Gene therapy: GS030-DP AND Medical device: GS030-MD
Gene therapy: GS030-DP AND Medical device: GS030-MD: GS030-Drug Product (GS030-DP) - Recombinant adeno-associated viral vector, derived from serotype 2 (rAAV2.7m8), containing the optimized channelrhodopsin ChrimsonR-tdTomato gene under the control of the ubiquitous CAG promoter (rAAV2.7m8-CAG-ChrimsonR-tdTomato) GS030-Medical Device (GS030-MD) - Visual Interface Stimulating Glasses (that amplify the external visual stimulus to the optogenetically engineered retina)
Study summary
The objective of this study is to evaluate the safety and tolerability of escalating doses of a gene therapy called GS030-DP (injected study treatment) administered via a single intravitreal injection and repeated light stimulation using a medical device called GS030-MD (stimulating glasses) in subjects with documented diagnosis of non-syndromic Retinitis Pigmentosa
Eligibility
Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
The safety and tolerability of escalating doses of GS030-DP administered via a single IVT and repeated light stimulation using GS030-MD in subjects with non-syndromic Retinitis Pigmentosa — Week 52/Year 1 Safety and tolerability of GS030 treatment at Week 52/Year 1, by assessments based on local and systemic safety issues, specifically those related to IVT of GS030-DP and the subsequent repeated use of GS030-MD, as assessed by incidence of Adverse Events.
Trial sites (3)
Facility
City
Region
Status
UPMC Eye Center
Pittsburgh
Pennsylvania
Centre Hospitalier National d'Ophtalmologie (CHNP) des Quinze-Vingts
Paris
France
Moorfields Eye Hospital NHS Foundation Trust, 162 City Road
London
United Kingdom
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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