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NOT YET RECRUITING
NCT07732439

An Ambispective Natural History Study in Myotonic Dystrophy Patients Linking Retrospective Data Captured From the DM-Scope Registry With a Prospective 24-month Follow-up Period

Sponsor: Lupin Ltd.

View on ClinicalTrials.gov

Summary

This natural history observational study is being conducted to follow patients with DM1 or DM2 over a 2 year period to study the presence of myotonia, how it's perceived and its impact on patients quality of life. This study will be conducted at 6 study sites located in France.100 Patients will be recruited from the DM Scope Registry only. The study involves two parts. Part 1 will look back up to 18 months of past medical history that is already available from the DM Scope Registry. Part 2 will follow the same patients for 24 months, with study visits at Day 1 (Baseline), 12 months and 24 months. The goal is to better understand how myotonia symptoms and complications such as heart and other systemic problems develop and change over time. A smaller, sub-study will take place at one site, using new exploratory methods in about 40 patients with DM1 who are also part of the Track DM Study.

Official title: An Ambispective 24-Month Longitudinal Natural History Study in Myotonic Dystrophy Patients Using DM-Scope Registry (TRACK-DM Study)

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

OBSERVATIONAL

Enrollment

100

Start Date

2026-08-03

Completion Date

2029-09

Last Updated

2026-07-28

Healthy Volunteers

No

Locations (6)

Centre hospitalier Universitaire d'Angers

Angers, France

CHU de Lille - Hôpital

Lille, France

CHU LA TIMONE - Service des Maladies

Marseille, France

Centre de référence des maladies neuromusculaires

Nantes, France

Hôpital Pitié Salpêtrière

Paris, France

CHU de Toulouse - Hôpital

Toulouse, France