A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Adimanebart IV, Placebo IV.
- Who it may be relevant to
- Registry conditions: Congenital Myasthenic Syndrome, CMS. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4-CMS
Overview
The purpose of this study is to assess efficacy and safety of adimanebart in participants at least 12 years of age with DOK7-, MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS). The study aims to determine whether adimanebart is safe and can help people with CMS feel better and perform daily activities more easily. The study includes a double-blinded treatment period (DBTP) and an Open- label extension period (OLE). In the DBTP, all participants will be randomized in a 1:1 ratio to adimanebart or placebo. Participants who complete the DBTP will continue to the OLE. Additionally, participants who complete part of the active-treatment period of ARGX-119-2302 study are eligible to enroll in the OLE of this study. In the OLE, all participants will receive open-label adimanebart. After final IMP dose, the participants will enter a follow-up period and their health will be monitored. The total duration of the study is up to approximately 152 weeks (2 years and 11 months). More information can be found here: clinicaltrials.argenx.com/Comets
Interventions
- Biological Adimanebart IV
Intravenous infusion of Adimanebart - Other Placebo IV
Intravenous infusion of Placebo
Primary outcome measures
- Change from baseline at week 24 in 6MWT distance [Time frame: Up to 24 weeks]
- Incidence of AEs and SAEs [Time frame: up to 104 weeks]
Secondary outcome measures (12)
- Change from baseline in 6MWT distance over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in PROMIS PF-10b T-score over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in QMG key component composite score over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in 6MWT cadence over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in the QMG key component raw values and scores over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in PROMIS PF-WMA-SF T-score over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in Neuro-QoL Short Form-Fatigue T-score over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in FVC over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in the Actigraphy measures over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in PGI-C over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in PGI-S over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
- Change from baseline in CGI-C over time [Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)]
Eligibility criteria
Inclusion criteria
DBTP:
- At least 12 years of age.
- Has a diagnosis of DOK7-, MUSK-, AGRN-, or LRP4-CMS with documented mutations.
- Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) or other CMS medication must have been receiving the medication for at least 6 months and agree to remain on a same stable dosing regimen of the same medication unless directed to change their CMS medication(s) by their treating physician.
OLE:
- Completed part of the active-treatment period of ARGX-119-2302.
Exclusion criteria
DBTP:
- Known medical condition that would interfere with an accurate assessment of CMS, confound the results of the study, or put the patient at undue risk, as assessed by the investigator.
OLE:
- Investigational study drug discontinuation in ARGX-119-2302.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07746089 · ARGX-119-17-CMS-3001 · 2025-524460-37-00