Retinitis Pigmentosa 11Phase 1NCT06455826
MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)
A Phase 1 study testing VP-001 in people with Retinitis Pigmentosa 11 and Retinal Degeneration, run by PYC Therapeutics at 5 sites in United States.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Retinitis Pigmentosa 11, Retinal Degeneration, Eye Diseases
- What is being tested?
- VP-001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 5 sitesUnited States
- Study dates
- 13 Jun 2024to 24 Sep 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female sex; ≥12 years of age at Baseline (Visit 2).
- Have a molecular (genetic) diagnosis of PRPF31 mutation.
- Have a clinical diagnosis of PRPF31 mutation-associated retinal dystrophy, that is, RP11. The following conditions are allowed for inclusion if due to RP11, if in the opinion of the investigator they will not interfere with study evaluations or have resolved: macular edema (intraretinal, sub-retinal or other fluid) requiring regular treatment at a frequency of less than every 6 weeks; macular edema must be stable for at least 3 months prior to Screening (Visit 1). The investigator must consult with the study Medical Monitor.
- If ≥18 years of age, understand the language of the informed consent and are willing and able to provide written informed consent prior to any study procedures. If a minor (12 to <18 years of age), a parent or legal guardian willing and able to provide written permission for the minor's participation prior to performing any study related procedures and pediatric participant able to provide age appropriate assent for study participation.
- If ≥18 years of age, are willing to comply with the instructions and attend all scheduled study visits. If a minor (12 to <18 years of age), able to complete all study assessments, comply with the protocol, and has a parent or caregiver willing and able to follow study instructions and attend study visits with the participant as required, in the opinion of the Investigator.
- Meets ≥1 of the following for visual function in the study eye:
Show all 12Show fewer
- V4e visual field >1000 deg2, per kinetic perimetry
- <Mean microperimetry threshold: >5 decibel (dB) to <15dB
- Visual acuity: 20/40 to 20/200 inclusive (>35 and <70 letters by Early-Treatment Diabetic Retinopathy Study [ETDRS])
- Ellipsoid zone (EZ) length >1000 microns, of which 500 microns is contiguous, by SD-OCT
- FST baseline no worse than -20 dB
- Participants of childbearing potential and male participants must not be pregnant or lactating and must be sexually inactive by abstinence, which is consistent with the preferred and usual lifestyle of the participant or agree to use adequate birth control throughout study duration. Adequate birth control is defined as hormonal - oral, implantable, injectable, or transdermal contraceptives; mechanical - spermicide in conjunction with a barrier such as a condom or diaphragm; intrauterine device (IUD); or surgical sterilization of partner. For nonsexually active participants, abstinence may be regarded as an adequate method of birth control. Participants of childbearing potential include all participants who have experienced menarche and have not undergone successful surgical sterilization (bilateral tubal ligation, hysterectomy, or bilateral oophorectomy) or are not postmenopausal (12 months after last menses).
Who cannot join
- Have any uncontrolled systemic disease that, in the opinion of the Investigator, would preclude participation in the study that include but are not limited to infection, uncontrolled elevated blood pressure, cardiovascular disease, or glycemic control issues, or any other medical condition that may put the participant at risk due to study procedures.
- Mutations in genes that cause autosomal dominant RP, Xlinked RP, or presence of biallelic mutations in autosomal recessive RP/retinal dystrophy genes other than PRPF31 mutations.
- Have used anti-vascular endothelial growth factor (VEGF) agents within 2 months or corticosteroid injections within the last 3 months.
- Have had Ozurdex
- implants placed within 3 months or Retisert
- or Iluvien
Show all 14Show fewer
- implants placed within 3 years prior to Baseline (Visit 2).
- Within 3 months prior to Baseline (Visit 2), have undergone any vitreoretinal surgery (scleral buckle, pars plana vitrectomy, retrieval of a dropped nucleus or intraocular lens, radial optic neurotomy, sheathotomy, cyclodestructive procedures or multiple filtration surgeries [2 or more]) or any other ocular surgery.
- Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmic evaluation or photography, as assessed by the investigator.
- Have used any investigational drug or device within 90 days or 5 estimated half-lives of Baseline (Visit 2), whichever is longer, or plan to participate in another study of drug or device during the study period. Participation in observational trials is allowable based on investigator discretion and consultation with the Medical Monitor. It is assumed that the observational trial evaluations would not interfere with participation in this study.
- Have received any prior cell, ribonucleic acid (RNA) (including VP-001), or gene therapy for a retinal condition.
