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NCT06630650 · ClinicalTrials.gov registry record
A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes
A clinical trial of Myasthenic Syndromes, Congenital, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Recruiting
- Registry status
- 75
- Enrollment target
- 1
- Study location
NCT06630650: Recruiting study of Myasthenic Syndromes, Congenital, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
NCT06630650 is a study of Myasthenic Syndromes, Congenital that is actively recruiting participants, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 75 participants. The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06630650, a study of Myasthenic Syndromes, Congenital, is actively recruiting participants, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- RECRUITING
- Registry status
- 75 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: Congenital myasthenic syndromes (CMSs) are a group of inherited disorders that affect how the nerves communicate with muscles. These can cause many problems that affect how people can move and use their bodies. Objective: This is a natural history study to learn more about how CMSs affect the body and cause changes over time. Eligibility: People aged 6 months or older with a CMS. The study will focus on DOK7- and COLQ-related CMSs, as well as other forms. Design: Participants will have up to 7 visits in 5 years. At each visit, participants will undergo many tests, including: Physical exam with blood and urine tests. Tests of their heart and lung function. Exams of the eyes, lungs, muscles, and nerves. These will be done with different specialists. Exams of the arms and hands and of body use and movements. These will also be done with specialists. Photos and videos may be taken. Muscle ultrasound. Participants will lie still as a wand is rubbed over their skin. Magnetic resonance imaging (MRI) scans. Participants will lie still on a bed that slides partway into a large tube. A parent or other person may remain in the room, too. The scan will take 60 minutes. Electromyography (EMG). Participants will lie still or may be asked to move around. A machine will measure the electrical activity in their muscles. An activity monitor may be placed on the participant s wrist, ankle, or hip for up to 2 weeks. The monitor is about the size of a wristwatch. A sample of skin may be removed....
Conditions Studied
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 75 participants |
| Start Date | 2025-05-12 |
| Est. Completion | 2044-12-02 |
What NCT06630650 shows while recruiting
NCT06630650 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 75 participants, a relatively small participant target.
The record links to 1 condition, with Myasthenic Syndromes, Congenital appearing as the primary indexed condition, and to 0 interventions.
NCT06630650 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT06630650 about?
NCT06630650 is a clinical study titled "A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes". Background: Congenital myasthenic syndromes (CMSs) are a group of inherited disorders that affect how the nerves communicate with muscles. These can cause many problems that affect how people can move and use their bodies. Objective: This is a natural history study to learn more about how CMSs af...
What is the current status of trial NCT06630650?
This trial is currently recruiting. The enrollment target is 75 participants. The study started on 2025-05-12. Estimated completion is 2044-12-02.
What conditions does trial NCT06630650 study?
This clinical trial studies the following conditions: Myasthenic Syndromes, Congenital.
Who is sponsoring clinical trial NCT06630650?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 567 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06630650 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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