a study of two doses of vp-001 administered intravitreally in participants with confirmed prpf31 mutation-associated retinal dystrophy previously treated with vp001

Conditions: Retinitis Pigmentosa 11; Retinal Degeneration; Retinal Disease; Eye Diseases Hereditary; Retinal Dystrophies

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.

Keywords: vp-001-cl103