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
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.
Completed part of the active-treatment period of ARGX-119-2302.
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.
Investigational study drug discontinuation in ARGX-119-2302.
Parallel
Adimanebart IV
Biological/VaccineIntravenous infusion of Adimanebart
Placebo IV
Other interventionIntravenous infusion of Placebo
Change from baseline at week 24 in 6MWT distance
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
Incidence of AEs and SAEs
AE: adverse event; SAE: serious adverse event
Change from baseline in 6MWT distance over time
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
Change from baseline in PROMIS PF-10b T-score over time
The PROMIS PF-10b T-score (Patient-Reported Outcomes Measurement Information System Physical Function 10b) is a 10- item, participant-reported short-form questionnaire designed to assess physical function. The questionnaire asks the participant to rate the items on a 5-point Likert scale of 5 (without any difficulty) to 1 (unable to do)
Change from baseline in QMG key component composite score over time
The Quantitative Myasthenia Gravis (QMG) scale is a standardized quantitative scoring system that was developed to assess disease severity based on impairment of body function and structures in patients with MG. Minimum value: 0 (no disease severity); Maximum value: 15 (highest disease severity).
Change from baseline in 6MWT cadence over time
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes. Cadence is the number of steps taken per unit of time (steps/min) and is a measure of functional mobility.
This trial is not recruiting at the moment. You can still explore other options:
