What side effects are associated with this type of intervention?

Gene therapy for Retinitis Pigmentosa, such as GS030-DP, involves intravitreal injections and has been primarily evaluated for safety and tolerability. Known side effects from similar gene therapy trials include mild to moderate inflammation, increased intraocular pressure, and potential retinal detachment. Light stimulation treatments, like GS030-MD, are generally well-tolerated but can cause discomfort, glare, or photophobia. Both treatments are still under investigation, and long-term safety data are required to fully understand their risk profiles.

What prior FDA approvals exist?

As of now, the FDA has approved Luxturna (voretigene neparvovec-rzyl) for the treatment of inherited retinal diseases, including certain forms of Retinitis Pigmentosa caused by mutations in the RPE65 gene. Luxturna is a gene therapy that delivers a normal copy of the RPE65 gene directly to retinal cells. There are no FDA-approved treatments specifically involving light stimulation for Retinitis Pigmentosa. Other treatments are generally supportive, focusing on managing symptoms and slowing disease progression.

What other types of research exist?

Promising alternatives to GS030-DP and GS030-MD for Retinitis Pigmentosa include: 1) Ad.BDNF gene therapy, which delivers brain-derived neurotrophic factor to Müller cells to protect photoreceptors from degeneration. 2) Intravitreal administration of Stanniocalcin-1 (STC-1), which has shown to improve retinal function and reduce oxidative stress and inflammation in a porcine model. 3) SRD005825, a pharmacologic chaperone of opsin, which promotes photoreceptor survival in animal models of autosomal dominant RP.

← Back to Search

Gene Therapy

Gene Therapy with Light-Stimulating Glasses for Retinitis Pigmentosa (PIONEER Trial)

Phase 1 & 2

Recruiting

Research Sponsored by GenSight Biologics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of non-syndromic RP confirmed on full-field ERG

Diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction, and fundoscopic appearance

Must not have

Prior receipt of any gene therapy

Significant ocular surgery within 3 months prior to Visit 1

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 52/year 1

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new gene therapy injected into the eye and special light-stimulating glasses for patients with a specific type of vision loss called non-syndromic Retinitis Pigmentosa. The gene therapy aims to fix genetic problems in the eye, while the glasses help activate the treated cells. Gene therapy has shown promise in treating retinal diseases.

Who is the study for?

Adults aged 18-75 with non-syndromic Retinitis Pigmentosa (RP), confirmed by specific tests, who have not had gene therapy before or significant eye surgery within the last 3 months. Participants should have a certain range of visual acuity and refractive error, as well as an appropriate interpupillary distance.

What is being tested?

The trial is testing GS030-DP, a new gene therapy given through an injection in the eye, alongside GS030-MD, special glasses that provide light stimulation. The study aims to find out how safe these treatments are and what doses are tolerable for people with RP.

What are the potential side effects?

Potential side effects aren't specified here but generally could include discomfort at the injection site, inflammation in the eye, headache from light stimulation or vision changes due to treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My retinitis pigmentosa diagnosis was confirmed with an ERG test.

Select...

I have been diagnosed with non-syndromic retinitis pigmentosa.

Select...

I am between 18 and 75 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have never received gene therapy.

Select...

I have not had major eye surgery in the last 3 months.

Select...

I have a condition affecting my eye's central vision due to vitreo-macular adhesion or similar issues.

Select...

I have had eye surgery for a vitreous or macular issue.

Select...

My central vision is affected by retinal detachment.

Select...

I cannot have my pupils dilated due to narrow angles in my eyes.

Select...

I have eye conditions that affect my vision.

Select...

I have a condition or am receiving treatment that could cause vision loss.

Select...

I have ongoing eye inflammation or a history of uveitis.

Select...

I haven't had any laser treatments on my retina in the last 3 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 52/year 1

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 52/year 1

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 52/year 1 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Secondary study objectives

Evaluate immune response to recombinant adeno associated viral vector, derived from serotype 2 (rAAV2.7m8) and ChR tdT protein.

Evaluate the treatment effect of GS030 as assessed by QoL

Therapeutic procedure

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: CohortExperimental Treatment1 Intervention

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.

0 / 13Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Gene therapy for Retinitis Pigmentosa (RP) involves introducing or correcting specific genes within retinal cells to restore or improve their function, addressing the genetic mutations that cause photoreceptor degeneration. Light stimulation uses devices to activate these treated retinal cells, enhancing the functionality of the corrected genes. This combination can halt or reverse vision loss and improve visual outcomes, offering a comprehensive treatment strategy for RP patients.

Protective effects of systemic treatment with methylprednisolone in a rodent model of non-arteritic anterior ischemic optic neuropathy (rAION).