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Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft (TTT-PT-DOP)

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ClinicalTrials.gov Identifier: NCT02878694
Recruitment Status : Unknown
Verified August 2016 by University Hospital, Caen.
Recruitment status was:  Active, not recruiting
First Posted : August 25, 2016
Last Update Posted : August 25, 2016
Sponsor:
Collaborators:
University Hospital, Rouen
Rennes University Hospital
Centre Hospitalier Universitaire, Amiens
University Hospital, Lille
University Hospital, Brest
Groupe Hospitalier Pitie-Salpetriere
Information provided by (Responsible Party):
University Hospital, Caen

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 .


Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Oculopharyngeal Ptosis Biological: Myoblast autologous graft Phase 2 Phase 3

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft
Study Start Date : July 2016
Estimated Primary Completion Date : April 2018
Estimated Study Completion Date : December 2018


Arm Intervention/treatment
Experimental: Myoblast autologous graft
30 million autologous myoblasts in 6 intramuscular injections
Biological: Myoblast autologous graft
30 million autologous myoblasts in 6 intramuscular injections No treatment in the second eye




Primary Outcome Measures :
  1. Improvement of ptosis [ Time Frame: 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.


Secondary Outcome Measures :
  1. acuity [ Time Frame: 12 months ]
    Measure of visual acuity ( without lifting the lid manually and without head tilt ) according Monoyer scale ,

  2. Goldmann Visual Field [ Time Frame: 12 months ]
  3. neck pain with Visual Analaogue Scale [ Time Frame: 12 months ]
  4. Tolerance of the sampling procedure and transplantation, swelling (yes / no) [ Time Frame: baseline ]
  5. Tolerance of the sampling procedure and transplantation : visual analogue scale Pain about the procedure [ Time Frame: baseline ]
  6. Tolerance of collection of the seat and transplantation: hematoma (yes / no ) [ Time Frame: baseline ]
  7. fever [ Time Frame: baseline ]
  8. strength of the levator muscle of the upper eyelid [ Time Frame: 12 months ]
    This is assessed by measuring the travel of the free edge of the upper eyelid between the eyes down and look up blocking the frontal muscle (photos and films) . Normal Range : 15 to 17 mm. Very impaired in patients with DOP .



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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02878694


Locations
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France
CHU Caen
Caen, France, 14000
Sponsors and Collaborators
University Hospital, Caen
University Hospital, Rouen
Rennes University Hospital
Centre Hospitalier Universitaire, Amiens
University Hospital, Lille
University Hospital, Brest
Groupe Hospitalier Pitie-Salpetriere
Investigators
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Study Chair: 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

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Responsible Party: University Hospital, Caen
ClinicalTrials.gov Identifier: NCT02878694     History of Changes
Other Study ID Numbers: 2015-001192-48
First Posted: August 25, 2016    Key Record Dates
Last Update Posted: August 25, 2016
Last Verified: August 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Oculopharyngeal
Prolapse
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Pathological Conditions, Anatomical