Study identification

EU PAS number

EUPAS37943

Study ID

46833

Official title and acronym

An Observational Study to Describe the Long-term Safety and Effectiveness of Namuscla in the Symptomatic Management of Myotonia in Adult Patients with Non-dystrophic Myotonic Disorders

DARWIN EU® study

No

Study countries

France
Germany
United Kingdom

Study description

Namuscla™ is approved in European Union (EU) for the symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders. Active ingredient of Namuscla is 167 mg mexiletine, a class Ib antiarrhythmic. To date, randomised studies conducted for mexiletine have assessed only short-term efficacy and safety with little supporting data for long-term use from observational research. This non-interventional study will collect data on the long-term (12 months to 3 years) safety of Namuscla in a real-world setting for the symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders over a period of up to 3 years. This study is being conducted as part of the agreed European Risk Management Plan (RMP).

Study status

Ongoing
Research institutions and networks

Institutions

University Hospital of Ulm
Germany
First published:
01/02/2024
Institution Educational Institution Hospital/Clinic/Other health care facility
Hôpital Universitaire de La Pitié Salpêtrière 47-83 bd de l’hôpital Batiment Babinski Paris, Cedex 13 75013, CHRU Lille 2 Avenue Oscar Lambret Lille, 59000, St. Josef-Hospital Klinikum der Ruhr Universitaet Bochum Gudrunstraße 56 44791 Bochum Bochum, North-Rhine Westphalia 44791, Universitätsklinikum Ulm, Klinik für Neurologie Oberer Eselsberg 45 Ulm, 89081, Nottingham University Hospitals NHS Trust Queen’s Medical Centre, South Block, Derby Road, Nottingham, England NG7 2UH, Institute of Neurology, Queen Square London, England WC1N 3BG

Contact details

Beatriz Borredá

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Data analysis start date

Planned:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Lupin EU GmbH
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 3 (required)