VP-001 for Retinal Dystrophy
(Platypus Trial)

Recruiting in Palo Alto (17 mi)

+5 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: PYC Therapeutics

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial tests a new eye injection treatment called VP-001 for people with a genetic condition that affects their vision. The goal is to see if it is safe and can help the damaged cells in their eyes.

Eligibility Criteria

Adults with a genetic diagnosis of PRPF31 mutation-associated retinal dystrophy (RP11) can join this trial. They must be willing to follow the study plan and attend all visits, use effective birth control if they can have children, and not be pregnant or breastfeeding. People with uncontrolled diseases, recent certain eye treatments or surgeries, other gene therapy for retinal conditions, or excessive drug/alcohol use cannot participate.

Inclusion Criteria

Have light perception (LP) or better vision in the study eye

Understand the language of the informed consent and willing to provide written informed consent

I have a genetic mutation in PRPF31.

+3 more

Exclusion Criteria

I have cloudiness or poor dilation in my eye's pupil.

Conditions that may put the participant at increased risk or interfere with study participation

I have recently used anti-VEGF treatments or had corticosteroid injections or implants.

+7 more

Participant Groups

The trial is testing VP-001 given as an injection into the eye to see if it's safe for people with RP11-related vision loss. It's an early-phase study where everyone gets the treatment but at different doses to find out which one is safest.

1Treatment groups

Experimental Treatment

Group I: Single arm dose escalation study of VP-001Experimental Treatment1 Intervention