Phase 2/3 Study of Efficacy and Tolerability of Methylphenidate in the Treatment of Excessive Daytime Sleepiness in Myotonic Dystrophy Type 1
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Laval University
- Enrollment
- 28
- Locations
- 1
- Primary Endpoint
- Change from baseline of excessive daytime sleepiness
Study Overview
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).
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Crossover
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 65 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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
Arms & Interventions
Arm 1: Methylphenidate versus baseline
Intervention: Methylphenidate (Drug)
Arm 2: Placebo versus baseline
One table placebo per day during 3 week
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Change from baseline of excessive daytime sleepiness
Time Frame: 3 weeks after treatment
Secondary Outcomes
- Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency(3 weeks after treatment)