RecruitingPhase 1/2

A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001

United States16 participantsStarted 2025-06-01

Plain-language summary

This is a Phase 1/2 repeat-dose, open-label, two-arm, parallel group safety and efficacy study of two doses of VP-001 (30 μg and 75 μg) in participants with confirmed PRPF31 mutation-associated retinal dystrophy, including participants previously treated with VP001 in the PLATYPUS Study or WALLABY Study for a minimum of 8 weeks.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Mean microperimetry threshold: \>5 decibel (dB) to \<15 dB
✓. Ellipsoid zone (EZ) length \>1000 microns of which 500 microns is contiguous, by SD-OCT
✓. In the opinion of the Investigator, rod function is observed in any direction \>10 degrees per static perimetry at Screening Visit (Visit 1)

What they're measuring

To determine the safety of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy.

Timeframe: 26 months

Trial details

NCT IDNCT06852963

SponsorPYC Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-01-30

Contact for this trial

Ora Inc

1-510-423-2680 VP001@oraclinical.com

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