- Have a recent history (<6 months) or current excessive recreational drug or alcohol use, in the opinion of the investigator.
- Any retinal pathology other than RP11 that in the investigator's opinion could affect study results.
- Participants should not have any conditions, in the investigator's opinion, that may put the participant at increased risk, confound study data, or interfere significantly with the participant's study participation.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, 48105
Retina Foundation of the Southwest
Dallas, Texas, 75321
Baylor College of Medicine- Alkek Eye Center
Houston, Texas, 77030
University of Florida Health
Jacksonville, Florida, 32209
Oregon Health and Science University - Casey Eye Institute
Portland, Oregon, 97239
About this study
Official title: 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
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
- Conditions
- Retinitis Pigmentosa 11, Retinal Degeneration, Eye Diseases, Retinal Disease, Retinal Dystrophies
- Being tested
- VP-001
- Medical subject headings
- Retinitis Pigmentosa 11, Retinal Degeneration, Eye Diseases, Retinal Diseases, Retinal Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VP-001Drug
A Phase 1 Open-Label, Multiple Ascending Dose Study of VP-001 in Participants with Confirmed PRPF31 Mutation- Associated Retinal Dystrophy
| Study group | Role | What they receive |
|---|---|---|
| Multi-ascending dose escalation study of VP-001 | Experimental | Not described Receives: Drug: VP-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence, severity, and relatedness of treatment-emergent ocular adverse events and treatment-emergent serious adverse events
Measured: over a 4-week post-dose period
The incidence, severity, and relatedness of treatment-emergent ocular adverse events
Measured: over a 52-week period
Secondary outcomes (15)
Adverse Events and Treatment Emergent serious adverse events (TESAEs) in the fellow eye eye
Measured: over a 4-week post-dose time period
Adverse Events and Treatment Emergent serious adverse events (TESAEs) in the fellow eye eye
Measured: over a 52-week period
Incidence, severity, and relatedness of non-ocular Treatment Emergent adverse events (TEAEs)
Measured: over a 4-week post-dose time period
Incidence, severity, and relatedness of non-ocular Treatment Emergent adverse events (TEAEs)
Measured: over a 52-week period
BCVA letter score using ETDRS charts
Measured: over a 52-week period
Change in lowest passing light level using Ora- VNC™ mobility test
Measured: over a 52-week period
Low luminance visual acuity (LLVA) letter score
Measured: over a 52-week period
Visual field sensitivity as measured by static perimetry with topographic analysis (Hill of Vision)
Measured: over a 52-week period
Mean retinal sensitivity as measured by fundusguided microperimetry
Measured: over a 52-week period
Visual fields as measured by kinetic perimetry utilizing I4e, II4e and V4e stimuli
Measured: over a 52-week period
Rod-and cone-mediated retinal function as measured by white, red and blue FST
Measured: over a 52-week period
Retinal thickness on SD-OCT, including retinal thickness in each ETDRS subfield and EZ area and volume
Measured: over a 52-week period
Retinal function using full-field electroretinography (ERG)
Measured: over a 52-week period
Area of hypo-autofluorescence captured by FAF
Measured: over a 52-week period
Abnormalities captured by wide-field fundus photography
Measured: over a 52-week period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2024
- Study start13 Jun 2024
- Primary completion24 Sep 2025
- Study completion24 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis Pigmentosa 11Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Participants will be assigned sequentially to the VP-001 escalation dose (30 μg and 75 μg) groups. Participants will receive three repeat IVT injections of VP-001, 8 weeks apart, in the study eye.
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- PYC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06455826
- Sponsor's protocol ID
- VP001-CL102
- Status last verified
- May 2026
- First submitted
- 3 Jun 2024
- Last updated
- 29 May 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06455826
- Acronym / sponsor ID
- VP001-CL102
- Conditions
- Retinitis Pigmentosa 11, Retinal Degeneration, Eye Diseases, Retinal Disease, Retinal Dystrophies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- PYC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 13 Jun 2024 (actual)
- Primary completion
- 24 Sep 2025 (actual)
- Study completion
- 24 Sep 2025 (actual)
- First posted
- 12 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Participants will be assigned sequentially to the VP-001 escalation dose (30 μg and 75 μg) groups. Participants will receive three repeat IVT injections of VP-001, 8 weeks apart, in the study eye.
| Arm | Type | Receives |
|---|---|---|
| Multi-ascending dose escalation study of VP-001 | Experimental | Drug: VP-001 |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Male or female sex; ≥12 years of age at Baseline (Visit 2).
- Have a molecular (genetic) diagnosis of PRPF31 mutation.
