Clinical Trial: Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft
Brief Summary:
Interventional , multicenter , comparative study. One eye receiving the cells and the contralateral eye as a negative control . If effectiveness following review of the primary endpoint and the advice of an independent expert committee , the experimental treatment will be offered to the patient to the contralateral eyelid.
Objective is to restore muscle function levator muscle of the upper eyelid by providing a registry of autologous myoblasts from a non- clinically affected muscle .
Detailed Summary:
Muscular dystrophy oculopharyngeal ( OPMD ) is an autosomal dominant inherited myopathy manifested as selective involvement of the levator muscles of the upper eyelids , head of ptosis, and muscles of the intersection pharyngolaryngeal originally disorders swallowing. Ptosis can be treated with surgery ( shortening of the levator muscle ), but the disease continues to evolve , relapses occur , necessitating another intervention , which is the suspension of the eyelid to the frontal muscle that can cause corneal complications by imperfect lid closure .
Main objective is to restore muscle function levator muscle of the upper eyelid by providing a registry of autologous myoblasts from a non- clinically affected muscle .
A study on the same model was conducted in patients in ENT OPMD crossroads of muscles with encouraging results , 12/12 patients had improved quality of life and no degradation of swallowing was observed in 10 / 12 patients.
This is an interventional study , multicenter, comparative , one eye receiving the cells and the contralateral eye as a negative control . If effectiveness following review of the primary endpoint and the advice of an independent expert committee , the experimental treatment will be offered to the patient to the contralateral eyelid.
Seven centers are associated with this project as inclusion and assessment center. Patients are referred by different clinical sites in Caen ophthalmology department which will be performed pre-transplant assessment, the collection of donor myoblast muscle graft and part tracking.
The expected duration is 3 years (2 years of inclusion and evaluation of the primary endpoint at 1 year) for inclusion of 10 patients.<
Sponsor: University Hospital, Caen
Current Primary Outcome: Improvement of ptosis [ Time Frame: baseline and 12 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- acuity [ Time Frame: 12 months ]Measure of visual acuity ( without lifting the lid manually and without head tilt ) according Monoyer scale ,
- Goldmann Visual Field [ Time Frame: 12 months ]
- neck pain with Visual Analaogue Scale [ Time Frame: 12 months ]
- Tolerance of the sampling procedure and transplantation, swelling (yes / no) [ Time Frame: baseline ]
- Tolerance of the sampling procedure and transplantation : visual analogue scale Pain about the procedure [ Time Frame: baseline ]
- Tolerance of collection of the seat and transplantation: hematoma (yes / no ) [ Time Frame: baseline ]
- fever [ Time Frame: baseline ]
- strength of the levator muscle of the upper eyelid [ Time Frame: 12 months ]This is assessed by measuring the travel of the free edge of the upper eyelid between the eyes down and look up blocking the frontal muscle (photos and films) . Normal Range : 15 to 17 mm. Very impaired in patients with DOP .
Original Secondary Outcome: Same as current
Information By: University Hospital, Caen
Dates:
Date Received: March 8, 2016
Date Started: July 2016
Date Completion: December 2018
Last Updated: August 22, 2016
Last Verified: August 2016
