Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft (TTT-PT-DOP)
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|ClinicalTrials.gov Identifier: NCT02878694|
Recruitment Status : Unknown
Verified August 2016 by University Hospital, Caen.
Recruitment status was: Active, not recruiting
First Posted : August 25, 2016
Last Update Posted : August 25, 2016
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 .
|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophy, Oculopharyngeal Ptosis||Biological: Myoblast autologous graft||Phase 2 Phase 3|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft|
|Study Start Date :||July 2016|
|Estimated Primary Completion Date :||April 2018|
|Estimated Study Completion Date :||December 2018|
Experimental: Myoblast autologous graft
30 million autologous myoblasts in 6 intramuscular injections
Biological: Myoblast autologous graft
30 million autologous myoblasts in 6 intramuscular injections No treatment in the second eye
- Improvement of ptosis [ Time Frame: baseline and 12 months ]The improvement in ptosis is considered by measuring the opening of the lid gap ( OFP) , the gaze straight ahead and no inclination of the head, the eye untreated and treated before and after treatment at 12 month.
- 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 .
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02878694
|Caen, France, 14000|
|Study Chair:||Françoise CF Chapon, PhD||CHU Caen|
|Study Chair:||Olivier BO Boyer, PhD||CHU Rouen|
|Study Chair:||Frederic MF Mouriaux, PhD||CHU Rennes|
|Study Chair:||Sophie PS Perie, PhD||APHP|