Natural History Study for Retinitis Pigmentosa

Not currently recruiting at 6 trial locations

Overseen By Clare Guerrero

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: PYC Therapeutics

Must not be taking: Anti-VEGF, Corticosteroids

Disqualifiers: Uncontrolled systemic disease, Other RP mutations, Others

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

Compensation: Varies

Number of Visits: 18

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to better understand the progression of a rare eye condition known as PRPF31 mutation-associated retinal dystrophy, or retinitis pigmentosa type 11 (RP11). Researchers seek to observe how this condition affects vision and quality of life over time, without testing any new treatments. Individuals with a confirmed diagnosis of this condition and the ability to attend all study visits might be suitable candidates for this trial.

As an unphased trial, this study offers participants the opportunity to contribute to valuable research that could enhance understanding and future management of RP11.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, if you have used certain eye treatments like anti-VEGF agents or specific implants recently, you may not be eligible to participate.

Why are researchers excited about this trial?

This trial is exciting because it aims to gather crucial information about PRPF31 mutation-associated retinal dystrophy, a rare genetic eye disorder. Understanding the natural progression of this condition can pave the way for future treatments by highlighting key areas that need intervention. Researchers are eager to identify potential biomarkers and disease progression patterns, which can lead to more targeted and effective therapies in the future. This foundational knowledge is essential for developing treatments that can slow or prevent vision loss in patients with this genetic mutation.

Who Is on the Research Team?

Sreenivasu Mudumba, PhD

Principal Investigator

PYC Therapeutics

Are You a Good Fit for This Trial?

Inclusion Criteria

Male or female, ≥ 10 years of age at baseline (Visit 2).

Have a clinical and molecular diagnosis of PRPF31 mutation-associated retinal dystrophy.

Are willing to comply with the instructions and attend all scheduled study visits.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

1 visit (in-person)

Baseline Assessment

Initial assessments including ophthalmic exams, imaging studies, and electrophysiological testing

1 visit

1 visit (in-person)

Follow-up

Participants are monitored for changes in visual function and retinal structure every 16 weeks for the first year and every 24 weeks for up to 4 years

4 years

Regular visits every 16-24 weeks

What Are the Treatments Tested in This Trial?

Interventions

• None

Who Is Running the Clinical Trial?

PYC Therapeutics

Lead Sponsor

Trials

7

Recruited

170+