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

 Cone Dystrophy / Posted 3 weeks ago

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.
  • Inclusion Criteria :
    • Ages Eligible for Study: 18 Years to 75 Years (Adult, Older Adult)
    • Sexes Eligible for Study: All
    • Accepts Healthy Volunteers: No
    • 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.
  • Exclusion 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.
  • Study start date : September 26, 2018
  • Study end date : December 2025
  • Wales-Based Study Contact : Please speak to your clinician
  • Principal Investigator : Dr. Simona Esposti
Contact details

Moorfields Eye Hospital NHS Foundation Trust, 162 City Road, London.London,England,EC1V 2PD  Show Phone Number s.esposti@nhs.net www.moorfields.nhs.uk

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