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

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).

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.

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