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

Korábbi kezelés

• 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.

Más betegség

• 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.

Egyéb

• 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.

• 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.