Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft (NCT02878694) | Clinical Trial Compass
TerminatedPhase 2/3
Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft
Stopped: insufficient recruitment
France1 participantsStarted 2019-11-14
Plain-language 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 .
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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
Exclusion Criteria:
* 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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.