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Gene Therapy Trial Report

Summary

Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa


NCTID NCT03326336 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Non-syndromic Retinitis Pigmentosa
Disease Ontology Term DOID:10584
Compound Name GS030
Compound Description rAAV2.7m8-CAG-ChrimsonR-tdTomato
Sponsor GenSight Biologics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 10 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant ChR-tdT
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravitreal
Drug Product Type Viral vector
Target Tissue/Cell Retinal ganglion
Delivery System Viral transduction
Vector Type AAV2.7m8
Editor Type none
Dose 1 5E10 vg/eye (n=3)
Dose 2 1.5E11 vg/eye (n=3)
Dose 3 5E11 vg/eye (n=4)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2017-10-11
Completion Date 2027-10-26
Last Update 2026-03-02

Participation Criteria


Eligible Age 18 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion criteria: * Age ≥18 years to ≤75 years at the time of ICF signature. * Diagnosis of non-syndromic RP defined as: * Clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction, and fundoscopic appearance. * Diagnosis of non-syndromic RP is confirmed on full-field ERG * Visual acuity: * Visual acuity in the dose-escalation cohorts of no better LP. * Visual acuity in the extension cohort of no better than CF pending review of dose-escalation cohort data by the DSMB. * Relatively preserved ganglion cell layer volume and retinal nerve fiber layer thickness, as measured with spectral domain optical coherence tomography (SD-OCT). * Interpupillary distance of ≥51 mm and ≤72 mm. * Refractive error of the study eye between -6 diopters and +6 diopters. Exclusion criteria * Prior receipt of any gene therapy. * Subjects who have undergone significant ocular surgery (per investigator determination) within 3 months prior to Visit 1. * Presence of narrow iridocorneal angles contraindicating pupillary dilation. * Presence of disorders of the ocular media which interfere with visual acuity and other ocular assessments, including SD-OCT, during the study period. * Presence of any systemic or ocular diseases, or pathologies, other than non-syndromic RP, or their associated therapies, that can cause or have the potential to cause vision loss. * Prior vitrectomy or vitreomacular surgery. * Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker and macular hole, evident by ophthalmoscopy and/or by SD-OCT examinations and assessed by the investigator to significantly affect central vision. * Current evidence of retinal detachment assessed by the investigator to significantly affect central vision. * Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis. * Presence of an Active Implantable Medical Device. * Subjects who have undergone thermal laser procedure to the retina within 3 months of trial entry, or any prior thermal laser procedure to the macular region.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations United States,United Kingdom,France

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates

Resources/Links