A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Public ClinicalTrials.gov record NCT06078553. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Multicenter, Multinational, Natural History Study in Participants With Congenital Myasthenic Syndromes Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7, MUSK, AGRN, or LRP4. More information can be found here: https://clinicaltrials.argenx.com/cms
Study identification
- NCT ID
- NCT06078553
- Recruitment status
- Recruiting
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 100 participants
Conditions and interventions
Conditions
Interventions
Not listed
Eligibility (public fields only)
- Age range
- 2 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 12, 2024
- Primary completion
- May 31, 2027
- Completion
- May 31, 2027
- Last update posted
- Jul 21, 2026
2024 – 2027
United States locations
- U.S. sites
- 6
- U.S. states
- 5
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UC Davis Health - UC Davis Health Midtown Ambulatory Care Center | Sacramento | California | 95816 | Recruiting |
| Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital | Chicago | Illinois | 60611 | Recruiting |
| Hospital Sisters Health System (HSHS) - St Elizabeth's Hospital | O'Fallon | Illinois | 62269 | Recruiting |
| Harvard Medical School - Boston Children's Hospital | Boston | Massachusetts | 02115 | Recruiting |
| Ohio State Martha Moorehouse | Columbus | Ohio | 43221 | Recruiting |
| The Children's Hospital of Philadelphia (CHOP) | Philadelphia | Pennsylvania | 19104 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 26 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06078553, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 21, 2026 · Synced Sep 2, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06078553 live on ClinicalTrials.gov.