Methylphenidate in Myotonic Dystrophy Type 1

Dystrophia Myotonica 1 Clinical Trial

Official title:

Phase 2/3 Study of Efficacy and Tolerability of Methylphenidate in the Treatment of Excessive Daytime Sleepiness in Myotonic Dystrophy Type 1

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01421992
• Other study ID #: AFM-12117
• Secondary ID: AFM-12117
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: June 1, 2011
• Last updated: August 22, 2011
• Start date: June 2008
• Est. completion date: September 2010

Study information

• Verified date: June 2011
• Source: Laval University
• Contact: n/a
• Is FDA regulated: No
• Health authority: Canada: Ethics Review Committee
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 28
• Est. completion date: September 2010
• Est. primary completion date: August 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Dystrophia Myotonica 1
• Myotonic Dystrophy

Intervention

Drug:
• Methylphenidate
One Tablet of methylphenidate, 20 mg per day during 3 weeks
• Placebo
one tablet placebo per day during 3 weeks

Locations

Country	Name	City	State
Canada	Institute of Readaptation in Physical Deficiency	Quebec

Sponsors (1)

Lead Sponsor	Collaborator
Laval University

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline of excessive daytime sleepiness		3 weeks after treatment	No
Secondary	Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency	Mean sleep latency was measured using the behavioural Osler's test	3 weeks after treatment	No