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

Part of paid clinical trials in Sacramento, California.

Sponsor
argenx
Study ID
NCT06078553
Status
Recruiting
Apply to this trial

Conditions

  • Congenital Myasthenic Syndrome

Eligibility Criteria

Sex
ALL
Age
2 Years - N/A
Healthy Volunteers
Not accepted

Study Details

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

Key Dates

Start date
Feb 13, 2024
Status verified
Apr 2026
Primary completion
Jun 30, 2027
Completion
Jun 30, 2027

Study Design

Enrollment
100 participants (estimated)

Primary Outcome Measure

Summary statistics of retrospective and prospective collection of data on diagnosis. [ Time Frame: Up to 12 months ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
UC Davis Health - UC Davis Health Midtown Ambulatory Care CenterSacramentoCalifornia95816
Ricardo Marselli, MD
[email protected]
857-350-4834
Ann and Robert H Lurie Children's Hospital of Chicago - Main HospitalChicagoIllinois60611
Nancy Kuntz, MD
[email protected]
857-350-4834
Hospital Sisters Health System (HSHS) - St Elizabeth's HospitalO'FallonIllinois62269
Raghav Govindarajan, MD
[email protected]
857-350-4834
Harvard Medical School - Boston Children's HospitalBostonMassachusetts02115
Partha Ghosh, MD
[email protected]
857-350-4834
[email protected]
+1 857-350-4834
Ohio State Martha MoorehouseColumbusOhio43221
Miriam Freimer, MD
[email protected]
+1 857-350-4834
[email protected]
+1 857-350-4834
The Children's Hospital of Philadelphia (CHOP)PhiladelphiaPennsylvania19104
John Brandsema, MD
[email protected]
+1 857-350-4834
[email protected]
+1 857-350-4834

Find similar trials in Sacramento, CA

By research site
UC Davis Health - UC Davis Health Midtown Ambulatory Care Center· Sacramento, CAAnn and Robert H Lurie Children's Hospital of Chicago - Main Hospital· Chicago, ILHospital Sisters Health System (HSHS) - St Elizabeth's Hospital· O'Fallon, ILHarvard Medical School - Boston Children's Hospital· Boston, MAOhio State Martha Moorehouse· Columbus, OHThe Children's Hospital of Philadelphia (CHOP)· Philadelphia, PA

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