Congenital Myasthenic SyndromePhase 3CoMetSNCT07746089
A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)
A Phase 3 study testing Adimanebart IV in people with Congenital Myasthenic Syndrome and CMS, run by argenx.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Congenital Myasthenic Syndrome, CMS
- What is being tested?
- Adimanebart IV
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 105 participants
- Where
- No sites listed yet
- Study dates
- Sep 2026to Oct 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
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
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.
Read more
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
- Conditions
- Congenital Myasthenic Syndrome, CMS
- Being tested
- Adimanebart IV, Placebo IV
- Medical subject headings
- Myasthenic Syndromes, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adimanebart IVBiological
Intravenous infusion of Adimanebart
Placebo IVOther
Intravenous infusion of Placebo
| Study group | Role | What they receive |
|---|---|---|
| Double-blinded treatment period (DBTP) - Adimanebart IV | Experimental | Participants randomized to receive Adimanebart IV Receives: Biological: Adimanebart IV |
| Double-blinded treatment period (DBTP) - Placebo IV | Placebo comparator | Participants randomized to receive Placebo IV Receives: Other: Placebo IV |
| Open-label extension (OLE) - Adimanebart IV | Experimental | Participants receive Adimanebart IV Receives: Biological: Adimanebart IV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline at week 24 in 6MWT distance
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
Measured: Up to 24 weeks
Incidence of AEs and SAEs
AE: adverse event; SAE: serious adverse event
Measured: up to 104 weeks
Secondary outcomes (18)
Change from baseline in 6MWT distance over time
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
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)
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
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).
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
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.
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in the QMG key component raw values and scores 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).
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in PROMIS PF-WMA-SF T-score over time
The PROMIS PF-WMA-SF T-score (Patient-Reported Outcomes Measurement Information System Physical Function With Mobility Aid Short Form) is an 11-item, participant-completed questionnaire that assesses lower and upper extremity function and associated activities of daily living. The questionnaire asks the participant to rate the items on a 5-point scale of 5 (without any difficulty) to 1 (unable to do).
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in Neuro-QoL Short Form-Fatigue T-score over time
Neuro-QoL Short Form-Fatigue (Quality of Life in Neurological Disorders Short Form - Fatigue) is a participant-completed short-form questionnaire designed to provide a measurement of fatigue in patients with neurological conditions and the impact of their fatigue on their quality of life and daily activities.
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in FVC over time
FVC: forced vital capacity to assess pulmonary function
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in the Actigraphy measures over time
Participants will be asked to wear a wrist-worn actigraphy device to assess physical behaviour and mobility.
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in PGI-C over time
The Patient Global Impression of Change (PGI-C) is a patient-reported rating scale to assess the change in symptom severity and functional problems since the first IMP administration. Participants are asked to rate their change in symptom severity and functional problems on a 7- point Likert scale from 1 (much improved) to 7 (much worse).
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in PGI-S over time
The Patient Global Impression of Severity (PGI-S) scale is a patient-reported rating scale to assess symptom severity and functional problems. Participants are asked to rate their symptom severity and functional problems on a 7-point Likert scale from 1 (no problems) to 7 (unable to do).
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in CGI-C over time
The Clinical Global Impression of Change (CGI-C) scale is a clinician-reported rating scale to assess changes in a participant's symptom severity and functional problems since the first IMP administration. Clinicians are asked to rate the change in the participant's symptom severity and functional problems on a 7-point Likert scale from 1 (much improved) to 7 (much worse)
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in CGI-S over time
The Clinical Global Impression of Severity (CGI-S scale) is a clinician-reported rating scale to assess a participant's symptom severity and functional problems. The scale comprises a single item. Based on clinical judgment, clinicians rate the participant's symptom severity and functional problems on a 4-point Likert scale from 0 (no problems) to 4 (unable to do).
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in EQ-5D-5L over time
The participant-completed EQ-5D-5L questionnaire is a standardized test recognized by many health authorities as a generic measure of health status.
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Incidence of AEs and SAEs
AE: adverse event; SAE: serious adverse event
Measured: up to 24 weeks
Adimanebart serum concentrations over time
PK=pharmacokinetic(s)
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Incidence of ADA against adimanebart
ADA: Anti-drug antibodies
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Incidence of NAb against adimanebart
Nab: neutralizing antibody(ies)
Measured: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2026
- Study startSep 2026 (estimated)
- Primary completionOct 2030 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Myasthenic SyndromeStudies of the same condition, open and under way.
- More trials run by argenxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 105 participants (estimated)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07746089
- Sponsor's protocol ID
- ARGX-119-17-CMS-3001
- Other IDs
- 2025-524460-37-00, 2025
- Acronym
- CoMetS
- Status last verified
- Oct 2026
- First submitted
- 30 Jul 2026
- Last updated
- 19 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07746089
- Acronym / sponsor ID
- CoMetS / ARGX-119-17-CMS-3001
- Conditions
- Congenital Myasthenic Syndrome, CMS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 105 participants (estimated)
- Lead sponsor
- argenxIndustry (pharma, biotech, devices)
- Study start
- Sep 2026 (estimated)
- Primary completion
- Oct 2030 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 4 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Double-blinded treatment period (DBTP) - Adimanebart IV | Experimental | Biological: Adimanebart IVParticipants randomized to receive Adimanebart IV |
| Double-blinded treatment period (DBTP) - Placebo IV | Placebo comparator | Other: Placebo IVParticipants randomized to receive Placebo IV |
| Open-label extension (OLE) - Adimanebart IV | Experimental | Biological: Adimanebart IVParticipants receive Adimanebart IV |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Adimanebart IVIntravenous infusion of Adimanebart | Biological | Double-blinded treatment period (DBTP) - Adimanebart IV, Open-label extension (OLE) - Adimanebart IV |
| Placebo IVIntravenous infusion of Placebo | Other | Double-blinded treatment period (DBTP) - Placebo IV |
Primary outcome
Change from baseline at week 24 in 6MWT distance
Time frame: Up to 24 weeks
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
Incidence of AEs and SAEs
Time frame: up to 104 weeks
AE: adverse event; SAE: serious adverse event
18 secondary outcomes
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)
Change from baseline in CGI-S over time
Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in EQ-5D-5L over time
Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Incidence of AEs and SAEs
Time frame: up to 24 weeks
Adimanebart serum concentrations over time
Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Incidence of ADA against adimanebart
Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Incidence of NAb against adimanebart
Time frame: up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2026.
- Study websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Adimanebart IV in people with Congenital Myasthenic Syndrome and CMS, run by argenx.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sabine Coppieters, MD (857-350-4834, clinicaltrials@argenx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline at week 24 in 6MWT distance (time frame: Up to 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026.