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Observational Prolonged Trial in Myotonic Dystrophy Type 1 (OPTIMISTIC)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02118779
First Posted: April 21, 2014
Last Update Posted: July 18, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborators:
University of Newcastle Upon-Tyne
Ludwig-Maximilians - University of Munich
Assistance Publique - Hôpitaux de Paris
Information provided by (Responsible Party):
Annet Geerlings, Radboud University
  Purpose

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.


Condition Intervention
Myotonic Dystrophy Type 1 Behavioral: Behavioural change intervention

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Observational Prolonged Trial in Myotonic Dystrophy Type 1 to Improve Quality of Life Standards, a Target Identification Collaboration

Resource links provided by NLM:


Further study details as provided by Annet Geerlings, Radboud University:

Primary Outcome Measures:
  • DM1-Activ [ Time Frame: Baseline and 10 months ]
    The primary outcome measure will be the change in DM1-Activ score. DM1-Activ is a specific outcome measure of activity and participation for patients with DM1.


Secondary Outcome Measures:
  • Six Minute Walk Test [ Time Frame: Baseline and 10 months ]
    Six minute walk test with BORG scale assessment

  • Myotonic Dystrophy Health Index (MDHI) [ Time Frame: Baseline and 10 months ]
  • Physical activity measured with actometer [ Time Frame: Baseline and 10 months ]
  • Fatigue and Daytime Sleepiness Scale (FDSS) [ Time Frame: Baseline and 10 months ]
  • Checklist Individual Strength (CIS) [ Time Frame: Baseline and 10 months ]
  • Individualised Neuromuscular Quality of Life Questionnaire (InQoL) [ Time Frame: Baseline and 10 months ]
  • Beck depression Inventory for Primary Care [ Time Frame: Baseline and 10 months ]
  • Apathy Evaluation Scale (AES) [ Time Frame: Baseline and 10 months ]
  • Stroop Test [ Time Frame: Baseline and 10 months ]

Other Outcome Measures:
  • Explanatory and/or Predictive outcomes [ Time Frame: Baseline, 10 and 16 months ]
    Biomarkers (urine and blood)


Enrollment: 255
Actual Study Start Date: April 2, 2014
Study Completion Date: October 17, 2016
Primary Completion Date: March 29, 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Behavioural change intervention
Cognitive behavioural therapy (CBT) combined with exercise
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.
No Intervention: Standard Patient Management
Standard care like usual (i.e. annual checks with neurologist, checks with cardiologist, if needed physical therapy)

  Show Detailed Description

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Able to provide informed consent
  • Genetically proven DM1
  • Suffering from severe fatigue (CIS fatigue >35
  • Able to walk independently

Exclusion Criteria:

  • Neurological or orthopaedic co-morbidity interfering with the interventions or possibly influencing outcomes.
  • Use of psychotropic drugs (except Modafinil, Ritalin and antidepressants where the dosing regimen has been stable for at least 12 months prior to screening). If the doses of Modafinil or Ritalin increase during the 10 months of the intervention then the participant will be excluded.
  • Severe depression as screening (judged as meeting DSM-IV criteria for a depressive episode).
  • Participation in another clinical trial of an investigational medicinal product (CTIMP) or other interventional study considered to influence outcomes being evaluated in OPTIMISTIC.
  • Unable to complete study questionnaires.
  • Subject participating in another clinical trial (other than observational trials and registries) concurrently or within 30 days prior to screening for entry into this study.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02118779


Locations
France
Assistance Publique-Hospitaux de Paris
Paris, France
Germany
Friedrich Naur Institute
Munich, Germany
Netherlands
Radboud University Nijmegen Medical Centre
Nijmegen, Netherlands
United Kingdom
Newcastle University
Newcastle, United Kingdom, NE1 3BZ
Sponsors and Collaborators
Radboud University
University of Newcastle Upon-Tyne
Ludwig-Maximilians - University of Munich
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Grainne Gorman, Dr Newcastle University
Principal Investigator: Baziel van Engelen, Prof Radboud University Nijmegen Medical Centre, The Netherlands
Principal Investigator: Benedikt Schoser, Prof Munich University, Germany
Principal Investigator: Guillaume Bassez, Prof Assistance Publique-Hospitaux de Paris, France
  More Information

Additional Information:
Publications:
Responsible Party: Annet Geerlings, Coordinator Trialoffice Neurology, Radboud University
ClinicalTrials.gov Identifier: NCT02118779     History of Changes
Other Study ID Numbers: 13/NE/0342
305697 ( Other Grant/Funding Number: EU Seventh Framework Programme )
6836 ( Other Identifier: Sponsor ref )
First Submitted: April 11, 2014
First Posted: April 21, 2014
Last Update Posted: July 18, 2017
Last Verified: July 2017

Keywords provided by Annet Geerlings, Radboud University:
Myotonic Dystrophy Type 1
DM1
Physical Exercise
Cognitive Behavioural Therapy
Biomarkers

Additional relevant MeSH terms:
Myotonic Dystrophy
Muscular Dystrophies
Myotonic Disorders
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn