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NCT02896387: PRIMAculture

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

Terminated Last updated 3 May 2022
What this trial tests

trial in Ectodermal Dysplasia in 5 participants. Terminated before completion.

Timeline
3 March 2017
Primary endpoint
1 January 2022
1 January 2022

Quick facts

Lead sponsorFondation Ophtalmologique Adolphe de Rothschild
StatusTerminated
Study typeOBSERVATIONAL
Enrollment5
Start date3 March 2017
Primary completion1 January 2022
Estimated completion1 January 2022
Sites2 locations across France

Conditions studied

Sponsor

Fondation Ophtalmologique Adolphe de Rothschild — full company profile →

Who can join

7 and older, any sex, with Ectodermal Dysplasia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

  1. Ethical and Safety Issues of Stem Cell-Based Therapy.
    Volarevic V, Markovic BS, Gazdic M, Volarevic A, et al · · 2018 · cited 552× · PMID 29333086 · DOI 10.7150/ijms.21666

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Other recruiting trials for Ectodermal Dysplasia

Currently open trials in the same condition.

Other Fondation Ophtalmologique Adolphe de Rothschild trials

Trials by the same sponsor.

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