VP-001 for Retinal Dystrophy

(DINGO Trial)

Recruiting at 5 trial locations

Overseen By Jessica Dunne

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: PYC Therapeutics

Must not be taking: Anti-VEGF, Corticosteroids, Ozurdex

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: 8

Duration: 24 months

16 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of VP-001, an experimental treatment for Retinal Dystrophy linked to a PRPF31 gene mutation. The trial examines various doses and schedules of VP-001 to assess its efficacy when administered multiple times directly into the eye. It targets individuals previously treated with VP-001 who have Retinitis Pigmentosa caused by the PRPF31 mutation. As a Phase 1, Phase 2 trial, this research aims to understand the treatment's function in people and measure its effectiveness in an initial, smaller group.

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 this trial's treatments are likely to be safe?

Research has shown that VP-001 has been generally well-tolerated in previous studies. One study found single injections of VP-001 at doses of 3 micrograms (µg) and 10 micrograms to be safe. Even a higher dose of 30 micrograms caused no major problems. Another report suggested that VP-001 appears safe for treating eye conditions.

These findings indicate that VP-001 is likely safe for humans. However, this trial involves higher doses and repeated treatments. Participants should consider these factors and discuss any concerns with the study team. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Unlike the standard treatments for retinal dystrophy, which often include gene therapies or vitamin A supplementation, VP-001 offers a unique approach by using an intravitreal (IVT) delivery method to administer a specific dosage of the active compound directly into the eye. Researchers are excited about VP-001 because it targets the retina more precisely, potentially leading to improved outcomes and fewer systemic side effects. Additionally, the varying dosages and frequency of administration being tested, such as 75ug or 120ug every 8 or 12 weeks, provide flexibility and could optimize treatment effectiveness for different patients. This innovative approach could significantly enhance how retinal dystrophy is managed, offering hope for better vision preservation.

What evidence suggests that this trial's treatments could be effective for Retinal Dystrophy?

Research has shown that VP-001 yields promising results for treating retinal dystrophy caused by the PRPF31 mutation. In earlier studies, patients experienced significant vision improvement, measured by logMAR scores, which assess vision clarity. The scores improved from about 20/47 to a better level, indicating clearer vision. The treatment also slowed the loss of photoreceptor cells, crucial for sight. Overall, these findings suggest that VP-001 could effectively treat retinal dystrophy linked to the PRPF31 mutation. ^{5 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for individuals with PRPF31 mutation-associated Retinal Dystrophy who have already been treated with VP-001. Specific eye conditions like fungal retinitis, Purtscher retinopathy, and other related diseases are the focus. Participants must meet certain health standards to be included.

Inclusion Criteria

I have Retinitis Pigmentosa due to a PRPF31 gene mutation.

Understand the language of the informed consent and are willing and able to provide written informed consent prior to any study procedures

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

Exclusion Criteria

I have had eye surgery within the last 3 months.

Have a recent history (<6 months) or current excessive recreational drug or alcohol use, in the opinion of the investigator

I've had Ozurdex, Retisert, or Iluvien implants within the specified time frames.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive repeat doses of VP-001 intravitreally, with dosing regimens of either 75ug or 120ug every 8 or 12 weeks

24 months

Every 8 or 12 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 study tests two doses of a drug called VP-001, given as an injection inside the eye (intravitreally). It's designed to see how safe and effective these doses are in treating specific types of inherited eye disorders.

How Is the Trial Designed?

Group I: Cohort 2: 75ug of VP-001 every 12 weeks Experimental Treatment 1 Intervention

Group II: Cohort 1: 30ug VP-001 every 8 weeks 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 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]

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]

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.

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 ...

3.

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 ...

4.

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, ...

5.

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 ...

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.

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 ...

VP-001 was quantitated by hybridization ELISA. شرح. VP-001 has PK and safety profiles favorable to an ocular therapeutic ... Molecular findings from 537 ...

8.

iovs.arvojournals.org

iovs.arvojournals.org/article.aspx?articleid=2797068

A Phase 1 First in Human Study of VP-001 - IOVS

Conclusions : A single intravitreal injection of VP-001 was safe and well tolerated at 3 µg and 10 µg doses. A 30-µg dose is also administered, and if shown ...

9.

fightingblindness.ca

fightingblindness.ca/news/2024-inherited-retinal-disease-research-updates/

2024 Inherited Retinal Disease Research Updates

PYC Therapeutics reported results from their Phase 1 PLATYPUS clinical trial for an RNA therapy, VP-001, for people with RP11, caused by ...