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Methylphenidate in Myotonic Dystrophy Type 1

This study has been completed.
Information provided by (Responsible Party):
Laval University Identifier:
First received: June 1, 2011
Last updated: August 22, 2011
Last verified: June 2011
The purpose of this study is to determine whether methylphenidate is effective in the treatment of excessive daytime sleepiness due to myotonic dystrophy type 1 (DM1).

Condition Intervention Phase
Dystrophia Myotonica 1 Drug: Methylphenidate Drug: Placebo Phase 2 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase 2/3 Study of Efficacy and Tolerability of Methylphenidate in the Treatment of Excessive Daytime Sleepiness in Myotonic Dystrophy Type 1

Resource links provided by NLM:

Further study details as provided by Laval University:

Primary Outcome Measures:
  • Change from baseline of excessive daytime sleepiness [ Time Frame: 3 weeks after treatment ]

Secondary Outcome Measures:
  • Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency [ Time Frame: 3 weeks after treatment ]
    Mean sleep latency was measured using the behavioural Osler's test

Enrollment: 28
Study Start Date: June 2008
Study Completion Date: September 2010
Primary Completion Date: August 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Arm 1: Methylphenidate versus baseline Drug: Methylphenidate
One Tablet of methylphenidate, 20 mg per day during 3 weeks
Other Name: Ritalin
Placebo Comparator: Arm 2: Placebo versus baseline
One table placebo per day during 3 week
Drug: Placebo
one tablet placebo per day during 3 weeks

Detailed Description:
Myotonic dystrophy type 1 (DM1) is a multisystemic disorder characterized by muscle weakness, myotonia, and the involvement of several systems. Hypersomnolence is one of the most frequently reported symptoms in patients with DM1 and often lead to handicap such as cessation of employment and withdrawal from social activities.The current investigation represents a prospective, double-blind, randomized, crossover, placebo-controlled study designed to evaluate the efficacy of methylphenidate for the treatment of excessive daytime sleepiness (EDS) in adults with DM1.

Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • adults
  • Epworth score ≥ 10
  • Diagnosis of myotonic dystrophy type 1

Exclusion Criteria:

  • hypersensibility to methylphenidate
  • Pregnancy
  • Patients who receive drugs that interfere with methylphenidate
  • Cognitive impairment
  • Sleep apnea
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01421992

Institute of Readaptation in Physical Deficiency
Quebec, Canada, G1M2S8
Sponsors and Collaborators
Laval University
Principal Investigator: Jack J Puymirat, MD University Laval
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Laval University Identifier: NCT01421992     History of Changes
Other Study ID Numbers: AFM-12117
AFM-12117 ( Other Grant/Funding Number: AFM-12117 )
Study First Received: June 1, 2011
Last Updated: August 22, 2011

Keywords provided by Laval University:
excessive daytime sleepiness

Additional relevant MeSH terms:
Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Central Nervous System Stimulants
Physiological Effects of Drugs
Dopamine Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Dopamine Agents
Neurotransmitter Agents processed this record on September 21, 2017