Evaluating a new gene therapy for 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
This study is testing a new gene therapy for people with Retinitis Pigmentosa to see if it is safe and how well it works when given through an eye injection along with a special light device.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | GenSight Biologics Industry-sponsored |
| Locations | 3 sites (Pittsburgh, Pennsylvania and 2 other locations) |
| Trial ID | NCT03326336 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety and tolerability of a gene therapy called GS030-DP, which is administered through a single intravitreal injection. Participants will also use a medical device known as GS030-MD, which provides repeated light stimulation. The study involves dose-escalation to determine the optimal dosage for patients diagnosed with non-syndromic Retinitis Pigmentosa. It includes adults aged 18 to 75 with specific visual acuity and retinal measurements.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 to 75 with a confirmed diagnosis of non-syndromic Retinitis Pigmentosa and specific visual acuity criteria.
Not a fit: Patients who have previously received gene therapy or undergone significant ocular surgery within the last three months may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could improve vision and quality of life for patients suffering from Retinitis Pigmentosa.
How similar studies have performed: While gene therapies for retinal diseases are emerging, this specific approach using GS030 has not been widely tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Main selection 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. Main non-selection 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.
Where this trial is running
Pittsburgh, Pennsylvania and 2 other locations
- UPMC Eye Center — Pittsburgh, Pennsylvania, United States (Recruiting)
- Centre Hospitalier National d'Ophtalmologie (CHNP) des Quinze-Vingts — Paris, France (Recruiting)
- Moorfields Eye Hospital NHS Foundation Trust, 162 City Road — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Magali Taiel, MD
- Email: mtaiel@gensight-biologics.com
- Phone: +33 (0)7 62 89 12 52
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.