NCT02118779: OPTIMISTIC

Observational Prolonged Trial in Myotonic Dystrophy Type 1

Completed NA Last updated 18 July 2017

What this trial tests

NA trial testing Behavioural change intervention in Myotonic Dystrophy Type 1 in 255 participants. Completed in 17 October 2016.

Timeline

2 April 2014

Primary endpoint
29 March 2016

17 October 2016

Quick facts

Lead sponsor	Radboud University Medical Center
Phase	NA
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	single
Primary purpose	treatment
Enrollment	255
Start date	2 April 2014
Primary completion	29 March 2016
Estimated completion	17 October 2016
Sites	4 locations across France, Netherlands, Germany, United Kingdom

Drugs / interventions tested

• Behavioural change intervention

Conditions studied

• Myotonic Dystrophy Type 1 — all drugs for Myotonic Dystrophy Type 1 →

Sponsor

Radboud University Medical Center

Who can join

18 and older, any sex, with Myotonic Dystrophy Type 1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Treating pediatric neuromuscular disorders: The future is now.
   Dowling JJ, D Gonorazky H, Cohn RD, Campbell C. · · 2018 · cited 83× · PMID 28889642 · DOI 10.1002/ajmg.a.38418
2. Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial.
   Okkersen K, Jimenez-Moreno C, Wenninger S, Daidj F, et al · · 2018 · cited 79× · PMID 29934199 · DOI 10.1016/s1474-4422(18)30203-5
3. Genetic determinants of disease severity in the myotonic dystrophy type 1 OPTIMISTIC cohort.
   Cumming SA, Jimenez-Moreno C, Okkersen K, Wenninger S, et al · · 2019 · cited 68× · PMID 31395669 · DOI 10.1212/wnl.0000000000008056
4. 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.
   van Engelen B, OPTIMISTIC Consortium. · · 2015 · cited 40× · PMID 26002596 · DOI 10.1186/s13063-015-0737-7
5. Lower extremity muscle pathology in myotonic dystrophy type 1 assessed by quantitative MRI.
   Heskamp L, van Nimwegen M, Ploegmakers MJ, Bassez G, et al · · 2019 · cited 31× · PMID 31118244 · DOI 10.1212/wnl.0000000000007648
6. Measuring Habitual Physical Activity in Neuromuscular Disorders: A Systematic Review.
   Jimenez-Moreno AC, Newman J, Charman SJ, Catt M, et al · · 2017 · cited 22× · PMID 28269791 · DOI 10.3233/jnd-160195
7. The UK Myotonic Dystrophy Patient Registry: facilitating and accelerating clinical research.
   Wood L, Cordts I, Atalaia A, Marini-Bettolo C, et al · · 2017 · cited 21× · PMID 28397002 · DOI 10.1007/s00415-017-8483-2
8. Associations Between Variant Repeat Interruptions and Clinical Outcomes in Myotonic Dystrophy Type 1.
   Wenninger S, Cumming SA, Gutschmidt K, Okkersen K, et al · · 2021 · cited 18× · PMID 33884298 · DOI 10.1212/nxg.0000000000000572