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

Plain-language summary

This Phase 1/2 study evaluates the safety and tolerability of escalating doses of the gene therapy GS030-DP, given as a single intravitreal injection, combined with repeated light stimulation using the GS030-MD medical device (stimulating glasses), in subjects with a documented diagnosis of non-syndromic Retinitis Pigmentosa (RP). It is sponsored by GenSight Biologics. Eligibility requires an age of 18 to 75 years at the time of consent and a clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction and fundoscopic appearance, confirmed on full-field testing.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Active, not recruiting

Sponsor

GenSight Biologics

Intervention

Gene therapy: GS030-DP AND Medical device: GS030-MD

Start date

9/26/2018

Estimated completion

10/26/2027

Countries

United States, France, United Kingdom

Related genes

Locations

• Pittsburgh, United States
• Paris, France
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion 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.

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.