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Ability of a Molecule (Prima) to Restore Physiological Differentiation in Epithelium Expressing Gene p63 (PRIMAculture)

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ClinicalTrials.gov Identifier: NCT02896387
Recruitment Status : Recruiting
First Posted : September 12, 2016
Last Update Posted : November 6, 2018
Sponsor:
Collaborator:
Institut National de la Santé Et de la Recherche Médicale, France
Information provided by (Responsible Party):
Fondation Ophtalmologique Adolphe de Rothschild

Brief Summary:

Ectodermal dysplasia associated with p63 is a rare disease which, in addition to limbic abnormalities, primarily affects the skin and cornea. The most common forms are called Ectrodactyly, Ectodermal dysplasia, palate Key for cleft lip and palate (EEC) and Ankyloblepharon, Ectodermal dysplasia, cleft lip and palate (AEC). Apart from symptomatic treatment, no cure is available. To understand the molecular defects associated with this disease and to identify therapeutic tools, a research team modelized the disease by reprograming EEC and AEC patient fibroblasts in pluripotent stem cells (iPSC), then induced iPSC differentiation in patients and controls epidermal (skin) and limbic (cornea) cells and demonstrated that the mutated cells can reproduce in vitro the abnormalities observed in patients.

P63 gene belongs to the family of p53 gene. The functions of the two proteins are very similar. Data suggest that molecule Prima could reactivate the p63 protein mutated in patients and thus alleviate skin defect healing and limbic regeneration.


Condition or disease
Ectodermal Dysplasia

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Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Other
Time Perspective: Cross-Sectional
Official Title: Ex Vivo and in Vitro Assessment of the Pharmacological Properties of Molecule Prima in the Restoration of Physiological Differentiation of Gene p63 Dependant Epithelium
Actual Study Start Date : March 3, 2017
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : March 2021





Primary Outcome Measures :
  1. the differentiation of corneal epithelium by histological observation [ Time Frame: baseline ]
    the differentiation of corneal epithelium by histological observation



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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patient with genetic pathology of the ocular surface
Criteria

Inclusion Criteria:

  • Patient with genetic pathology of the ocular surface
  • Age ≥ 7 years

Exclusion Criteria:

  • Agonal glaucoma
  • low vision mostly related to retinal pathology
  • Pregnant or breast-feeding patient

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): NCT02896387


Contacts
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Contact: Laurence SALOMON, MD, PhD 0033148036431 lsalomon@for.paris

Locations
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France
Fondation Opthalmologique A de Rothschild Recruiting
Paris, France, 75019
Contact: Eric GABISON         
Hopital Necker Not yet recruiting
Paris, France, 75019
Contact: Smail HADJ RABIA         
Sponsors and Collaborators
Fondation Ophtalmologique Adolphe de Rothschild
Institut National de la Santé Et de la Recherche Médicale, France
Investigators
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Principal Investigator: Eric GABISON Fondation ophtalmologique de Rothschild

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Responsible Party: Fondation Ophtalmologique Adolphe de Rothschild
ClinicalTrials.gov Identifier: NCT02896387     History of Changes
Other Study ID Numbers: EGN_2016_14
First Posted: September 12, 2016    Key Record Dates
Last Update Posted: November 6, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Ectodermal Dysplasia
Abnormalities, Multiple
Congenital Abnormalities
Skin Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases