Methylphenidate in Myotonic Dystrophy Type 1
Phase 2/3 Study of Efficacy and Tolerability of Methylphenidate in the Treatment of Excessive Daytime Sleepiness in Myotonic Dystrophy Type 1
1 other identifier
interventional
28
1 country
1
Brief Summary
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jun 2008
1 active site
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
June 1, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2010
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2010
CompletedFirst Submitted
Initial submission to the registry
June 1, 2011
CompletedFirst Posted
Study publicly available on registry
August 23, 2011
CompletedAugust 23, 2011
June 1, 2011
2.2 years
June 1, 2011
August 22, 2011
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change from baseline of excessive daytime sleepiness
3 weeks after treatment
Secondary Outcomes (1)
Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency
3 weeks after treatment
Study Arms (2)
Arm 1: Methylphenidate versus baseline
PLACEBO COMPARATORArm 2: Placebo versus baseline
PLACEBO COMPARATOROne table placebo per day during 3 week
Interventions
One Tablet of methylphenidate, 20 mg per day during 3 weeks
Eligibility Criteria
You may qualify if:
- adults
- Epworth score ≥ 10
- Diagnosis of myotonic dystrophy type 1
You may not qualify if:
- hypersensibility to methylphenidate
- Pregnancy
- Patients who receive drugs that interfere with methylphenidate
- Cognitive impairment
- Sleep apnea
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Laval Universitylead
Study Sites (1)
Institute of Readaptation in Physical Deficiency
Québec, Quebec, G1M2S8, Canada
Related Publications (2)
Annane D, Laberge L, Gallais B, Chevret S. Psychostimulants for hypersomnia (excessive daytime sleepiness) in myotonic dystrophy. Cochrane Database Syst Rev. 2024 Nov 18;11(11):CD003218. doi: 10.1002/14651858.CD003218.pub3.
PMID: 39555632DERIVEDPuymirat J, Bouchard JP, Mathieu J. Efficacy and tolerability of a 20-mg dose of methylphenidate for the treatment of daytime sleepiness in adult patients with myotonic dystrophy type 1: a 2-center, randomized, double-blind, placebo-controlled, 3-week crossover trial. Clin Ther. 2012 May;34(5):1103-11. doi: 10.1016/j.clinthera.2012.03.060.
PMID: 22578232DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Jack J Puymirat, MD
University Laval
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 1, 2011
First Posted
August 23, 2011
Study Start
June 1, 2008
Primary Completion
August 1, 2010
Study Completion
September 1, 2010
Last Updated
August 23, 2011
Record last verified: 2011-06