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Virus Therapy
Gene Therapy for Retinitis Pigmentosa (HORIZON Trial)
Phase 1 & 2
Waitlist Available
Research Sponsored by Beacon Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Male subjects with a documented RPGR mutation
Clinical diagnosis of X-linked retinitis pigmentosa (XLRP)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 0 - month 36
Awards & highlights
HORIZON Trial Summary
This trial will test a new gene therapy for X-linked retinitis pigmentosa, a degenerative eye disease.
Who is the study for?
This trial is for males aged 6-50 with X-linked retinitis pigmentosa (XLRP) due to RPGR gene mutations. Participants must have a certain level of visual acuity, not better than 20/32 and no worse than 20/200. They should not have other retinal diseases or previous AAV gene therapy treatments.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of rAAV2tYF-GRK1-RPGR, a genetic treatment delivered via a virus vector, in patients with XLRP caused by specific RPGR mutations.See study design
What are the potential side effects?
While the side effects are not specified here, similar gene therapies can cause immune reactions, eye irritation or discomfort, changes in vision, headache or mild pain.
HORIZON Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am a male with a confirmed RPGR mutation.
Select...
I have been diagnosed with X-linked retinitis pigmentosa.
HORIZON Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ day 0 - month 36
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 0 - month 36
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number and proportion of Adverse Events
Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters.
Secondary outcome measures
Change from baseline in full-field light sensitivity threshold (FST)
Change from baseline in fundus imaging
Change from baseline in visual function by light-adapted perimetry
+5 moreOther outcome measures
Phase 1/2 Dose Escalation: Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters
Phase 1/2 Dose Escalation: Number and proportion of treatment-emergent adverse events
Phase 2 Dose Expansion: Overall safety evaluation
HORIZON Trial Design
5Treatment groups
Experimental Treatment
Group I: Group 6 Phase 1/2 Dose EscalationExperimental Treatment1 Intervention
Male subjects at least 18 y/o treated with Dose 6 of rAAV2tYF-GRK1-RPGR study drug.
Group II: Group 5 Phase 1/2 Dose EscalationExperimental Treatment1 Intervention
Male subjects at least 18 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug.
Group III: Group 3 and Group 4 Phase 1/2 Dose EscalationExperimental Treatment1 Intervention
Group 3 male subjects at least 18 y/o and Group 4 male subjects at least 6 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
Group IV: Group 2: Phase 1/2 Dose EscalationExperimental Treatment1 Intervention
Male subjects at least 18 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug.
Group V: Group 1: Phase 1/2 Dose EscalationExperimental Treatment1 Intervention
Male subjects at least 18 y/o treated with Dose 1 of rAAV2tYF-GRK1-RPGR study drug.
Find a Location
Who is running the clinical trial?
Beacon TherapeuticsLead Sponsor
3 Previous Clinical Trials
107 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
107 Patients Enrolled for Retinitis Pigmentosa
Applied Genetic Technologies CorpLead Sponsor
10 Previous Clinical Trials
324 Total Patients Enrolled
2 Trials studying Retinitis Pigmentosa
89 Patients Enrolled for Retinitis Pigmentosa
Matthew Feinsod, MDStudy DirectorApplied Genetics Technologies Corporation
2 Previous Clinical Trials
89 Total Patients Enrolled
2 Trials studying Retinitis Pigmentosa
89 Patients Enrolled for Retinitis Pigmentosa
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am a male with a confirmed RPGR mutation.My vision is between 20/32 and 20/200 in both eyes.I have a known retinal disease mutation or received AAV gene therapy and cannot meet the study requirements.I am participating in an early phase of a clinical trial.I have been diagnosed with X-linked retinitis pigmentosa.I am a male aged 6-50 with a confirmed RPGR mutation.I have a confirmed genetic mutation in the RPGR gene.I do not have eye conditions like glaucoma or diabetic retinopathy that could complicate surgery.I have never received AAV gene therapy before.I have been diagnosed with X-linked retinitis pigmentosa.I am a male aged 6-50 with a confirmed RPGR mutation.I haven't taken blood thinners in the last 7 days.I haven't taken steroids or immunosuppressants in the last 3 months.You must meet all of the criteria for the study.
Research Study Groups:
This trial has the following groups:- Group 1: Group 2: Phase 1/2 Dose Escalation
- Group 2: Group 5 Phase 1/2 Dose Escalation
- Group 3: Group 1: Phase 1/2 Dose Escalation
- Group 4: Group 3 and Group 4 Phase 1/2 Dose Escalation
- Group 5: Group 6 Phase 1/2 Dose Escalation
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.
Retinitis Pigmentosa Patient Testimony for trial: Trial Name: NCT03316560 — Phase 1 & 2
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Does the age cutoff for this research encompass octogenarians?
"The present study is seeking participants aged between 6 and 50 years old."
Answered by AI
What is the current capacity of individuals accepted into this research project?
"Affirmative. Information provided on clinicaltrials.gov endorses the active search for participants in this medical trial, first posted on April 16th 2018 and recently updated July 13 th 2021. A total of 42 patients are needed to be recruited across 5 different sites."
Answered by AI
Is it possible for me to enroll in this research endeavor?
"This medical study requires that applicants are between 6 and 50 years of age, suffering from retinitis pigmentosa. Up to 42 individuals may be accepted for the trial."
Answered by AI
In what capacity is this experimentation running geographically?
"At present, this trial is being conducted at five different medical centres. These sites are located in Dallas, Cincinnati and Portland among a few other cities. If you decide to join the study, picking the closest location would help minimize travel requirements."
Answered by AI
Is this experiment currently accepting participants?
"According to clinicialtrials.gov, this research is currently searching for test subjects; it was first published on April 16th 2018 and has had recent updates made as of July 13th 2021."
Answered by AI
Who else is applying?
What state do they live in?
Other
Puerto Rico
Texas
Florida
How old are they?
18 - 65
What site did they apply to?
Casey Eye Institute, Oregon Health and Science University
University of Florida
Retina Foundation of the Southwest
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
0
Why did patients apply to this trial?
I don't want to go blind. Please i am more than willing to be part of your trial and ready to start anytime.
PatientReceived 1 prior treatment
I’ve been living with this for 16 years. No trials in Australia & I want to play with my grandkids.
PatientReceived 1 prior treatment
I am currently still able to do and move with the eyesight i have and i am hoping to retain and hopefully re gain some so i can fly and drive.
PatientReceived 2+ prior treatments
What questions have other patients asked about this trial?
Does it match the type of X-linked RP that I have?
PatientReceived no prior treatments
How responsive is this trial?
Typically responds via
Email
Most responsive sites:
- Retina Foundation of the Southwest: < 24 hours
Average response time
- < 1 Day
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