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

GenSight Biologics

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Non-syndromic Retinitis Pigmentosa

Treatments: Combination Product: Gene therapy: GS030-DP AND Medical device: GS030-MD

Study type: Interventional

Funder types: Industry

Identifiers

NCT03326336

2017-002204-27 (EudraCT Number)

GS030_CLIN_001

Details and patient eligibility

About

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

Full description

Study GS030_CLIN_001 is a multicenter, Phase 1/2a, open-label, non-randomized, dose-escalation, safety and tolerability study of GS030-DP in association with GS030-MD in subjects with non-syndromic RP that is confirmed by full-field ERG. The study design includes a dose escalation of the vector encoding the ChR-tdT. This first-in-human study design evaluates the safety and tolerability of GS030, the associated GS030-MD and GS030-DP, which is the treatment to be developed and considered for marketing.

Enrollment: 10 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Cohort

Other group

Description:

3 dose escalation cohorts (low, medium and high dose) with 3 subjects per cohort followed by an extension cohort at the highest-well tolerated dose with 3 to 9 subjects.

Treatment:

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