Myotonic DystrophyPhase 2/3NCT00167609
Efficacy and Safety of DHEA for Myotonic Dystrophy
A Phase 2/3 study testing dehydroepiandrosterone 100 and 400 mg in people with Myotonic Dystrophy, run by University of Versailles at 10 sites in France.
Trial summary
To test the efficacy and safety of two doses of dehydroepiandrosterone (DHEA) in adults with myotonic dystrophy Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myotonic Dystrophy
- What is being tested?
- dehydroepiandrosterone 100 and 400 mg
- How many people
- 75 participants
- Where
- 10 sitesFrance
- Study dates
- Nov 2004to Dec 2006
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Myotonic dystrophy (genetically proven)
- MDRS score of 3 or 4
- Severe cardiac disease: acute myocardial infarction in the preceding 6 months, unstable heart failure, uncontrolled hypertension (systolic blood pressure >180 mmHg or diastolic blood pressure >100 mmHg after 10 minutes of rest in the lying position), severe arteritis, any past history of thrombose or embolic event, any past history of symptomatic arrhythmia)
- Chronic renal failure
- Chronic liver disease
- Long term mechanical ventilation
Show all 10Show fewer
- Any ongoing cancer
- Any underlying endocrine disorders
- Impaired swallowing
- Previous treatment with DHEA
Who cannot join
- Age <18 years or >70 years
- Pregnancy or breastfeeding
- Poor compliance to treatment and follow up
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
France10 sites
CHU
Angers
CHU
Bordeaux
Raymond Poincaré Hospital
Garches, 92380
CHU
Grenoble
CHU
Lyon
CHU Pouget
Marseille
CHU
Nice
Institut de myologie
Paris
CHU
Strasbourg
CHU
Toulouse
About this study
Official title: Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy
To test the efficacy and safety of two doses of dehydroepiandrosterone (DHEA) in adults with myotonic dystrophy
Read the detailed description
Myotonic dystrophy is an inherited disorder that affects 1 per 8000 adults. The disease is characterize by muscular dystrophy, myotonia, cardiac disorders, cognitive function impairment, hypersomnia, hair loss, endocrine disorders. Recent small studies suggested that DHEA treatment may improve muscle strength in adults with myotonic dystrophy. Thus, the current study aims at investigating the safety and efficacy of a prolonged treatment with DHEA in adults with myotonic dystrophy.
- Conditions
- Myotonic Dystrophy
- Being tested
- dehydroepiandrosterone 100 and 400 mg
- Keywords
- myotonic dystrophy, hypersomnia, dehydroepiandrosterone
- 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.
dehydroepiandrosterone 100 and 400 mgDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Variation in a Muscle Strength Score between randomization and study week 12
Measured: 3 months
Secondary outcomes (7)
evaluation of myotonia
Measured: 3 months
Appeal score
Measured: 3 months
Epworth score
Measured: 3 months
Forced vital capacity
Measured: 3 months
arterial blood gas
Measured: 3 months
changes in EKG and echocardiography
Measured: 3 months
tolerance
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2005 (estimated)
- Study startNov 2004
- Study completionDec 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myotonic DystrophyStudies 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
- Parallel groups
- Masking
- Double blind
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Versailles
- Sponsor type
- University, hospital or other
- Collaborators
- Association Française contre les Myopathies (AFM), Paris, Assistance Publique - Hôpitaux de Paris
- Investigators
- Djillali annane, MD, PhD, Study chair, Assistance Publique Hôpitaux de Paris - University of Versaillesmartine devillers, MD, Study director, AFM
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00167609
- Sponsor's protocol ID
- P001108
- Other IDs
- PS001108
- Status last verified
- Apr 2010
- First submitted
- 10 Sep 2005
- Last updated
- 6 Apr 2010
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Penisson-Besnier I, Devillers M, Porcher R, Orlikowski D, Doppler V, Desnuelle C, Ferrer X, Bes MC, Bouhour F, Tranchant C, Lagrange E, Vershueren A, Uzenot D, Cintas P, Sole G, Hogrel JY, Laforet P, Vial C, Vila AL, Sacconi S, Pouget J, Eymard B, Chevret S, Annane D. Dehydroepiandrosterone for myotonic dystrophy type 1. Neurology. 2008 Aug 5;71(6):407-12. doi: 10.1212/01.wnl.0000324257.35759.40.
