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 range18 Years – 75 Years

SexALL

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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

What they're measuring

Improvement of ptosis

Timeframe: baseline and 12 months

Trial details

NCT IDNCT02878694

SponsorUniversity Hospital, Caen

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2020-10-09

View on ClinicalTrials.gov More Muscular Dystrophy, Oculopharyngeal trials