Methylphenidate in Myotonic Dystrophy Type 1

Laval University

Status and phase: Completed; Phase 3; Phase 2

Conditions: Dystrophia Myotonica 1

Treatments: Drug: Methylphenidate; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT01421992

AFM-12117 (Other Grant/Funding Number)

Details and patient eligibility

About

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).

Full 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.

Enrollment: 28 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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28 participants in 2 patient groups, including a placebo group

Arm 1: Methylphenidate versus baseline

Placebo Comparator group

Treatment:

Drug: Methylphenidate

Arm 2: Placebo versus baseline

Placebo Comparator group

Description:

One table placebo per day during 3 week

Treatment:

Drug: Placebo