Prospective, long-term, observational study (patient registry) of paediatric myotonic disorders from birth to less than six years of age who are treated with mexiletine (PEGASUS Study)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/1000000185
• EU PAS number:
  EUPAS1000000185
• Study ID:
  1000000185
• Official title and acronym:
  Prospective, long-term, observational study (patient registry) of paediatric myotonic disorders from birth to less than six years of age who are treated with mexiletine (PEGASUS Study)
• DARWIN EU® study:
  No
• Study countries:
  France

  Italy
• Study description:
  This is a prospective, open-label, multi-centre, single arm, registry study to collect standard relevant clinical and epidemiological data during routine medical evaluation and treatment in paediatric patients with myotonic disorders who are being treated with mexiletine therapy according to the physician.
• Study status:
  Planned

Research institutions and networks

Institutions

Lupin Europe GmbH

Study timelines

Date when funding contract was signed

Planned: 05/01/2024

Study start date

Planned: 23/09/2024

Date of final study report

Planned: 05/01/2028

Sources of funding

Pharmaceutical company and other private sector 

Study protocol

Initial protocol

Namuscla_PIP_Study_5_MEX-NM-401R_V1.0_31May2024_clean.docx_.pdf

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)

Other study registration identification numbers and links

MEX-NM-401