Efficacy and Safety of DHEA for Myotonic Dystrophy | NCT00167609

Studies
Efficacy and Safety of DHEA for Myotonic Dystrophy

NCT00167609

Sponsor

University of Versailles

Status

Completed

Last Update Posted

Apr 6, 2010Estimated

Enrollment

75Actual

Phase

Phase 2Phase 3

Conditions

Myotonic Dystrophy

Interventions

dehydroepiandrosterone 100 and 400 mg

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
PS001108

Status Module

Record Verification Date

Apr 2010

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 2004

Primary Completion Date

Not provided

Completion Date

Dec 2006Actual

First Submitted Date

Sep 10, 2005

First Submission Date that Met QC Criteria

Sep 10, 2005

First Posted Date

Sep 14, 2005Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 5, 2010

Last Update Posted Date

Apr 6, 2010Estimated

Sponsor/Collaborators Module

Responsible Party, by Official Title

Not provided

Lead Sponsor

University of VersaillesOTHER

Collaborators

Name	Class
Association Française contre les Myopathies (AFM), Paris	OTHER
Assistance Publique - Hôpitaux de Paris	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

75Actual

Name	Type	Description	Arm Group Labels	Other Names
dehydroepiandrosterone 100 and 400 mg	Drug

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Variation in a Muscle Strength Score between randomization and study week 12		3 months

Secondary Outcomes

Measure	Description	Time Frame
evaluation of myotonia		3 months
Appeal score		3 months
Epworth score

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Myotonic dystrophy (genetically proven)
MDRS score of 3 or 4

Exclusion Criteria:

Age <18 years or >70 years
Pregnancy or breastfeeding
Poor compliance to treatment and follow up
Inclusion in any other clinical trial
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
Any ongoing cancer
Any underlying endocrine disorders
Impaired swallowing
Previous treatment with DHEA

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

70 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Djillali annane, MD, PhD	Assistance Publique Hôpitaux de Paris - University of Versailles	Study Chair
martine devillers, MD	AFM	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
CHU		Angers			France
CHU

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
18678823	Result	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.

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