A Phase 1/2 interventional study of Gene therapy: GS030-DP AND Medical device: GS030-MD in Non-syndromic Retinitis Pigmentosa, sponsored by GenSight Biologics. Active, not recruiting at 3 sites in 3 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-03-02.
Sponsored by GenSight Biologics · Phase 1/2, Interventional, and Treatment
Among 119 studies in Retinitis Pigmentosa with a site in United States — 20 open to participants now, 44 under way, 6 of them phase 3. Retinitis Pigmentosa · United States.
Among the 119 studies in Retinitis Pigmentosa with a site in United States, this one is the 58th largest by enrollment of the 78 interventional ones that give a count, is one of 4 that started in 2018, is one of 20 in phase 1/2, reaches primary completion later than 22 of the 42 other live ones, and was the 57th registered.
278 studies on the registry are indexed under Retinitis Pigmentosa; 72 are open to participants now.
This study's enrollment of 10 is below the median of 27 across 179 interventional studies indexed under Retinitis Pigmentosa.
Browse Retinitis Pigmentosa studies →20 of the 119 studies in Retinitis Pigmentosa with a site in United States are open now, in 12 countries. Each count lists the studies.
Of the 49 Retinitis Pigmentosa studies recruiting now, 37 (76%) set no upper age limit and 7 (14%) stop at 75 or younger; 16 take children under 18; 10 take healthy volunteers.
This study: 18 Years to 75 Years · all sexes · no healthy volunteers.
The full table on the hub →119 studies; the count open now beside each. This study's row is marked.
First in 1993; this study’s year is marked. 4 started in 2018.
Median enrollment 21 across the 78 interventional studies, 73 across the 38 observational. Of the 69 that completed or were terminated, 30 (43%) have results posted.
60 of 119 have an industry lead sponsor. The 10 most frequent:
GenSight Biologics is the lead sponsor of 9 studies on the registry; 1 is open to participants now.
GenSight Biologics has 1 study filed under Retinitis Pigmentosa — fewer than ten, so there is no comparison to draw.
11 of the studies in Retinitis Pigmentosa with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06388200Phase 3OcugenA Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa | 2027-02-12 |
| NCT06912633jCyte, IncSafety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP) | 2026-12 |
| NCT02086890Nova Southeastern UniversityElectro-acupuncture and Transcorneal Electrical Stimulation (TES) for Retinitis Pigmentosa | 2026-12 |
| NCT04278131Bionic Sight LLCBS01 in Patients With Retinitis Pigmentosa | 2026-12-30 |
| NCT05748873SparingVisionPromising ROd-cone DYstrophy Gene therapY | 2027-02 |
Among the 119 studies in Retinitis Pigmentosa with a site in United States, from their registry updates:
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
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.
278 studies on the registry are indexed under Retinitis Pigmentosa; 72 are open to participants now.
This study's enrollment of 10 is below the median of 27 across 179 interventional studies indexed under Retinitis Pigmentosa.
Browse Retinitis Pigmentosa studies →GenSight Biologics is the lead sponsor of 9 studies on the registry; 1 is open to participants now.
Compared within Retinitis Pigmentosa, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Eye Diseases. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Diagnosis of non-syndromic RP defined as:
Visual acuity:
Exclusion criteria
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
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)
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.
Time frame: Week 52/Year 1
Evaluate the treatment effect of GS030 as assessed by visual acuity
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).
Time frame: Week 52/Year 1
Evaluate the treatment effect of GS030 as assessed by visual function
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).
Time frame: Week 52/Year 1
Evaluate the treatment effect of GS030 as assessed by mobility (door task).
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).
Time frame: Week 52/Year 1
Evaluate the treatment effect of GS030 as assessed by mobility (line task).
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).
Time frame: Week 52/Year 1
Evaluate the treatment effect of GS030 as assessed by QoL (VFQ25)
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.
Time frame: Week 52/Year 1
Evaluate the treatment effect of GS030 as assessed by QoL (SF36)
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).
Time frame: Week 52/Year 1
Evaluate immune response to recombinant adeno associated viral vector, derived from serotype 2 (rAAV2.7m8) and ChR tdT protein.
Immune response to rAAV2.7m8 and ChR-tdT protein from baseline to week 52
Time frame: Week 52/Year 1
Started Sep 2018 (actual). Primary completion is estimated for Oct 2027, 109 months after the start.
Of 28 phase 1/2 interventional studies under Retinitis Pigmentosa, half reached primary completion within 53 months of starting (the median), counting those still running; a quarter within 22 and three quarters within 92. This study's planned 109 months is above that.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
GenSight Biologics