Clinical trial NCT06852963: A Study of Two Doses of VP-001 Administered Intravitreally in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy Previously Treated with VP001
Official title: A Repeat-Dose, Open-Label, Four Arm Safety and Efficacy Study of Two Doses of VP-001 (75ug and 120ug) Administered Intravitreally in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy and Previously Treated with VP001 in the PLATYPUS Study (Protocol# VP001-101) or WALLABY Study (Protocol #VP001-102) for a Minimum of 8 Weeks
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Retinitis Pigmentosa 11
Retinal Degeneration
Retinal Disease
Eye Diseases Hereditary
Retinal Dystrophies
Keywords: VP-001-CL103
Lead sponsor: PYC Therapeutics
Sponsor class: INDUSTRY
First posted: 2025-02-28
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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 mutation-associated Retinal Dystrophy and previously treated with VP001.


Eligibility criteria: Inclusion Criteria:

* Male or female sex; ≥18 years of age at Day 1/Baseline (Visit 2)
* 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
* Must have been previously enrolled in PLATYPUS Part B (Protocol #VP001-CL101) or WALLABY (Protocol #VP001-CL102) study. At Screening Visit in this study, participants must have completed at least 8 weeks after last study agent administration in PLATYPUS Part B (Protocol #VP001-CL101) or WALLABY (Protocol # VP001-CL102) study
* Have a confirmed clinical diagnosis of Retinitis Pigmentosa.
* Have a confirmed genetic diagnosis of Retinitis Pigmentosa secondary to mutation in the PRPF31 gene.
* 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 non-sexually 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 post-menopausal (12 months after last menses).

Exclusion Criteria:

* 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.
* Known mutations in genes that cause autosomal dominant RP, X-linked RP, or presence of biallelic mutations in autosomal recessive RP/retinal dystrophy genes other than PRPF31 mutations.
* Have used anti-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® 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.
* 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; IUD; or surgical sterilization of partner. For non-sexually 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 post-menopausal (12 months after last menses).
* 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.

Primary outcomes:
measure: To determine the safety of two doses and two dosing regimens of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy
timeFrame: 24 months
Secondary outcomes:
measure: To determine the change from Baseline (Visit 2) through End of Study/Early Termination in BCVA letter score using ETDRS charts
timeFrame: 24 months
measure: To determine the change from baseline (Visit 2) through End of Study/Early Termination in Low Luminance Visual Acuity letter score
timeFrame: 24 months
measure: Change from Baseline (Visit 2) through End of Study/Early Termination in visual field sensitivity
timeFrame: 24 months
measure: Change from Baseline (Visit 2) through End of Study/Early Termination in mean retinal sensitivity
timeFrame: 24 months
measure: Change from Baseline (Visit 2) through End of Study/Early Termination in preserved EZ area on SD-OCT
timeFrame: 24 months
measure: Change from Baseline (Visit 2) through End of Study/Early Termination in participant reported outcome measures utilizing the Michigan Retinal Degeneration Questionnaire (MRDQ)
timeFrame: 24 months
Other outcomes:
Locations:
city: Jacksonville
country: United States
facility: University of Florida College of Medicine
state: Florida
city: Miami
country: United States
facility: Bascom Palmer Eye Institute - University of Miami
state: Florida
city: Ann Arbor
country: United States
facility: Kellogg Eye Center - University of Michigan
state: Michigan
city: Portland
country: United States
facility: Casey Eye Institute - OHSU
state: Oregon
city: Dallas
country: United States
facility: Retina Foundation of the Southwest
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
Study officials:
References: