Thinking of joining a study?

Register your interest

Become a volunteer?

NCT06852963 | NOT YET RECRUITING | Retinitis Pigmentosa 11

A Study of Two Doses of VP-001 Administered Intravitreally in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy Previously Treated with VP001
Sponsor:

PYC Therapeutics

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.

Condition or disease

Retinitis Pigmentosa 11

Retinal Degeneration

Retinal Disease

Eye Diseases Hereditary

Retinal Dystrophies

Intervention/treatment

VP-001

Phase

PHASE1

PHASE2

Study Type : INTERVENTIONAL
Estimated Enrollment : 12 participants
Masking : NONE
Primary Purpose : TREATMENT
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
Actual Study Start Date : 2025-06-01
Estimated Primary Completion Date : 2027-06-01
Estimated Study Completion Date : 2027-08-01

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 18 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
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.
A Study of Two Doses of VP-001 Administered Intravitreally in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy Previously Treated with VP001

Location Details

NCT06852963

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Not yet recruiting

United States, Florida

University of Florida College of Medicine

Jacksonville, Florida, United States, 32209

Not yet recruiting

United States, Florida

Bascom Palmer Eye Institute - University of Miami

Miami, Florida, United States, 33136

Not yet recruiting

United States, Road cancer

Kellogg Eye Center - University of Michigan

Ann Arbor, Road cancer, United States, 48105

Not yet recruiting

United States, Oregon

Casey Eye Institute - OHSU

Portland, Oregon, United States, 97239

Not yet recruiting

United States, Texas

Retina Foundation of the Southwest

Dallas, Texas, United States, 75231

Not yet recruiting

United States, Texas

Baylor College of Medicine

Houston, Texas, United States, 77030