VP-001 for Retinal Dystrophy

Recruiting at 4 trial locations

Overseen By Ora Inc

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: PYC Therapeutics

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

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

12 Participants Needed

Trial Summary

What is the purpose of this trial?

A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy

Eligibility Criteria

This trial is for individuals with confirmed PRPF31 mutation-associated retinal dystrophy, a genetic disorder causing vision loss. Details on specific inclusion and exclusion criteria are not provided, but typically participants must meet certain health standards and not have conditions that could interfere with the study.

Inclusion Criteria

I am at least 12 years old.

I have a PRPF31 genetic mutation.

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

Exclusion Criteria

I do not have any uncontrolled illnesses that would stop me from joining the study.

Recent excessive recreational drug or alcohol use

I have recently used eye treatments.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple ascending doses of intravitreally administered VP-001

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• VP-001

Trial Overview The trial is testing multiple increasing doses of VP-001 given through an injection into the eye (intravitreally) to assess its safety and how well it's tolerated in patients with retinal dystrophy due to PRPF31 mutations.

Participant Groups

Group I: Multi-ascending dose escalation study of VP-001 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

PYC Therapeutics

Lead Sponsor

Trials

7

Recruited

170+