJ). The NMJ is a specialised synapse between motor neurons and muscle cells, essential for movement and breathing. One of the more frequent causes of CMS is mutation of the DOK7 gene (DOK7-CMS), which accounts for 15% to 20% of CMS cases DOK7 mutations account for 15-20% of CMS cases. Currently, there are no FDA- (or other health authority) approved treatments for CMS. There is an unmet need for new treatments that improve NMJ stability and function to provide clinical benefit to CMS patients. ARGX-119 is an experimental study drug that may help maintain the links between nerves and muscles and improve the signaling between them when this may have been disrupted by a disease or syndrome, such as CMS. This may improve muscle strength and reduce tiredness from physical activity. The purpose of this study is to learn if ARGX-119 is safe for participants with CMS who have changes (mutations) in a gene called DOK7. The study will also research how the body processes and removes ARGX-119, how the immune system reacts to ARGX-119, and how ARGX-119 affects the way participants feel and function. This clinical study is planned to be conducted at approximately 12 study sites in Austria, France, Italy Spain, Canada, United Kingdom and North America. The study population will include adult participants with confirmed diagnosis of DOK7-CMS. Approximately 15 participants will be enrolled, screened, and randomised to receive ARGX-119 or placebo. The study will be up to approximately 11 months long. This is the first time that ARGX-119 will be given to participants with CMS.
REC name
HSC REC B
REC reference
24/NI/0087
Date of REC Opinion
14 Aug 2024
REC opinion
Further Information Favourable Opinion