Status:
COMPLETED
Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborating Sponsors:
Association Française contre les Myopathies (AFM), Paris
Conditions:
Muscular Dystrophy, Oculopharyngeal
Eligibility:
All Genders
18-75 years
Phase:
PHASE2
Brief Summary
The OCULO-Pharyngeal Muscular Dystrophy (OPMD) is a late onset hereditary muscle disease which is characterised by the selective affection of the pharyngeal muscles resulting in swallowing disorders, ...
Detailed Description
OCULO-Pharyngeal Muscular Dystrophy (OPMD) is characterised by the selective affection of the superior sphincter muscles of the oesophagus (SSO) and the pharyngeal.muscles resulting in dysphagia. The ...
Eligibility Criteria
Inclusion
- Man or woman \<18-75\> years old
- Oculopharyngeal muscular dystrophy confirmed by genetic diagnosis (mutation of the GCG) on the chromosome 14)
- Oculopharyngeal muscular dystrophy with UES dysfunction
- salivary or alimentary stasis at fibroscopy of swallowing above the UES,
- decreased opening of the UES at videofluoroscopy of swallowing A decreased of the pharyngeal propulsion may be associated Written consent of the patient
Exclusion
- History of myotomy of the UES in the context of the Oculopharyngeal muscular
- Dystrophy;
- HIV, hepatitis B or C tuberculosis);
- Lupus, rheumatoid polyarthritis, sarcoïdosis, collagenosis) ;
- Other neuromuscular diseases ;
- History of malignant tumor ;
- History of neck radiotherapy ;
- Renal failure (creatinine clearance \<60ml/min)
- Liver failure ;
- Pregnancy ;
- Follow up less than 24 months:
- Patients who refuse to sign the consent;
- No social security.
Key Trial Info
Start Date :
April 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2015
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT00773227
Start Date
April 1 2004
End Date
October 1 2015
Last Update
November 20 2015
Active Locations (1)
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1
Hospital Tenon
Paris, France, 75020