Natural History Study for Retinitis Pigmentosa

(PHENOROD2 Trial)

Not currently recruiting at 1 trial location

Overseen By Daniel Chung, PhD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: SparingVision

Disqualifiers: Other gene mutations, Ocular disorders, Pregnancy

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to study how vision changes over time in people with Retinitis Pigmentosa (RP), a genetic eye condition affecting night and peripheral vision. Researchers will track vision changes through eye exams and mobility tests over four years. The trial seeks participants with RP caused by specific gene mutations (RHO, PDE6A, or PDE6B) who have some remaining vision in at least one eye. This study helps scientists better understand RP, potentially leading to improved treatments. As an unphased trial, it offers participants the chance to contribute to groundbreaking research that could pave the way for future advancements in RP treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications.

What prior data suggests that these examinations and tests are safe for patients with Retinitis Pigmentosa?

This study does not test new treatments on patients. As a natural history study, researchers observe how patients' conditions change over time without administering new drugs. The focus is on vision changes in individuals with Retinitis Pigmentosa, a genetic eye condition.

The study involves regular eye exams and mobility tests, which are routine procedures with no known risks. Since no new treatments are involved, there are no safety concerns related to experimental medications. Participants can feel reassured that the study is observational, not interventional.

Why are researchers excited about this trial?

Researchers are excited about this trial for retinitis pigmentosa because it offers a chance to better understand the progression of the disease over time. Unlike current treatments that often focus on managing symptoms or slowing progression, this study aims to gather detailed data through repeated ophthalmic examinations and mobility tests over four years. By closely monitoring these aspects, researchers hope to identify new patterns or markers that could lead to more effective and targeted treatments in the future. This long-term follow-up is key to unlocking insights that could revolutionize how retinitis pigmentosa is treated.

What evidence suggests that this trial's methods could be effective for Retinitis Pigmentosa?

This study aims to understand retinitis pigmentosa (RP), a condition where certain cells in the eye gradually stop working. People with RP often have trouble seeing at night and lose their side vision. Currently, no treatments exist to stop or reverse RP, but research is ongoing. This trial includes two study groups: Study Group 1 involves a four-year follow-up with ophthalmic examinations, while Study Group 2 includes both ophthalmic examinations and mobility testing over the same period. Scientists are exploring the genetic causes and progression of RP, especially in individuals with specific gene changes like RHO, PDE6a, or PDE6b. By studying how RP develops over time, researchers hope to find clues that could lead to new treatments in the future.

Who Is on the Research Team?

Isabelle Audo, MD, PhD

Principal Investigator

CHNO XV-XX Paris - CIC 1423

Are You a Good Fit for This Trial?

This study is for individuals with Retinitis Pigmentosa, a degenerative eye condition, specifically caused by mutations in the RHO, PDE6A or PDE6B genes. Participants must have a certain level of visual acuity and field diameter, understand the local language well enough to follow instructions, and be part of a health system if they're in France. Pregnant women or those with other retinal issues are excluded.

Inclusion Criteria

Binocular Visual field diameter ≥ 5° as measured on the Goldmann III-4e isopter at inclusion visit

Patient affiliated to a Health Security System if they are included in a clinical site based in France (per law)

Patients having signed the informed consent form

Exclusion Criteria

You are currently pregnant or breastfeeding.

I have a gene mutation linked to retinitis pigmentosa.

I have an eye condition that could affect my retina's function.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observation

Participants undergo regular ophthalmic examinations and mobility testing to monitor disease progression

4 years

Annual visits for ophthalmic examination and mobility testing

Follow-up

Participants are monitored for safety and effectiveness after the main observation period

1 year

What Are the Treatments Tested in This Trial?

Interventions

• Mobility Test
• Ophthalmic examination
• Ophthalmic examinations

Trial Overview The trial involves regular eye exams and mobility tests to track how Retinitis Pigmentosa progresses over time when it's caused by specific genetic mutations. It's an observational study meaning there's no treatment being tested; instead, researchers are studying the natural course of the disease.

How Is the Trial Designed?

Group I: Study Group 2 Experimental Treatment 2 Interventions

Four years follow-up of patients with ophthalmic examination and mobility testing.

Group II: Study Group 1 Experimental Treatment 1 Intervention

Four years follow up of patients with ophthalmic examination.

Who Is Running the Clinical Trial?

SparingVision

Lead Sponsor

Trials

3

Recruited

230+

Published Research Related to This Trial

In a study of 106 patients with retinitis pigmentosa (RP) over 1 to 5 years, various imaging techniques (SD-OCT, FAF, and MP) were found to reliably measure disease progression, showing strong correlations between structural and functional outcomes.

The annual rates of decline in visual acuity and other parameters were significant, with visual acuity decreasing by an average of 2.3 letters per year, indicating a measurable progression of RP that can be tracked using these imaging methods.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Progression of retinitis pigmentosa on multimodal imaging: The PREP-1 study. Iftikhar, M., Usmani, B., Sanyal, A., et al.[2020]

Citations