By InvitationNot Applicable

A Registered Observational Cohort Study of Myotonic Dystrophy Type 1

China300 participantsStarted 2008-01

Plain-language summary

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy.There is little phenotype and genetic data for Chinese DM1 patients. The data to be collected is intended to fill this gap and provide complementary data

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male or female subjects of all ages at baseline * Subjects, with or without symptoms, with DM1 genetic confirmation through triplet-primed PCR or long-read sequencing * Unrelated healthy controls Exclusion Criteria: * Decline to participate * Other neuromuscular disease (such as Limb-girdle muscular dystrophy or Oculopharyngodistal Myopathy) * Serious systemic illness (such as heart, liver, kidney disease or major mental illness)

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Triplet-primed PCR or Long-read sequencing

Timeframe: Baseline

Muscle Impairment Rating Scale (MIRS)

Timeframe: Baseline through study completion (an average of 1 year)

The modified Medical Research Council (MRC) scale

Timeframe: Baseline through study completion (an average of 1 year)

Trial details

NCT IDNCT06979024

SponsorFirst Affiliated Hospital of Fujian Medical University

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2038-12

View on ClinicalTrials.gov More Myotonic Dystrophy Type 1 (DM1) trials

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.