Status:

TERMINATED

Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene

Lead Sponsor:

eyeDNA Therapeutics

Conditions:

Retinitis Pigmentosa

Eligibility:

All Genders

13+ years

Phase:

PHASE1

PHASE2

Brief Summary

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 ...

Detailed Description

Retinitis pigmentosa (RP) is a disease where part of the eye (the retina) is degenerating over time. Patients initially present with night blindness, and later in life experience loss of central visio...

Eligibility Criteria

Inclusion

  • Key
  • 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

Exclusion

  • 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

Key Trial Info

Start Date :

November 6 2017

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

June 30 2025

Estimated Enrollment :

19 Patients enrolled

Trial Details

Trial ID

NCT03328130

Start Date

November 6 2017

End Date

June 30 2025

Last Update

December 18 2025

Active Locations (1)

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Page 1 of 1 (1 locations)

1

Clinique Ophtalmologique, CHU de Nantes

Nantes, France, 44093