Find Congenital Myasthenic Syndrome Clinical Trials Near You
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
Status: Recruiting
Location: See all (9) locations...
Intervention Type: Other, Biological
Study Type: Interventional
Study Phase: Phase 1
SUMMARY
The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function. After the screening period, eligible participants will be randomized in a 4:1 ratio to receive intravenous infusions of ARGX-119 or placebo during the double-blinded treatment period. Participants will then enter the follow-up period. After the follow-up period, participants may enrol in the active-treatment period, where they will receive open-label ARGX-119. The full duration of the study is approximately 38 months.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• At least 18 years of age.
• Has genetically confirmed congenital myasthenic syndromes due to mutation of downstream of kinase 7 (DOK7-CMS).
• Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) must have been receiving the medication for more than 3 months and agree to remain on a same stable dosing regimen of the same medication until the end of the study.
Locations
United States
California
UC Davis Medical Center
ACTIVE_NOT_RECRUITING
Sacramento
Illinois
Ann and Robert H Lurie Childrens Hospital of Chicago
RECRUITING
Chicago
Other Locations
Canada
Ottawa Hospital Research Institute - Civic Campus
RECRUITING
Ottawa
France
CHU - Hospital de la Timone
COMPLETED
Marseille
Group Hospitalier Pitie-Salpetriere
RECRUITING
Paris
Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITING
Milan
Spain
Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)
ACTIVE_NOT_RECRUITING
Valencia
United Kingdom
Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital
RECRUITING
Belfast
John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
ACTIVE_NOT_RECRUITING
Oxford
Contact Information
Primary
Sabine Coppieters, MD
clinicaltrials@argenx.com
857-350-4834
Time Frame
Start Date:2024-09-24
Estimated Completion Date:2028-01-24
Participants
Target number of participants:16
Treatments
Experimental: Double-blinded treatment period - ARGX-119 IV
Participants receive ARGX-119 during the double-blinded treatment period
Placebo_comparator: Double-blinded treatment period - Placebo IV
Participants receive placebo during the double-blinded treatment period
Experimental: Active-treatment period - ARGX-119 IV
Participants receive ARGX-119 during the active-treatment period