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

NCT ID: NCT03326336

Last Updated: 2025-08-26

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-09-26
• Study Completion Date: 2027-10-26

Brief 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

Detailed Description

Study GS030_CLIN_001 is a multicenter, Phase 1/2a, open-label, non-randomized, dose-escalation, safety and tolerability study of GS030-DP in association with GS030-MD in subjects with non-syndromic RP that is confirmed by full-field ERG. The study design includes a dose escalation of the vector encoding the ChR-tdT. This first-in-human study design evaluates the safety and tolerability of GS030, the associated GS030-MD and GS030-DP, which is the treatment to be developed and considered for marketing.

Conditions

Non-syndromic Retinitis Pigmentosa

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort

3 dose escalation cohorts (low, medium and high dose) with 3 subjects per cohort followed by an extension cohort at the highest-well tolerated dose with 3 to 9 subjects.

Group Type: OTHER

Gene therapy: GS030-DP AND Medical device: GS030-MD

Intervention Type: COMBINATION_PRODUCT

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)

Interventions

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)

Intervention Type: COMBINATION_PRODUCT

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

GenSight Biologics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Magali Taiel, MD

Role: STUDY_DIRECTOR

GenSight Biologics

Locations

UPMC Eye Center

Pittsburgh, Pennsylvania, United States

Centre Hospitalier National d'Ophtalmologie (CHNP) des Quinze-Vingts

Paris, , France

Moorfields Eye Hospital NHS Foundation Trust, 162 City Road

London, , United Kingdom

Countries

United States; France; United Kingdom

Related Links

http://www.gensight-biologics.com/

Website of GenSight Biologics

Other Identifiers

2017-002204-27

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GS030_CLIN_001

Identifier Type: -

Identifier Source: org_study_id