Can watching the course of myotonic dystrophy unlock better care?
NCT ID NCT07732439
First seen Jul 28, 2026 · Last updated Jul 29, 2026 · Updated 1 time
Summary
This study follows 100 adults with myotonic dystrophy (types 1 or 2) for two years to understand how muscle stiffness, daily function, and heart health change over time. Researchers will look back at up to 18 months of past medical records and then track participants with clinic visits at the start, 12 months, and 24 months. The goal is to map the natural course of the disease and identify which symptoms most affect quality of life.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this study could help doctors better predict how myotonic dystrophy progresses and identify which symptoms matter most to patients.
- What could go wrong
- This is an observational study, not a treatment trial, so it will not test any new therapy. The small number of participants (100) may limit how broadly the findings apply.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 100 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Aug 2026
An estimate. Start dates often move.
- Expected to finish
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Sep 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
All patients will be recruited from DM-Scope Registry
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Enrolled in DM-scope registry genetically diagnosed with DM1 or DM2. * Affiliation or beneficiary of a social security system or of such a regime. * Ability to comprehend and willingness to sign an informed consent (ICF). * Male or non-pregnant female ≥18 years of age at screening. * Body Mass Index (BMI) of 18.5 kg/m2 to 30 kg/m2, and weight ≥45 kg. * Medical history data covering up to 18 months prior to enrollment. * Clinical sign of myotonia * DM1 patients only - Muscular impairment rating scale (MIRS) score of 2, 3 or 4. * Be able to walk independently 10 meters (cane, walker, orthoses allowed). Exclusion Criteria: * No informed consent. * Pregnant or lactating women. * Subjects benefiting from laws aimed at protecting vulnerable adults: subjects being deprived of liberty by judicial or administrative decision, subjects under guardianship /curatorship. * Any medical condition or serious medical illness which in the opinion of the Investigator, precludes the participant's participation in the study or the participant is unlikely to comply with the protocol-defined procedures and therefore is unlikely to complete the study. * Medical conditions that could affect hand functioning including (but not limited to) rheumatoid arthritis, Dupuytren's contracture, hand deformity, severe arthritis or any other medical condition (other than DM1/DM2) that would significantly impact ambulation. * Patients with no documented record of myotonia assessment in the clinical records of the DM-scope database or myotonia absence at last visit prior to study enrolment. * Not able to perform study specific performance tests and evaluations e.g. hand grip dynamometry, 10mWT, etc. (in the opinion of the investigator). * Treatment with mexiletine within 18 months prior to baseline (Day 1).
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
6 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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CHU LA TIMONE - Service des Maladies
Marseille, 13005, France
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CHU de Lille - Hôpital
Lille, 59037, France
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CHU de Toulouse - Hôpital
Toulouse, 40031, France
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Centre de référence des maladies neuromusculaires
Nantes, 44093, France
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Centre hospitalier Universitaire d'Angers
Angers, 75651, France
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Hôpital Pitié Salpêtrière
Paris, 75013, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- 700-Patient study seeks key clues to muscle disease
- Brain scans reveal diabetes link to cognitive decline in rare disease
- New drug AOC 1001 tested for rare muscle disease
- Spanish researchers launch massive DM1 registry to unlock disease secrets