Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Congenital Myasthenic Syndrome: early-phase trial registry
3 of 3 indexed Congenital Myasthenic Syndrome trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #5,209 of 28,707 conditions by registered trial count.
3 US clinical trials · 3 currently recruiting · #5,209 of 28,707 conditions by registered trial count
Congenital Myasthenic Syndrome: 3 US clinical trials tracked, 3 recruiting.
Congenital Myasthenic Syndrome is the subject of 3 registered US clinical trials on ClinicalTrials.gov, 3 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 2 in Phase 1-2 (earlier-stage). The most active sponsor is argenx, running 2 of these trials.
Enrollment posture brief
Registry condition congenital-myasthenic-syndrome
OPEN 3 | RECR 100% | LATE 0 | EARLY 2 | RANK #5209 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Aortic Arch Aneurysm (100% recruiting · 5 trials)
Congenital Myasthenic Syndrome lists 3 open slots (100% of 3 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Congenital Myasthenic Syndrome on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Congenital Myasthenic Syndrome is recruiting above its size-band peers.
3 of 3 indexed trials (100%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
Open and recent trials
3 total, page 1 of 1
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
argenx
NCT06078553
Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution
The Foundation for Orthopaedics and Regenerative Medicine
NCT07226726
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
argenx
NCT06436742
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Congenital Myasthenic Syndrome records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Congenital Myasthenic Syndrome trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Congenital Myasthenic Syndrome page
3 registered trials, 3 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 3 Congenital Myasthenic Syndrome trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- argenx sponsors the most Congenital Myasthenic Syndrome trials on record, review their full trial history before evaluating a specific study. See argenx's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Congenital Myasthenic Syndrome condition card totals 3 registered trials; most-linked intervention AlloEx exosomes; top US site state Illinois. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.