- Have a clinical diagnosis of PRPF31 mutation-associated retinal dystrophy, that is, RP11. The following conditions are allowed for inclusion if due to RP11, if in the opinion of the investigator they will not interfere with study evaluations or have resolved: macular edema (intraretinal, sub-retinal or other fluid) requiring regular treatment at a frequency of less than every 6 weeks; macular edema must be stable for at least 3 months prior to Screening (Visit 1). The investigator must consult with the study Medical Monitor.
- If ≥18 years of age, understand the language of the informed consent and are willing and able to provide written informed consent prior to any study procedures. If a minor (12 to <18 years of age), a parent or legal guardian willing and able to provide written permission for the minor's participation prior to performing any study related procedures and pediatric participant able to provide age appropriate assent for study participation.
- If ≥18 years of age, are willing to comply with the instructions and attend all scheduled study visits. If a minor (12 to <18 years of age), able to complete all study assessments, comply with the protocol, and has a parent or caregiver willing and able to follow study instructions and attend study visits with the participant as required, in the opinion of the Investigator.
Key exclusion criteria (14 listed)
- Have any uncontrolled systemic disease that, in the opinion of the Investigator, would preclude participation in the study that include but are not limited to infection, uncontrolled elevated blood pressure, cardiovascular disease, or glycemic control issues, or any other medical condition that may put the participant at risk due to study procedures.
- Mutations in genes that cause autosomal dominant RP, Xlinked RP, or presence of biallelic mutations in autosomal recessive RP/retinal dystrophy genes other than PRPF31 mutations.
- Have used anti-vascular endothelial growth factor (VEGF) agents within 2 months or corticosteroid injections within the last 3 months.
- Have had Ozurdex
- Within 3 months prior to Baseline (Visit 2), have undergone any vitreoretinal surgery (scleral buckle, pars plana vitrectomy, retrieval of a dropped nucleus or intraocular lens, radial optic neurotomy, sheathotomy, cyclodestructive procedures or multiple filtration surgeries [2 or more]) or any other ocular surgery.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VP-001A Phase 1 Open-Label, Multiple Ascending Dose Study of VP-001 in Participants with Confirmed PRPF31 Mutation- Associated Retinal Dystrophy | Drug | Multi-ascending dose escalation study of VP-001 |
Primary outcome
The incidence, severity, and relatedness of treatment-emergent ocular adverse events and treatment-emergent serious adverse events
Time frame: over a 4-week post-dose period
The incidence, severity, and relatedness of treatment-emergent ocular adverse events
Time frame: over a 52-week period
15 secondary outcomes
Adverse Events and Treatment Emergent serious adverse events (TESAEs) in the fellow eye eye
Time frame: over a 4-week post-dose time period
Adverse Events and Treatment Emergent serious adverse events (TESAEs) in the fellow eye eye
Time frame: over a 52-week period
Incidence, severity, and relatedness of non-ocular Treatment Emergent adverse events (TEAEs)
Time frame: over a 4-week post-dose time period
Incidence, severity, and relatedness of non-ocular Treatment Emergent adverse events (TEAEs)
Time frame: over a 52-week period
BCVA letter score using ETDRS charts
Time frame: over a 52-week period
Change in lowest passing light level using Ora- VNC™ mobility test
Time frame: over a 52-week period
Low luminance visual acuity (LLVA) letter score
Time frame: over a 52-week period
Visual field sensitivity as measured by static perimetry with topographic analysis (Hill of Vision)
Time frame: over a 52-week period
Mean retinal sensitivity as measured by fundusguided microperimetry
Time frame: over a 52-week period
Visual fields as measured by kinetic perimetry utilizing I4e, II4e and V4e stimuli
Time frame: over a 52-week period
Rod-and cone-mediated retinal function as measured by white, red and blue FST
Time frame: over a 52-week period
Retinal thickness on SD-OCT, including retinal thickness in each ETDRS subfield and EZ area and volume
Time frame: over a 52-week period
Retinal function using full-field electroretinography (ERG)
Time frame: over a 52-week period
Area of hypo-autofluorescence captured by FAF
Time frame: over a 52-week period
Abnormalities captured by wide-field fundus photography
Time frame: over a 52-week period
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VP-001 in people with Retinitis Pigmentosa 11 and Retinal Degeneration, run by PYC Therapeutics at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence, severity, and relatedness of treatment-emergent ocular adverse events and treatment-emergent serious adverse events (time frame: over a 4-week post-dose period). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 May 2026; AzTrials last refreshed its copy on 8 Nov 2026.