A Phase 1/2 interventional study of VP-001 in Retinitis Pigmentosa 11, Retinal Degeneration and Retinal Disease, sponsored by PYC Therapeutics. Active, not recruiting at 6 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-05-29.
Sponsored by PYC Therapeutics · Phase 1/2, Interventional, and Treatment
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.
60 studies on the registry are indexed under Retinal Dystrophies; 20 are open to participants now.
This study's enrollment of 17 is close to the median of 18 across 34 interventional studies indexed under Retinal Dystrophies.
Browse Retinal Dystrophies studies →PYC Therapeutics is the lead sponsor of 8 studies on the registry; 2 are open to participants now.
Compared within Retinal Dystrophies, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Retinal Degeneration. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Key Inclusion Criteria:
For participants not previously enrolled in VP001-CL101 or VP001-CL102 studies: Meet all of the following for visual function in the study eye at the Screening Visit:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: VP-001
Drug: VP-001
VP-001 is an oligonucleotide-peptide conjugate administered intravitreally.
To determine the safety of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy.
The incidence, severity, and relatedness of ocular TEAEs and TE-SAEs in the study eye over a 26-month time period for each of the repeat doses
Time frame: 26 months
To determine the change from Baseline (Visit 2) through End of Study/Early Termination in BCVA letter score using ETDRS charts
To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. A gain of Early Treatment Diabetic Retinopathy Study (ETDRS) letters in Best-Corrected Visual Acuity (BCVA) from Baseline through EOS/ET would indicate vision improvement.
Time frame: 24 months
To determine the change from baseline (Visit 2) through End of Study/Early Termination in Low Luminance Visual Acuity (LLVA) letter score
To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11.
Time frame: 24 months
Change from Baseline (Visit 2) through End of Study/Early Termination in visual field sensitivity, Mean deviation (Mean Defect) as measured by standard static perimetry (Humphries)
To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11.
Time frame: 24 months
Change from Baseline (Visit 2) through End of Study/Early Termination in mean retinal sensitivity
To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11. \> mean retinal sensitivity will be measured by fundus-guided microperimetry, measured in dB (Decibels).
Time frame: 24 months
Change from Baseline (Visit 2) through End of Study/Early Termination in preserved EZ area on SD-OCT
SD-OCT refers to Spectral Domain Optical Coherence Tomography. A preserved EZ area refers to the region of the retina where the Ellipsoid Zone (EZ), a distinct hyperreflective band representing the inner segments of photoreceptor cells, remains intact and visible, indicating relatively healthy photoreceptor function, often measured in a specific area on the scan to monitor disease progression in conditions like retinitis pigmentosa. To determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11.
Time frame: 24 months
Change from Baseline (Visit 2) through End of Study/Early Termination in participant reported outcome measures utilizing the Michigan Retinal Degeneration Questionnaire (MRDQ)
The Michigan Retinal Degeneration Questionnaire (MRDQ) is a psychometrically validated patient reported outcomes (PRO) measure for patients with inherited retinal degenerations. This is to determine the efficacy of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy, RP11.
Time frame: 24 months
Plan to share: No
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This study is active, not recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: VP-001
PYC Therapeutics