Active, Not Recruiting

Phase 1

Phase 2

Age: 18Years - 75Years

All Genders

ID03326336

A Phase 12a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

Led by GenSight Biologics · Updated on 2026-03-02

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What This Trial Is About

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

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years - 75Years

All Genders

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.

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

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A Phase 12a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

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AI-Summary

What This Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

How is the study designed?

Not the Right Trial for You?

On this page

Sponsors

Sponsors

Who Can Participate

Eligibility Criteria

How is the study designed?

Sponsors