Skip to content
DataLookout
Start free
Menu

NCT06078553

A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4

StatusActive, Not Recruiting
PhaseNot specified
Sponsorargenx
Primary completion (estimated)

At risk: Primary completion moved at least 2 months later.

See at-risk trials from Argenx

Track this trial

Free. We email you which fields changed when ClinicalTrials.gov updates this record.

Record History

All 23 versions: 22 with changes below, 1 re-verified with no change. See every version on ClinicalTrials.gov ↗

  1. Enrollment closed, study ongoing
    • Primary and study completion pushed from Jun 2027 to Sep 2027
    • Final enrollment: 152 participants (planned 100)
    • Site details revised at 33 of 33 sites
  2. Eligibility criteria expanded, about 8 words
    • Study sites: 32 to 33
    • Secondary endpoints: details revised at 6 of 7 entries
  3. Study sites: 31 to 32v21
  4. Study sites: 30 to 31v20
  5. Trial sites expanded: 23 → 30 locations
    • Site details revised at 10 of 30 sites
    v19
  6. Trial sites expanded: 20 → 23 locationsv18
  7. Study sites: 20 to 23v16
  8. Study sites: 19 to 20v15
  9. Study Status, Study Description, Referencesv14
  10. Study sites: 18 to 19v13
  11. Study sites: 16 to 18v12
  12. Study sites: 13 to 16v11
  13. Study sites: 11 to 13v10
  14. Study sites: 10 to 11v9
  15. Enrollment target: 30 to 100v8 Show 5 more changesShow less
    • Primary and study completion dates: Jul 2025 to Jun 2027
    • Eligibility criteria expanded, about 19 words
    • Primary endpoints revised (4)
    • Secondary endpoints: 6 to 7
    • Study title expanded, about 8 words
  16. Study sites: 8 to 10v7
  17. Study sites: 7 to 8v6
  18. Study sites: 6 to 7v5
  19. Study sites: 5 to 6v4
  20. Study sites: 3 to 5v3
  21. Trial status: Not Yet Recruiting to Recruiting
    • Start date changed from estimate of Oct 2023 to Feb 13, 2024
    • Study sites: 0 to 3
    v2
  22. Study First Submitted
  • Notable: the plan changed
  • Minor: routine updates

Endpoints

Primary

Summary statistics of retrospective and prospective collection of data on diagnosis.
Timeframe: Up to 12 months
Summary statistics of retrospective and prospective collection of data on health care utilization.
Timeframe: Up to 12 months
Summary statistics of retrospective and prospective collection of data on medications.
Timeframe: Up to 12 months
Summary statistics of retrospective and prospective collection of data on change in health status related to CMS
Timeframe: Up to 12 months

Secondary (7)

Change from baseline for QMG total score, each component score, and raw values
Change from baseline for MG-ADL total score
Change from baseline for PROMIS-GHS
Change from baseline for PROMIS-DFL
Change from baseline for EQ-5D-5L
Show 2 moreShow less
Change from baseline for 3TUG
Change from baseline for Neuro-QoL Fatigue score

Sites

33 sites in 12 countries

United States 7, France 6, Germany 3, Poland 3, Spain 3, Brazil 2, Canada 2, Japan 2, United Kingdom 2, Austria 1, Belgium 1, Italy 1