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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02878694 |
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Date of registration:
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08/03/2016 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft
TTT-PT-DOP |
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Scientific title:
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Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft |
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Date of first enrolment:
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November 14, 2019 |
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Target sample size:
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1 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT02878694 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2/Phase 3
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Countries of recruitment
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France
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Contacts
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Name:
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Olivier BO Boyer, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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CHU Rouen |
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Name:
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Frederic MF Mouriaux, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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CHU Rennes |
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Name:
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Sophie PS Perie, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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APHP |
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Name:
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Françoise CF Chapon, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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CHU Caen |
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Key inclusion & exclusion criteria
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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
Age minimum:
18 Years
Age maximum:
75 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Ptosis
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Muscular Dystrophy, Oculopharyngeal
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Intervention(s)
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Biological: Myoblast autologous graft
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Primary Outcome(s)
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Improvement of ptosis
[Time Frame: baseline and 12 months]
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Secondary Outcome(s)
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fever
[Time Frame: baseline]
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Tolerance of collection of the seat and transplantation: hematoma (yes / no )
[Time Frame: baseline]
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neck pain with Visual Analaogue Scale
[Time Frame: 12 months]
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acuity
[Time Frame: 12 months]
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Goldmann Visual Field
[Time Frame: 12 months]
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strength of the levator muscle of the upper eyelid
[Time Frame: 12 months]
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Tolerance of the sampling procedure and transplantation : visual analogue scale Pain about the procedure
[Time Frame: baseline]
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Tolerance of the sampling procedure and transplantation, swelling (yes / no)
[Time Frame: baseline]
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Secondary ID(s)
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2015-001192-48
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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