What side effects are associated with this type of intervention?

Gene therapy for Retinitis Pigmentosa, such as the one studied in the Promising ROd-cone DYstrophy Gene therapY trial, generally involves the use of viral vectors to deliver functional copies of the defective gene to retinal cells. Common side effects of gene therapy can include ocular inflammation, immune responses to the viral vector, and potential retinal damage. Other treatments for RP, such as vitamin A supplementation, may cause side effects like liver toxicity and hypervitaminosis A. Overall, while gene therapy shows promise, it is essential to monitor for these potential adverse effects.

What prior FDA approvals exist?

The FDA has approved voretigene neparvovec (Luxturna) for the treatment of Retinitis Pigmentosa and Leber congenital amaurosis caused by mutations in the RPE65 gene. This gene therapy involves delivering a normal copy of the RPE65 gene directly to retinal cells, thereby compensating for the defective gene and improving vision. Other gene therapies for RP are still under investigation in clinical trials, such as those targeting mutations in the RHO, PDE6A, or PDE6B genes.

What other types of research exist?

Promising novel alternatives to gene therapy for Retinitis Pigmentosa patients include: 1) Retinal implants and prostheses, which can restore some vision by electrically stimulating the retina. 2) Stem cell therapy, which aims to replace damaged retinal cells with healthy ones derived from stem cells. 3) Neuroprotective agents, which can slow the progression of retinal degeneration by protecting retinal cells from damage. 4) Optogenetics, which involves making remaining retinal cells light-sensitive to restore vision. These approaches are in various stages of research and clinical trials and may offer viable treatment options in the near future.

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Gene Therapy

Gene Therapy with SPVN06 for Cone-Rod Dystrophy (PRODYGY Trial)

Phase 1 & 2

Recruiting

Research Sponsored by SparingVision

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to 12 months after administration of gene therapy

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment for patients with advanced vision problems caused by specific genetic mutations. The study aims to determine the appropriate dose and evaluate its effectiveness.

Who is the study for?

Adults over 18 with advanced Retinal Cone Dystrophy (RCD) due to specific genetic mutations, who can consent and follow study rules. They must use birth control if they can have children, be in good general health without serious heart, liver or kidney issues, and not pregnant or breastfeeding. Vision loss should be similar in both eyes.

What is being tested?

The trial is testing SPVN06 gene therapy for RCD in two parts: first, different doses are given to find the safest one; second, patients are randomly put into three groups to compare results while neither doctors nor patients know who gets what treatment.

What are the potential side effects?

Potential side effects aren't specified but may include typical gene therapy reactions like immune responses leading to inflammation or discomfort at injection site. Other risks could involve eye-specific reactions since it's an ocular treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1 year after treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1 year after treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 1 year after treatment for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Evaluation of the safety and tolerability of a single injection of SPVN06 in subjects with advanced RCD due to a mutation in the RHO, PDE6A, or PDE6B gene, 12 months after administration of gene therapy.

Secondary study objectives

Evaluation of preliminary efficacy as assessed by FAF

Evaluation of preliminary efficacy as assessed by color vision

Evaluation of preliminary efficacy as assessed by optical coherence tomography

+7 more

Other study objectives

Exploratory objective - Exploration of biomarkers

Exploratory objective - Photoreceptor mosaic imaging

Awards & Highlights

No Placebo-Only Group

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

Trial Design

6Treatment groups

Experimental Treatment

Active Control

Group I: Step 2 : SPVN06 Dose Recommended 2Experimental Treatment1 Intervention

Participants will receive a single subretinal injection of SPVN06 recommended dose 2 on Day 0

Group II: Step 2 : SPVN06 Dose Recommended 1Experimental Treatment1 Intervention

Participants will receive a single subretinal injection of SPVN06 recommended dose 1 on Day 0

Group III: Step 1 : SPVN06 dose 3Experimental Treatment1 Intervention

Participants will receive a single subretinal injection of SPVN06 Dose 3 on Day 0

Group IV: Step 1 : SPVN06 dose 2Experimental Treatment1 Intervention

Participants will receive a single subretinal injection of SPVN06 Dose 2 on Day 0

Group V: Step 1 : SPVN06 dose 1Experimental Treatment1 Intervention

Participants will receive a single subretinal injection of SPVN06 Dose 1 on Day 0.

Group VI: Step 2 : Control groupActive Control1 Intervention

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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 delivering a functional copy of the defective gene to retinal cells using viral vectors. This helps restore the production of essential proteins, improving or stabilizing vision. This treatment is crucial for RP patients as it addresses the root cause of the disease at the genetic level, potentially halting or reversing vision loss.

The safety and efficacy of gene therapy treatment for monogenic retinal and optic nerve diseases: A systematic review.