Informations générales (source: ClinicalTrials.gov)

NCT03328130 En recrutement
Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients With Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression
Interventional
  • Rétinite
  • Rétinite pigmentaire
Phase 1/Phase 2
eyeDNA Therapeutics (Voir sur ClinicalTrials)
novembre 2017
décembre 2029
29 juin 2024
The study is a Phase I/II, monocentric, open-label, dose-ranging safety and efficacy gene therapy intervention by subretinal administration of AAV2/5-hPDE6B. At least twelve patients 18 years of age or older, within four consecutive cohorts of patients, will be recruited. Then at least four patients 13 years of age or older, within a fifth cohort, will be recruited.
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Etablissements

Les établissements hors Île-de-France dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données
Clinique Ophtalmologique, CHU de Nantes - 44093 - Nantes - France Pierre Lebranchu En recrutement Contact (sur clinicalTrials)

Critères

Tous
Inclusion Criteria:

- Clinical and molecular diagnosis of retinitis pigmentosa caused by defect in PDE6B
gene without other syndromic manifestations

- Aged above 13 years

- Ability to give informed consent

Key


- Previous ocular surgery or thermal laser within 6 months before the surgery

- Lens opacities or obscured ocular media upon recruitment such reliable evaluation or
grading of the posterior segment cannot be performed

- Known serious allergies to the fluorescein dye used in angiography, to the
mydriatic, steroidal and non-steroidal eye drops

- Participation in another clinical trial with an investigational agent

- Enrolled or being enrolled in another gene therapy clinical trial

- Active, extraocular infection requiring the prolonged or chronic use of
antimicrobial agents

- Chronic medical conditions, cancer

- Abnormal laboratory values

- On immunosuppressive therapy