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

Phase 1

Active, not recruiting

• Conditions: Non-syndromic Retinitis Pigmentosa

• Registration Number: NCT03326336
• Lead Sponsor: GenSight Biologics

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.

Study Timeline

• Study First Submitted: 2017-10-11
• Study First Submitted QC: 2017-10-25
• Study First Posted: 2017-10-31
• Study Start: 2018-09-26
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-20
• Last Update Posted: 2025-08-26
• Primary Completion: 2027-10-26
• Study Completion: 2027-10-26

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
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.

Secondary Outcome Measures

Name	Time	Method
Evaluate the treatment effect of GS030 as assessed by visual acuity	Week 52/Year 1	Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with the Freiburg Visual Acuity \& Contrast Test (FrACT) free computer program that uses graphics capabilities and psychometric methods to provide automated, self-paced measurement and the full field threshold stimulus test (FST) measuring the illuminance necessary to stimulate the most sensitive parts of the retina, and thus determines a quantifiable stimulation threshold (before and after gene transfer, with GS030-MD turned ON and turned OFF).
Evaluate the treatment effect of GS030 as assessed by mobility (line task).	Week 52/Year 1	Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with line task (before and after gene transfer, with GS030-MD turned ON and turned OFF).
Evaluate the treatment effect of GS030 as assessed by QoL (SF36)	Week 52/Year 1	Assessment of the treatment effect on quality of life changes from baseline to Week 52 with the Short Form Survey 36 Version 2 (SF-36v2). The SF-36v2 is a subject-rated 36-item questionnaire assessing subject health. There are 8 scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0 to 100 scale. A lower score indicates more disability, and a higher score indicates less disability (a score of 0 is equivalent to maximum disability, and a score of 100 is equivalent to no disability).
Evaluate the treatment effect of GS030 as assessed by visual function	Week 52/Year 1	Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Humphrey visual field 10-2 Standardized automated perimetry, square localization test, displaying white square at a random location on a black background and direction of motion test measuring the ability of subjects to determine the direction of an object moving in the visual field (before and after gene transfer, with GS030-MD turned ON and turned OFF).
Evaluate the treatment effect of GS030 as assessed by mobility (door task).	Week 52/Year 1	Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with door task (before and after gene transfer, with GS030-MD turned ON and turned OFF).
Evaluate the treatment effect of GS030 as assessed by QoL (VFQ25)	Week 52/Year 1	Assessment of the treatment effect on quality of life changes from baseline to Week 52 with the Visual Functioning Questionnaire-25 (VFQ-25). VFQ25 is a 25-item questionnaire with 47 questions, each question has several responses scored on a scale from 0 to 5, 0 to 6, or 0 to 10. Values are calculated in percentages.
Evaluate immune response to recombinant adeno associated viral vector, derived from serotype 2 (rAAV2.7m8) and ChR tdT protein.	Week 52/Year 1	Immune response to rAAV2.7m8 and ChR-tdT protein from baseline to week 52

Trial Locations

Locations (3)

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