Clinical trial NCT03326336: Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
Official title: A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Non-syndromic Retinitis Pigmentosa
Keywords: Eye Diseases
Hereditary Eye Diseases
Retinal degeneration
Inherited retinal diseases
Rod & cone dystrophies
Retinitis Pigmentosa
Gene Therapy
Optogenetic
Intravitreal Injections
AAV Vectors
Medical device
Visual Interface
Lead sponsor: GenSight Biologics
Sponsor class: INDUSTRY
First posted: 2017-10-31
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Intervention model description: 3 dose-escalation cohorts with 3 subjects per each cohort plus 1 extension cohort at the highest well-tolerated dose.
Primary purpose: TREATMENT

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


Eligibility 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.

Primary outcomes:
measure: The safety and tolerability of escalating doses of GS030-DP administered via a single IVT and repeated light stimulation using GS030-MD in subjects with non-syndromic Retinitis Pigmentosa
timeFrame: Week 52/Year 1
Secondary outcomes:
measure: Evaluate the treatment effect of GS030 as assessed by visual acuity
timeFrame: Week 52/Year 1
measure: Evaluate the treatment effect of GS030 as assessed by visual function
timeFrame: Week 52/Year 1
measure: Evaluate the treatment effect of GS030 as assessed by mobility.
timeFrame: Week 52/Year 1
measure: Evaluate the treatment effect of GS030 as assessed by mobility.
timeFrame: Week 52/Year 1
measure: Evaluate the treatment effect of GS030 as assessed by QoL
timeFrame: Week 52/Year 1
measure: Evaluate the treatment effect of GS030 as assessed by QoL
timeFrame: Week 52/Year 1
measure: Evaluate immune response to recombinant adeno associated viral vector, derived from serotype 2 (rAAV2.7m8) and ChR tdT protein.
timeFrame: Week 52/Year 1
Other outcomes:
Locations:
city: Pittsburgh
country: United States
facility: UPMC Eye Center
state: Pennsylvania
city: Paris
country: France
facility: Centre Hospitalier National d'Ophtalmologie (CHNP) des Quinze-Vingts
city: London
country: United Kingdom
facility: Moorfields Eye Hospital NHS Foundation Trust, 162 City Road
Study officials:
References: