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

NCT03326336 | Active Not Recruiting Phase 1 DMC FDA Drug FDA Device

• 2 other identifiers: GS030_CLIN_0012017-002204-27
• Study Type: interventional
• Target: 10
• Locations: 3 countries
• Sites: 3

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

Conditions

Non-syndromic Retinitis Pigmentosa

Keywords

Eye Diseases; Hereditary Eye Diseases; Retinal degeneration; Inherited retinal diseases; Rod & cone dystrophies; Retinitis Pigmentosa; Gene Therapy; Optogenetic; Intravitreal Injections; AAV Vectors; Medical device; Visual Interface

Outcome Measures

Primary Outcomes (1)

• 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

  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.

  Week 52/Year 1

Secondary Outcomes (7)

• Evaluate the treatment effect of GS030 as assessed by visual acuity

  Week 52/Year 1

• Evaluate the treatment effect of GS030 as assessed by visual function

  Week 52/Year 1

• Evaluate the treatment effect of GS030 as assessed by mobility (door task).

  Week 52/Year 1

• Evaluate the treatment effect of GS030 as assessed by mobility (line task).

  Week 52/Year 1

• Evaluate the treatment effect of GS030 as assessed by QoL (VFQ25)

  Week 52/Year 1

Study Arms (1)

Cohort

OTHER

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.

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

Interventions

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

Cohort

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• GenSight Biologics: lead

Study Sites (3)

UPMC Eye Center

Pittsburgh, Pennsylvania, 15213, United States

Location

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

Paris, 75012, France

Location

Moorfields Eye Hospital NHS Foundation Trust, 162 City Road

London, United Kingdom

Location

Related Links

• Website of GenSight Biologics

MeSH Terms

Conditions

Eye Diseases; Eye Diseases, Hereditary; Retinal Degeneration; Cone-Rod Dystrophies; Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Retinal Diseases; Retinal Dystrophies

Study Officials

• GenSight Biologics

  GenSight Biologics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 3 dose-escalation cohorts with 3 subjects per each cohort plus 1 extension cohort at the highest well-tolerated dose.

Study Record Dates

• First Submitted: October 11, 2017
• First Posted: October 31, 2017
• Study Start: September 26, 2018
• Primary Completion (Estimated): October 26, 2027
• Study Completion (Estimated): October 26, 2027
• Last Updated: March 2, 2026

Record last verified: 2026-02

Locations

FR (1); US (1); GB (1)