VP-001 for Retinal Dystrophy

(Platypus Trial)

Not currently recruiting at 5 trial locations

Overseen By Clare Guerrero

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: PYC Therapeutics

Must not be taking: Anti-VEGF, Corticosteroid injections

Disqualifiers: Uncontrolled systemic disease, Other RP mutations, Recent ocular surgery, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 6-8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called VP-001, a potential drug for retinal dystrophy caused by a PRPF31 gene mutation. The main goal is to determine if VP-001 is safe and tolerable for individuals with this genetic form of vision loss. Participants must have a diagnosis of this condition, which affects sight, and retain some vision in at least one eye. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you cannot have used anti-VEGF agents within 2 months or corticosteroid injections within the last 3 months before starting the trial.

Is there any evidence suggesting that VP-001 is likely to be safe for humans?

Studies have shown that VP-001, administered as an eye injection, is generally safe. In earlier tests, participants did not experience major side effects. Some reported mild discomfort, such as redness or irritation, but these effects were temporary. Research indicates that the treatment is well-tolerated. Although this study is in the early stages with limited data, the main goal is to ensure the treatment's safety. Prospective participants should remember that the primary focus is on assessing safety first. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

VP-001 is unique because it offers a new approach for treating retinal dystrophy, potentially addressing the root causes of the condition rather than just managing symptoms. Unlike current treatments that may focus on slowing disease progression, VP-001 targets specific genetic factors involved in retinal degeneration, which could lead to more effective and lasting results. Researchers are excited about VP-001 because it could represent a significant advancement in precision medicine for eye disorders, offering hope for improved vision outcomes for patients with retinal dystrophy.

What evidence suggests that VP-001 might be an effective treatment for retinal dystrophy?

Research shows that VP-001 can improve vision in people with an eye condition caused by the PRPF31 gene mutation. Previous studies have found that this treatment makes it easier for patients to see clearly. In one study, vision scores improved from about 20/47 to a better score. This suggests that VP-001 might slow down or even reverse some vision loss related to this condition. While more research is needed, these early results are promising and suggest that VP-001 could be effective. ^{2 5 6 7 8}

Who Is on the Research Team?

Sreenivasu Mudumba

Principal Investigator

PYC

Are You a Good Fit for This Trial?

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.

Exclusion Criteria

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

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

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intravitreal administration of VP-001 in a dose escalation manner to evaluate safety and tolerability

6-8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VP-001

Trial Overview

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.

How Is the Trial Designed?

Group I: Single arm dose escalation study of VP-001 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

PYC Therapeutics

Lead Sponsor

Trials

7

Recruited

170+

Published Research Related to This Trial

In a study of 24 patients with retinitis pigmentosa treated with valproic acid (VPA) for an average of 9.4 months, there was no significant improvement in best-corrected visual acuity (BCVA) or visual field analyses after treatment.

VPA treatment was associated with a decline in certain electroretinography (ERG) parameters, indicating potential negative effects, leading to the recommendation that VPA should not be prescribed for retinitis pigmentosa until further safety and efficacy evaluations are conducted.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The adverse effects of valproic acid on visual functions in the treatment of retinitis pigmentosa. Totan, Y., Güler, E., Yüce, A., et al.[2018]

In a follow-up phase 1 trial involving 11 patients aged 11-46 years, the subretinal injection of AAV2-hRPE65v2 into the contralateral eye showed no serious adverse events related to the treatment, indicating a favorable safety profile.

Most patients experienced significant improvements in mobility and light sensitivity in the newly treated eye, with effects lasting up to 3 years, demonstrating the efficacy of AAV2-hRPE65v2 as a gene therapy for inherited retinal dystrophy caused by RPE65 mutations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Bennett, J., Wellman, J., Marshall, KA., et al.[2022]

In a study of 13 eyes treated with valproic acid (VPA) for an average of 4 months, 9 eyes showed improved visual fields, resulting in a statistically significant average increase of 11% compared to baseline (p<0.02).

The treatment also led to a significant improvement in visual acuity, with an average decrease in logMAR scores indicating a change from approximately 20/47 to 20/32 (p<0.02), and side effects were mild and well tolerated.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Therapeutic potential of valproic acid for retinitis pigmentosa. Clemson, CM., Tzekov, R., Krebs, M., et al.[2022]

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06852963?cond=(RETINITIS%20PIGMENTOSA)%20OR%20(PRPF31)&rank=6

NCT06852963 | A Study of Two Doses of VP-001 ...

This is a repeat-dose, open-label, four arm safety and efficacy study of two doses of VP-001 administered intravitreally in participants with confirmed PRPF31 ...

2.

connect.careboxhealth.com

connect.careboxhealth.com/en-US/trial/listing/579679

A Study of Two Doses of VP-001 Administered Intravitreally in ...

This is a repeat-dose, open-label, four arm safety and efficacy study of two doses of VP-001 administered intravitreally in participants with confirmed PRPF31 ...

3.

withpower.com

withpower.com/trial/phase-2-eye-diseases-5-2025-9e0ef

VP-001 for Retinal Dystrophy (DINGO Trial)

The treatment also led to a significant improvement in visual acuity, with an average decrease in logMAR scores indicating a change from approximately 20/47 to ...

4.

ctv.veeva.com

ctv.veeva.com/study/a-study-of-two-doses-of-vp-001-administered-intravitreally-in-participants-with-confirmed-prpf31-mut

A Study of Two Doses of VP-001 Administered Intravitreally in ...

This is a repeat-dose, open-label, four arm safety and efficacy study of two doses of VP-001 administered intravitreally in participants with confirmed PRPF31 ...

5.

nacuity.com

nacuity.com/?page_id=624

Nacuity Pharmaceuticals Announces Positive Data from ...

NPI-001 shows more than 50% reduction in photoreceptor loss caused by RP associated with USH over two years. NPI-001 was well tolerated, ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06455826

NCT06455826 | MAD of IVT VP-001 in PRPF31 Mutation- ...

A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with ...

7.

withpower.com

withpower.com/trial/phase-1-retinal-dystrophies-3-2023-81be4

VP-001 for Retinal Dystrophy (Platypus Trial)

These studies generally focus on both the effectiveness and safety of the treatment, indicating that safety data is available for this condition.

8.

pyctx.com

pyctx.com/wp-content/uploads/VP-001-poster-TIDES_FINAL_v2GM-A4-1.pdf

VP-001 as an interventional therapy for patients with ...

In vitro studies were performed on 45-day old ROS after a single treatment of VP-001, followed by half media changes every 2-3 days for 28 days. PRPF31 protein ...