Methylphenidate in Myotonic Dystrophy Type 1

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Laval University
ClinicalTrials.gov Identifier:
NCT01421992
First received: June 1, 2011
Last updated: August 22, 2011
Last verified: June 2011
  Purpose

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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency [ Time Frame: 3 weeks after treatment ] [ Designated as safety issue: No ]
    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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 ClinicalTrials.gov identifier: NCT01421992

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

No publications provided by Laval University

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Laval University
ClinicalTrials.gov Identifier: NCT01421992     History of Changes
Other Study ID Numbers: AFM-12117, AFM-12117
Study First Received: June 1, 2011
Last Updated: August 22, 2011
Health Authority: Canada: Ethics Review Committee

Keywords provided by Laval University:
randomized
double-blind
crossover
methylphenidate
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
Methylphenidate
Dopamine Uptake Inhibitors
Dopamine Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Neurotransmitter Uptake Inhibitors
Physiological Effects of Drugs
Central Nervous System Stimulants
Central Nervous System Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on July 26, 2014