NCT03326336Active, not recruitingPhase 1/2
Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
Source: ClinicalTrials.gov · Last updated 2 Mar 2026
Brief summary (registry, in English)
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
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 10 (actual)
- First submitted
- 11 Oct 2017
- Start date
- 26 Sept 2018
- Primary completion date
- 26 Oct 2027
- Lead sponsor
- GenSight Biologics
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT03326336
- EU CT number
- 2024-516059-42-00
- EudraCT number
- 2017-002204-27
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 10
- From 26 Sept 2018 to the primary completion date, 26 Oct 2027
- 3 sites in 3 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior receipt of any gene therapy.
- Subjects who have undergone significant ocular surgery (per investigator determination) within 3 months prior to Visit 1.
- Prior vitrectomy or vitreomacular surgery.
- 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.
Other illness
- 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.
- Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
Other
- Presence of narrow iridocorneal angles contraindicating pupillary dilation.
- 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.
- Presence of an Active Implantable Medical Device.
Who the study is looking for
- 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.
Age in the registry: 18 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Quinze-Vingts National Ophthalmology Hospital Coordinator of this site? Confirm its status | Paris | France | Recruiting |
| Moorfields Eye Hospital NHS Foundation Trust, 162 City Road Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| UPMC Eye Center Coordinator of this site? Confirm its status | Pittsburgh | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.