Dystrophia Myotonica 1Phase 2/3NCT01421992
Methylphenidate in Myotonic Dystrophy Type 1
A Phase 2/3 study testing Methylphenidate in people with Dystrophia Myotonica 1, run by Laval University at 1 site in Canada.
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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Dystrophia Myotonica 1
- What is being tested?
- Methylphenidate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 28 participants
- Where
- 1 siteCanada
- Study dates
- Jun 2008to Sep 2010
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- adults
- Epworth score ≥ 10
- Diagnosis of myotonic dystrophy type 1
Who cannot join
- hypersensibility to methylphenidate
- Pregnancy
- Patients who receive drugs that interfere with methylphenidate
- Cognitive impairment
- Sleep apnea
Trial locations and contacts
1 site in 1 country.
Canada1 site
Institute of Readaptation in Physical Deficiency
Québec, Quebec, G1M2S8
About this study
Official title: 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).
Read the 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.
- Conditions
- Dystrophia Myotonica 1
- Being tested
- Methylphenidate, Placebo
- Keywords
- randomized, double-blind, crossover, methylphenidate, excessive daytime sleepiness
- Medical subject headings
- Myotonic Dystrophy, Disorders of Excessive Somnolence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MethylphenidateDrug
Also called Ritalin
One Tablet of methylphenidate, 20 mg per day during 3 weeks
PlaceboDrug
one tablet placebo per day during 3 weeks
| Study group | Role | What they receive |
|---|---|---|
| Arm 1: Methylphenidate versus baseline | Placebo comparator | Not described Receives: Drug: Methylphenidate |
| Arm 2: Placebo versus baseline | Placebo comparator | One table placebo per day during 3 week Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline of excessive daytime sleepiness
Measured: 3 weeks after treatment
Secondary outcomes (1)
Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency
Mean sleep latency was measured using the behavioural Osler's test
Measured: 3 weeks after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2011 (estimated)
- Study startJun 2008
- Primary completionAug 2010
- Study completionSep 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Dystrophia Myotonica 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Laval University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jack J Puymirat, MD, Principal investigator, University Laval
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01421992
- Sponsor's protocol ID
- AFM-12117
- Other IDs
- AFM-12117
- Status last verified
- Jun 2011
- First submitted
- 1 Jun 2011
- Last updated
- 23 Aug 2011
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 39555632Puymirat 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.
PubMed 22578232
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2/3 Study of Efficacy and Tolerability of Methylphenidate in the Treatment of Excessive Daytime Sleepiness in Myotonic Dystrophy Type 1
- ClinicalTrials.gov ID
- NCT01421992
- Acronym / sponsor ID
- AFM-12117
- Conditions
- Dystrophia Myotonica 1
- Keywords
- randomized, double-blind, crossover, methylphenidate, excessive daytime sleepiness
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Laval UniversityUniversity, hospital or other
- Study start
- Jun 2008
- Primary completion
- Aug 2010 (actual)
- Study completion
- Sep 2010 (actual)
- First posted
- 23 Aug 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1: Methylphenidate versus baseline | Placebo comparator | Drug: Methylphenidate |
| Arm 2: Placebo versus baseline | Placebo comparator | Drug: PlaceboOne table placebo per day during 3 week |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- adults
- Epworth score ≥ 10
- Diagnosis of myotonic dystrophy type 1
Key exclusion criteria (5 listed)
- hypersensibility to methylphenidate
- Pregnancy
- Patients who receive drugs that interfere with methylphenidate
- Cognitive impairment
- Sleep apnea
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MethylphenidateAlso: RitalinOne Tablet of methylphenidate, 20 mg per day during 3 weeks | Drug | Arm 1: Methylphenidate versus baseline |
| Placeboone tablet placebo per day during 3 weeks | Drug | Arm 2: Placebo versus baseline |
Primary outcome
Change from baseline of excessive daytime sleepiness
Time frame: 3 weeks after treatment
1 secondary outcome
Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency
Time frame: 3 weeks after treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2011.
- Derived from this study: PubMed 39555632Annane 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.
- Derived from this study: PubMed 22578232Puymirat 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jack J Puymirat, MD principal investigator
University Laval
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Methylphenidate in people with Dystrophia Myotonica 1, run by Laval University at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Aug 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline of excessive daytime sleepiness (time frame: 3 weeks after treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01421992, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Aug 2011.