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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT03328130
Other study ID # HORA-PDE6B-001
Secondary ID 2016-001429-16
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date November 6, 2017
Est. completion date December 2029

Study information

Verified date March 2024
Source eyeDNA Therapeutics
Contact Medical Director / Chief Development Officer, MD
Phone 01 81 69 87 70
Email contact@eyednatx.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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 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.


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 vision which leads to blindness. RP is a highly variable disorder with some patients developing symptomatic visual loss in childhood whereas others remain asymptomatic until mid-adulthood. There are no treatments available. This study focuses on the form of RP caused by mutations (modifications) in the genetic information necessary to make the protein called rod cGMP phosphodiesterase 6 β subunit (or PDE6β). Clinical diagnosis is made by function tests of the eye and confirmed using a specific method called molecular testing to verify that the PDE6B gene is not correct. This study uses a gene therapy vector inspired from an adeno-associated virus (AAV) called AAV2/5-hPDE6B. This vector intends to supply to the target cells the PDE6B therapeutic gene that is not functioning properly in the cell. The AAV parts of the gene therapy vector work as a vehicle to deliver the normal human PDE6B gene into the cells of the retina.


Recruitment information / eligibility

Status Recruiting
Enrollment 23
Est. completion date December 2029
Est. primary completion date December 2029
Accepts healthy volunteers No
Gender All
Age group 13 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
AAV2/5-hPDE6B
Subretinal administration in one eye

Locations

Country Name City State
France Clinique Ophtalmologique, CHU de Nantes Nantes

Sponsors (1)

Lead Sponsor Collaborator
eyeDNA Therapeutics

Country where clinical trial is conducted

France, 

References & Publications (2)

Petit L, Lheriteau E, Weber M, Le Meur G, Deschamps JY, Provost N, Mendes-Madeira A, Libeau L, Guihal C, Colle MA, Moullier P, Rolling F. Restoration of vision in the pde6beta-deficient dog, a large animal model of rod-cone dystrophy. Mol Ther. 2012 Nov;2 — View Citation

Pichard V, Provost N, Mendes-Madeira A, Libeau L, Hulin P, Tshilenge KT, Biget M, Ameline B, Deschamps JY, Weber M, Le Meur G, Colle MA, Moullier P, Rolling F. AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6beta-deficient Dogs. Mol Ther. 2016 — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary Incidence of ocular and non-ocular adverse events 1 year + 4 years follow-up
Secondary Improvement in visual function Improvement in visual function as assessed by mobility test 1 year + 4 years follow-up
Secondary Improvement in visual fields Improvement in visual fields as assessed by visual fields measurements 1 year + 4 years follow-up
Secondary Improvement in visual function Improvement in visual function as assessed by reading speed 1 year + 4 years follow-up
Secondary Improvement in Quality of Life Quality of life will be measured by Quality of Life questionnaire National Eye Institute Visual Function Questionnaire (NEI VFQ-25) 1 year + 4 years follow-up
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