search
Back to results

Methylphenidate in Myotonic Dystrophy Type 1

Primary Purpose

Dystrophia Myotonica 1

Status
Completed
Phase
Phase 2
Locations
Canada
Study Type
Interventional
Intervention
Methylphenidate
Placebo
Sponsored by
Laval University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Dystrophia Myotonica 1 focused on measuring randomized, double-blind, crossover, methylphenidate, excessive daytime sleepiness

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Institute of Readaptation in Physical Deficiency

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Placebo Comparator

Arm Label

Arm 1: Methylphenidate versus baseline

Arm 2: Placebo versus baseline

Arm Description

One table placebo per day during 3 week

Outcomes

Primary Outcome Measures

Change from baseline of excessive daytime sleepiness

Secondary Outcome Measures

Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency
Mean sleep latency was measured using the behavioural Osler's test

Full Information

First Posted
June 1, 2011
Last Updated
August 22, 2011
Sponsor
Laval University
search

1. Study Identification

Unique Protocol Identification Number
NCT01421992
Brief Title
Methylphenidate in Myotonic Dystrophy Type 1
Official Title
Phase 2/3 Study of Efficacy and Tolerability of Methylphenidate in the Treatment of Excessive Daytime Sleepiness in Myotonic Dystrophy Type 1
Study Type
Interventional

2. Study Status

Record Verification Date
June 2011
Overall Recruitment Status
Completed
Study Start Date
June 2008 (undefined)
Primary Completion Date
August 2010 (Actual)
Study Completion Date
September 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Laval University

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dystrophia Myotonica 1
Keywords
randomized, double-blind, crossover, methylphenidate, excessive daytime sleepiness

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
28 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arm 1: Methylphenidate versus baseline
Arm Type
Placebo Comparator
Arm Title
Arm 2: Placebo versus baseline
Arm Type
Placebo Comparator
Arm Description
One table placebo per day during 3 week
Intervention Type
Drug
Intervention Name(s)
Methylphenidate
Other Intervention Name(s)
Ritalin
Intervention Description
One Tablet of methylphenidate, 20 mg per day during 3 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
one tablet placebo per day during 3 weeks
Primary Outcome Measure Information:
Title
Change from baseline of excessive daytime sleepiness
Time Frame
3 weeks after treatment
Secondary Outcome Measure Information:
Title
Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency
Description
Mean sleep latency was measured using the behavioural Osler's test
Time Frame
3 weeks after treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jack J Puymirat, MD
Organizational Affiliation
University Laval
Official's Role
Principal Investigator
Facility Information:
Facility Name
Institute of Readaptation in Physical Deficiency
City
Quebec
ZIP/Postal Code
G1M2S8
Country
Canada

12. IPD Sharing Statement

Citations:
PubMed Identifier
22578232
Citation
Puymirat 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.
Results Reference
derived

Learn more about this trial

Methylphenidate in Myotonic Dystrophy Type 1

We'll reach out to this number within 24 hrs