NCT02878694

Brief 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 .

Trial Health

57
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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Nov 2019

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 8, 2016

Completed
6 months until next milestone

First Posted

Study publicly available on registry

August 25, 2016

Completed
3.2 years until next milestone

Study Start

First participant enrolled

November 14, 2019

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 9, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 9, 2020

Completed
Last Updated

November 25, 2020

Status Verified

November 1, 2020

Enrollment Period

11 months

First QC Date

March 8, 2016

Last Update Submit

November 23, 2020

Conditions

Muscular Dystrophy, OculopharyngealPtosis

Outcome Measures

Primary Outcomes (1)

  • Improvement of ptosis

    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.

    baseline and 12 months

Secondary Outcomes (8)

  • acuity

    12 months

  • Goldmann Visual Field

    12 months

  • neck pain with Visual Analaogue Scale

    12 months

  • Tolerance of the sampling procedure and transplantation, swelling (yes / no)

    baseline

  • Tolerance of the sampling procedure and transplantation : visual analogue scale Pain about the procedure

    baseline

  • +3 more secondary outcomes

Study Arms (1)

Myoblast autologous graft

EXPERIMENTAL

30 million autologous myoblasts in 6 intramuscular injections

Biological: Myoblast autologous graft

Interventions

Myoblast autologous graftBIOLOGICAL

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

Myoblast autologous graft

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

CHU Caen

Caen, 14000, France

Location

MeSH Terms

Conditions

Muscular Dystrophy, OculopharyngealBlepharoptosis

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesEyelid DiseasesEye Diseases

Study Officials

  • 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

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 8, 2016

First Posted

August 25, 2016

Study Start

November 14, 2019

Primary Completion

October 9, 2020

Study Completion

October 9, 2020

Last Updated

November 25, 2020

Record last verified: 2020-11

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)