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

Research summary

The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7-CMS. The study will also assess how ARGX-119 is processed by the body (PK), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function. This phase 1b, multicenter, randomized, double-blinded, placebo-controlled study is designed to assess the safety, tolerability, PK, immunogenicity, and preliminary efficacy of ARGX-119 in participants with DOK7-CMS. Eligible participants will be aged at least 18 years and have a confirmed diagnosis of CMS due to biallelic pathogenic mutations in DOK7.

Principal Investigator

Dr Jackie A Palace

Contact us

Email: ana.cavey@ouh.nhs.uk

IRAS number

1010165