Observational Prolonged Trial in Myotonic Dystrophy Type 1

Summary

Myotonic dystrophy type1 (DM1) is a rare, inherited, chronic progressive disease as well as an autosomal dominant multisystemic disorder. It is the most common adult form of muscular dystrophy, with a prevalence of approximately 10 per 100,000 people affected. With 733 million people in Europe, we estimate that 75,000 people are DM1 patients in Europe.

The aim of OPTIMISTIC is to improve clinical practice in the management of patients with this rare disease for which no dedicated treatment is currently available. OPTIMISTIC is a multi-centre, randomised controlled trial designed to compare a two component tailored behavioural change intervention to increase physical activity against standard patient management regimes, with particular attention given to the definition of appropriate outcome measures and new clinical guidelines for DM1 management. The two components of the intervention are 1) cognitive behavioural therapy (CBT) and 2) graded physical activity and we will evaluate the intervention's effectiveness and safety against standard patient management.

Participants will be recruited from myotonic dystrophy clinics and neuromuscular centres in France, Germany, the Netherlands and the UK. A total of 286 male and female patients aged 18 years and older with genetically proven classical or adult DM1 suffering from severe fatigue (only DM1 patients with a CIS subscale fatigue score \> 35 are likely to benefit from the intervention), able to walk independently and able to complete the trial interventions will be included.

A key objective of OPTIMISTIC is to provide outcome measures that are relevant for the patients and have a rate of change that is appropriate for a clinical trial timeframe. In addition, OPTIMISTIC will identify genetic factors that predict outcome and potential biomarkers as surrogate outcome measures that best explain the observed clinical variation.

Conditions

• Myotonic Dystrophy Type 1

Interventions

BEHAVIORAL

Behavioural change intervention

The intervention is cognitive behaviour therapy (CBT). The CBT consists of six different modules. All patients will start with individual goal setting and psycho-education about the role of cognitive-behavioural variables in the disabilities patients' experience. The patient formulates his or her treatment goals in concrete terms and later on in the therapy the goals are realised step by step by the patient. The treatment is tailored to the patient's problems: which of the six modules a patient will receive is dependent on the scores on measures that have been collected at baseline assessment. Based on our previous experience with modular interventions we expect that most patients will receive less than four modules.

Sponsors & Collaborators

• University of Newcastle Upon-Tyne

  collaborator OTHER

• Ludwig-Maximilians - University of Munich

  collaborator OTHER

• Assistance Publique - Hôpitaux de Paris

  collaborator OTHER

• Radboud University Medical Center

  lead OTHER

Principal Investigators

• Grainne Gorman, Dr · Newcastle University

• Baziel van Engelen, Prof · Radboud University Nijmegen Medical Centre, The Netherlands

• Benedikt Schoser, Prof · Munich University, Germany

• Guillaume Bassez, Prof · Assistance Publique-Hospitaux de Paris, France

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2014-04-02

Primary Completion

2016-03-29

Completion

2016-10-17

Countries

• France
• Germany
• Netherlands
• United Kingdom

Study Locations