ACTIVE_NOT_RECRUITING

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

Conditions

Non-syndromic Retinitis Pigmentosa 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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

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

Quick Facts

Study Start:2018-09-26

Study Completion:2027-10-26

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT03326336

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 75 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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. * 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.	Pregnancy or breastfeeding Severe psychiatric disorders Active substance abuse Unstable medical conditions Inability to comply with study requirements

Inclusion Criteria

Exclusion 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.
* 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.

Pregnancy or breastfeeding
Severe psychiatric disorders
Active substance abuse
Unstable medical conditions
Inability to comply with study requirements

Contacts and Locations

Principal Investigator

Magali Taiel, MD

STUDY_DIRECTOR

GenSight Biologics

Study Locations (Sites)

UPMC Eye Center

Pittsburgh, Pennsylvania, 15213

United States

Collaborators and Investigators

Sponsor: GenSight Biologics

Magali Taiel, MD, STUDY_DIRECTOR, GenSight Biologics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2018-09-26

Study Completion Date2027-10-26

Study Record Updates

Study Start Date2018-09-26

Study Completion Date2027-10-26

Terms related to this study

Keywords Provided by Researchers

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

Additional Relevant MeSH Terms

Non-syndromic Retinitis Pigmentosa