Phase 1 Congenital Myasthenic Syndrome Clinical Trials

2 recruitingPhase 1

What is a Phase 1 trial?

Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.

Showing 1–2 of 2 trials

Recruiting

Phase 1

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

Congenital Myasthenic SyndromeCMS

argenx16 enrolled9 locationsNCT06436742

Recruiting

Phase 1

Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution

Congenital Myasthenic Syndrome

The Foundation for Orthopaedics and Regenerative Medicine20 enrolled3 locationsNCT07226726