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| ID | Type | Description | Link |
|---|---|---|---|
| AFM-12117 | Other Grant/Funding Number | AFM-12117 |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm 1: Methylphenidate versus baseline | Placebo Comparator |
| |
| Arm 2: Placebo versus baseline | Placebo Comparator | One table placebo per day during 3 week |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Methylphenidate | Drug | One Tablet of methylphenidate, 20 mg per day during 3 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline of excessive daytime sleepiness | 3 weeks after treatment |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jack J Puymirat, MD | University Laval | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Institute of Readaptation in Physical Deficiency | Québec | Quebec | G1M2S8 | Canada |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39555632 | Derived | Annane D, Laberge L, Gallais B, Chevret S. Psychostimulants for hypersomnia (excessive daytime sleepiness) in myotonic dystrophy. Cochrane Database Syst Rev. 2024 Nov 18;11(11):CD003218. doi: 10.1002/14651858.CD003218.pub3. | |
| 22578232 | Derived | 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. |
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| ID | Term |
|---|---|
| D009223 | Myotonic Dystrophy |
| D006970 | Disorders of Excessive Somnolence |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| D008774 | Methylphenidate |
| ID | Term |
|---|---|
| D010648 | Phenylacetates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
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| Placebo | Drug | one tablet placebo per day during 3 weeks |
|
| D020967 | Myotonic Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020919 | Sleep Disorders, Intrinsic |
| D020920 | Dyssomnias |
| D012893 | Sleep Wake Disorders |
| D001523 | Mental Disorders |
| D010880 |
| Piperidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |