NCT02118779

Brief 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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
255

participants targeted

Target at P75+ for not_applicable

Timeline
Completed

Started Apr 2014

Typical duration for not_applicable

Geographic Reach
4 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 2, 2014

Completed
9 days until next milestone

First Submitted

Initial submission to the registry

April 11, 2014

Completed
10 days until next milestone

First Posted

Study publicly available on registry

April 21, 2014

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 29, 2016

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 17, 2016

Completed
Last Updated

July 18, 2017

Status Verified

July 1, 2017

Enrollment Period

2 years

First QC Date

April 11, 2014

Last Update Submit

July 13, 2017

Conditions

Myotonic Dystrophy Type 1

Keywords

Myotonic Dystrophy Type 1DM1Physical ExerciseCognitive Behavioural TherapyBiomarkers

Outcome Measures

Primary Outcomes (1)

  • DM1-Activ

    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.

    Baseline and 10 months

Secondary Outcomes (9)

  • Six Minute Walk Test

    Baseline and 10 months

  • Myotonic Dystrophy Health Index (MDHI)

    Baseline and 10 months

  • Physical activity measured with actometer

    Baseline and 10 months

  • Fatigue and Daytime Sleepiness Scale (FDSS)

    Baseline and 10 months

  • Checklist Individual Strength (CIS)

    Baseline and 10 months

  • +4 more secondary outcomes

Other Outcomes (1)

  • Explanatory and/or Predictive outcomes

    Baseline, 10 and 16 months

Study Arms (2)

Behavioural change intervention

EXPERIMENTAL

Cognitive behavioural therapy (CBT) combined with exercise

Behavioral: Behavioural change intervention

Standard Patient Management

NO INTERVENTION

Standard care like usual (i.e. annual checks with neurologist, checks with cardiologist, if needed physical therapy)

Interventions

Behavioural change interventionBEHAVIORAL

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.

Behavioural change intervention

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Assistance Publique-Hospitaux de Paris

Paris, France

Location

Friedrich Naur Institute

Munich, Germany

Location

Radboud University Nijmegen Medical Centre

Nijmegen, Netherlands

Location

Newcastle University

Newcastle, NE1 3BZ, United Kingdom

Location

Related Publications (4)

  • van Engelen B; OPTIMISTIC Consortium. Cognitive behaviour therapy plus aerobic exercise training to increase activity in patients with myotonic dystrophy type 1 (DM1) compared to usual care (OPTIMISTIC): study protocol for randomised controlled trial. Trials. 2015 May 23;16:224. doi: 10.1186/s13063-015-0737-7.

    PMID: 26002596BACKGROUND

  • Wenninger S, Cumming SA, Gutschmidt K, Okkersen K, Jimenez-Moreno AC, Daidj F, Lochmuller H, Hogarth F, Knoop H, Bassez G, Monckton DG, van Engelen BGM, Schoser B. Associations Between Variant Repeat Interruptions and Clinical Outcomes in Myotonic Dystrophy Type 1. Neurol Genet. 2021 Mar 9;7(2):e572. doi: 10.1212/NXG.0000000000000572. eCollection 2021 Apr.

    PMID: 33884298DERIVED

  • Heskamp L, van Nimwegen M, Ploegmakers MJ, Bassez G, Deux JF, Cumming SA, Monckton DG, van Engelen BGM, Heerschap A. Lower extremity muscle pathology in myotonic dystrophy type 1 assessed by quantitative MRI. Neurology. 2019 Jun 11;92(24):e2803-e2814. doi: 10.1212/WNL.0000000000007648. Epub 2019 May 22.

    PMID: 31118244DERIVED

  • Okkersen K, Jimenez-Moreno C, Wenninger S, Daidj F, Glennon J, Cumming S, Littleford R, Monckton DG, Lochmuller H, Catt M, Faber CG, Hapca A, Donnan PT, Gorman G, Bassez G, Schoser B, Knoop H, Treweek S, van Engelen BGM; OPTIMISTIC consortium. Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial. Lancet Neurol. 2018 Aug;17(8):671-680. doi: 10.1016/S1474-4422(18)30203-5. Epub 2018 Jun 19.

    PMID: 29934199DERIVED

Related Links

MeSH Terms

Conditions

Myotonic DystrophyMotor Activity

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesMyotonic DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesBehavior

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Coordinator Trialoffice Neurology

Study Record Dates

First Submitted

April 11, 2014

First Posted

April 21, 2014

Study Start

April 2, 2014

Primary Completion

March 29, 2016

Study Completion

October 17, 2016

Last Updated

July 18, 2017

Record last verified: 2017-07

Locations

NL(1)GB(1)DE(1)FR(1)