Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Improvement of ptosis
研究概览
简要总结
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 .
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female ( with suitable contraception) over 18 years of age 75
- •OPMD confirmed by genetic diagnosis ( gene mutation PABPN1 by triplet expansion GCG)
- •OPMD with ptosis
- •Obtaining informed consent signed
排除标准
- •Evolutionary contagious infectious pathology
- •Inflammatory diseases
- •Any other neuromuscular disease
- •Malignant tumor pathology of history
- •Renal impairment ( creatinine clearance < 60ml / min)
- •Hepatic insufficiency
- •Pregnant woman confirmed by a dosing B-HCG or lactating
- •Inability to perform a muscle biopsy
- •Preparation of myoblasts uncommitted to step 2nd release
- •Inability to follow up to 36 months
- •Refusal to sign the consent form
- •No Social Security
研究组 & 干预措施
Myoblast autologous graft
30 million autologous myoblasts in 6 intramuscular injections
干预措施: Myoblast autologous graft (Biological)
结局指标
主要结局
Improvement of ptosis
时间窗: 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.
次要结局
- fever(baseline)
- Tolerance of collection of the seat and transplantation: hematoma (yes / no )(baseline)
- Tolerance of the sampling procedure and transplantation : visual analogue scale Pain about the procedure(baseline)
- Goldmann Visual Field(12 months)
- Tolerance of the sampling procedure and transplantation, swelling (yes / no)(baseline)
- strength of the levator muscle of the upper eyelid(12 months)
- acuity(12 months)
- neck pain with Visual Analaogue Scale(12 months)