PubMed 18678823
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy
- ClinicalTrials.gov ID
- NCT00167609
- Acronym / sponsor ID
- P001108
- Conditions
- Myotonic Dystrophy
- Keywords
- myotonic dystrophy, hypersomnia, dehydroepiandrosterone
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- University of VersaillesUniversity, hospital or other
- Collaborators
- Association Française contre les Myopathies (AFM), Paris, Assistance Publique - Hôpitaux de Paris
- Study start
- Nov 2004
- Study completion
- Dec 2006 (actual)
- First posted
- 14 Sep 2005 (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.
To test the efficacy and safety of two doses of dehydroepiandrosterone (DHEA) in adults with myotonic dystrophy
Detailed description
Myotonic dystrophy is an inherited disorder that affects 1 per 8000 adults. The disease is characterize by muscular dystrophy, myotonia, cardiac disorders, cognitive function impairment, hypersomnia, hair loss, endocrine disorders. Recent small studies suggested that DHEA treatment may improve muscle strength in adults with myotonic dystrophy. Thus, the current study aims at investigating the safety and efficacy of a prolonged treatment with DHEA in adults with myotonic dystrophy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Myotonic dystrophy (genetically proven)
- MDRS score of 3 or 4
- Severe cardiac disease: acute myocardial infarction in the preceding 6 months, unstable heart failure, uncontrolled hypertension (systolic blood pressure >180 mmHg or diastolic blood pressure >100 mmHg after 10 minutes of rest in the lying position), severe arteritis, any past history of thrombose or embolic event, any past history of symptomatic arrhythmia)
- Chronic renal failure
- Chronic liver disease
Key exclusion criteria (3 listed)
- Age <18 years or >70 years
- Pregnancy or breastfeeding
- Poor compliance to treatment and follow up
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 |
|---|---|---|
| dehydroepiandrosterone 100 and 400 mg | Drug | – |
Primary outcome
Variation in a Muscle Strength Score between randomization and study week 12
Time frame: 3 months
7 secondary outcomes
evaluation of myotonia
Time frame: 3 months
Appeal score
Time frame: 3 months
Epworth score
Time frame: 3 months
Forced vital capacity
Time frame: 3 months
arterial blood gas
Time frame: 3 months
changes in EKG and echocardiography
Time frame: 3 months
tolerance
Time frame: 3 months
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 6 Apr 2010.
- Results publication: PubMed 18678823Penisson-Besnier I, Devillers M, Porcher R, Orlikowski D, Doppler V, Desnuelle C, Ferrer X, Bes MC, Bouhour F, Tranchant C, Lagrange E, Vershueren A, Uzenot D, Cintas P, Sole G, Hogrel JY, Laforet P, Vial C, Vila AL, Sacconi S, Pouget J, Eymard B, Chevret S, Annane D. Dehydroepiandrosterone for myotonic dystrophy type 1. Neurology. 2008 Aug 5;71(6):407-12. doi: 10.1212/01.wnl.0000324257.35759.40.
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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.
Djillali annane, MD, PhD study chair
Assistance Publique Hôpitaux de Paris - University of Versailles
martine devillers, MD study director
AFM
10 sites 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 dehydroepiandrosterone 100 and 400 mg in people with Myotonic Dystrophy, run by University of Versailles at 10 sites in France.
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 10 inclusion and 3 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 6 Apr 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in France. 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. 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?
Variation in a Muscle Strength Score between randomization and study week 12 (time frame: 3 months). 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 NCT00167609, which also lists 1 linked publication.
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 6 Apr 2010.