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Virus Therapy
VP-001 for Retinal Dystrophy (Platypus Trial)
Phase 1
Recruiting
Research Sponsored by PYC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up over a 48-week time period
Awards & highlights
Platypus Trial Summary
This trial tests a drug to see if it can safely help people with a genetic eye condition called retinal dystrophy.
Who is the study for?
Adults with a genetic diagnosis of PRPF31 mutation-associated retinal dystrophy (RP11) can join this trial. They must be willing to follow the study plan and attend all visits, use effective birth control if they can have children, and not be pregnant or breastfeeding. People with uncontrolled diseases, recent certain eye treatments or surgeries, other gene therapy for retinal conditions, or excessive drug/alcohol use cannot participate.Check my eligibility
What is being tested?
The trial is testing VP-001 given as an injection into the eye to see if it's safe for people with RP11-related vision loss. It's an early-phase study where everyone gets the treatment but at different doses to find out which one is safest.See study design
What are the potential side effects?
Since this is a Phase 1 trial primarily focused on safety and tolerability, specific side effects are being investigated but may include typical reactions related to eye injections such as discomfort, redness, swelling around the eyes and potential visual disturbances.
Platypus Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ over a 48-week time period
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~over a 48-week time period
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
The incidence, severity, and relatedness of treatment-emergent ocular adverse events (TEAEs) and treatment-emergent serious adverse events (TE-SAEs) in the study eye
Secondary outcome measures
Adverse Events and Treatment Emergent Serious adverse events (SAEs) in the fellow eye
Adverse Events and Treatment Emergent serious adverse events (SAEs) in the fellow eye
Incidence, severity relatedness and of non-ocular TEAEs
Other outcome measures
Change from Baseline in Abnormalities captured by wide-field fundus photography
Change from Baseline in Area of hypo-autofluorescence captured by FAF
Change from Baseline in Best-corrected visual acuity (BCVA) letter score using Early Treatment Diabetic Retinopathy Study (ETDRS) charts
+9 morePlatypus Trial Design
1Treatment groups
Experimental Treatment
Group I: Single arm dose escalation study of VP-001Experimental Treatment1 Intervention
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Who is running the clinical trial?
PYC TherapeuticsLead Sponsor
2 Previous Clinical Trials
90 Total Patients Enrolled
Sreenivasu MudumbaStudy ChairPYC
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have cloudiness or poor dilation in my eye's pupil.I have recently used anti-VEGF treatments or had corticosteroid injections or implants.My eye condition is not RP11.I have a genetic mutation in PRPF31.I do not have any uncontrolled illnesses that would stop me from joining the study.My retinal disease is due to specific genetic mutations, not including PRPF31.I have had recent eye surgery.I am 18 years old or older.I have not used any experimental drugs or devices recently.I am not pregnant or breastfeeding and will not become pregnant or father a child during the study.I have RP11-related eye disease but my macular edema is stable.I have PRPF31-related eye disease with stable swelling in my macula, if any.I have had cell or gene therapy for an eye condition before.
Research Study Groups:
This trial has the following groups:- Group 1: Single arm dose escalation study of VP-001
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Retinal Dystrophy Patient Testimony for trial: Trial Name: NCT05902962 — Phase 1
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any remaining opportunities to participate in this trial?
"Affirmative. The clinical trial is actively registering patients in accordance with the information hosted on clinicaltrials.gov, which was first published on April 20th of 2023 and most recently modified on June 5th of that same year. For this research endeavour, only 20 participants are needed from a single recruitment site."
Answered by AI
Is there any evidence to suggest that Single arm dose escalation study of VP-001 may be detrimental for individuals?
"Single arm dose escalation study of VP-001 has limited data to support its efficacy and safety, thus receiving a rating of 1 on our scale."
Answered by AI
How many people can take part in this trial at its peak?
"Yes, the data posted on clinicaltrials.gov certifies that this medical experiment is in progress and recruiting patients. This research was first announced on April 20th of 2023 and has been recently revised as of June 5th, 2023; they are currently looking for a total of twenty participants from one location."
Answered by AI
Who else is applying?
What state do they live in?
Texas
Puerto Rico
What site did they apply to?
Bascom Palmer Eye Institute University of Miami
Baylor College of Medicine- Alkek Eye Center
Retina Foundation of the Southwest
What portion of applicants met pre-screening criteria?
Did not meet criteria
How many prior treatments have patients received?
0
Why did patients apply to this trial?
I live in McKinney Dallas TX. I have tried several medications, and it didn't work.
PatientReceived 1 prior treatment
I am Edgar Babilonia disabled veteran with hope of preventing loosing my eyesight completely and hope that i can be in your studies and trial.
PatientReceived no prior treatments
I have tried several different medications and drop. It seam it is not working. I am hoping through this I would gain some vision or slow the process down.
PatientReceived 2+ prior treatments
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