Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft

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 .

Conditions

• Muscular Dystrophy, Oculopharyngeal
• Ptosis

Interventions

BIOLOGICAL

Myoblast autologous graft

30 million autologous myoblasts in 6 intramuscular injections No treatment in the second eye

Sponsors & Collaborators

• University Hospital, Rouen

  collaborator OTHER

• Rennes University Hospital

  collaborator OTHER

• Centre Hospitalier Universitaire, Amiens

  collaborator OTHER

• University Hospital, Lille

  collaborator OTHER

• University Hospital, Brest

  collaborator OTHER

• Groupe Hospitalier Pitie-Salpetriere

  collaborator OTHER

• University Hospital, Caen

  lead OTHER

Principal Investigators

• Françoise CF Chapon, PhD · CHU CAEN

• Olivier BO Boyer, PhD · CHU Rouen

• Frederic MF Mouriaux, PhD · CHU Rennes

• Sophie PS Perie, PhD · APHP

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Max Age

75 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2019-11-14

Primary Completion

2020-10-09

Completion

2020-10-09

Countries

• France

Study Locations