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Gene Therapy Trial Report

Summary

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


NCTID NCT03328130 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Retinitis Pigmentosa
Disease Ontology Term DOID:0110375
Compound Name CTX-PDE6B
Compound Description HORA-PDE6B
Sponsor eyeDNA Therapeutics
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 19 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant PDE6B
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2/5
Editor Type none
Dose 1 3.4E11 vg/eye (n=7)
Dose 2 6.4E11 vg/eye (n=10)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2017-10-05
Completion Date 2025-06-30
Last Update 2025-12-18

Participation Criteria


Eligible Age >=13 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key 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 Exclusion Criteria: * 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND False
FDA Designations Rare Pediatric Disease Designation
Recent Updates

Resources/Links