Lambert-Eaton Myasthenic Syndrome (LEMS) Registry (LEMS-01)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/39278
• EU PAS number:
  EUPAS6106
• Study ID:
  39278
• Official title and acronym:
  Lambert-Eaton Myasthenic Syndrome (LEMS) Registry (LEMS-01)
• DARWIN EU® study:
  No
• Study countries:
  France

  Germany

  Italy

  Spain

  United Kingdom
• Study description:
  Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder that affects voltage-gated calcium channels on the pre-synaptic membrane of the nerve-muscle (neuromuscular) junction. LEMS is estimated to affect 1 in 100,000 people in the European Community and is clinically characterized by weakness and frequent fatigue (mainly of the legs and trunk), and is associated with autonomic dysfunction (e.g. impotence, dry mouth, constipation). The onset of symptoms is usually gradual and insidious. The age at onset is typically ≥ 40 years in patients with cancer and between 20 and 50 years in those without. Slightly more men than women are affected. LEMS has also been reported in children, sometimes associated with neuroblastoma, but is an extremely rare condition in this patient group. In January 2010 the EU approved the Marketing Authorisation of 3, 4-DAP Phosphate (Amifamdripine, Firdapse®) for the treatment of LEMS. Firdapse is the only approved 3, 4-DAP compound for the treatment of LEMS. The purpose of the LEMS registry is to collect additional data on the long term safety and efficacy of Firdapse for patients who have been prescribed Firdapse by their treating physician. This registry will also increase knowledge about the course of disease in patients with LEMS by evaluating neurological and muscular function. The LEMS registry is a voluntary multi-centre, multinational, observational program for patients with LEMS disease, intended to track the routine clinical outcomes of patients with LEMS over time. As per condition of the EU Marketing Authorisation, the registry will also track the use of treatment for LEMS including drugs other than Firdapse. The data collected by the registry are intended to enable better characterisation of the natural history of LEMS.All patients with a confirmed diagnosis of LEMS disease may be eligible to participate in this program. No experimental treatments or assessments are involved in this program.
• Study status:
  Finalised

Research institution and networks

Institutions

BioMarin Pharmaceuticals

Multiple centres: 30 centres are involved in the study

Study timelines

Date when funding contract was signed

Planned: 01/10/2009

Actual: 09/01/2014

Study start date

Planned: 31/03/2010

Actual: 25/05/2010

Date of final study report

Planned: 28/06/2020

Actual: 21/02/2020

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

BioMarin Clinical Limted

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)