Virus Therapy
rAAV2tYF-GRK1-RPGR for Retinitis Pigmentosa
Boston Children's Hospital, Boston, MA
rAAV2tYF-GRK1-RPGRPhase 1 & 2RecruitingResearch Sponsored by Applied Genetic Technologies CorpEligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Must not have
Be older than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Upday 0 - month 36
Awards & highlights
No Placebo-Only Group
Study Summary
This trial will test a new gene therapy for X-linked retinitis pigmentosa, a degenerative eye disease.
Eligible Conditions
- X-Linked Retinitis Pigmentosa
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
Phase 1/2 Dose Escalation: Number and proportion of Adverse Events
Phase 1/2 Dose Escalation: Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters.
Phase 2 Dose Expansion: The difference in the proportion of responding eyes between treated and control eyes in low dose group and high dose group.
Secondary outcome measures
Phase 1/2 Dose Escalation: Changes from baseline in quality of life questionnaire responses
Phase 1/2 Dose Escalation: Changes from baseline in retinal structure as assessed by spectral-domain optical coherence tomography (SD-OCT)
Phase 1/2 Dose Escalation: Changes from baseline in visual acuity
+6 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
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
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 Escalation and High Dose Group Phase 2 Dose ExpansionExperimental Treatment1 Intervention
Phase 1/2 Dose Escalation: male subjects at least 18 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug.
Phase 2 Dose Expansion: male subjects at least 8 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug. This will be the high dose group for the phase 2 expansion.
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 Escalation and Low Dose Group Phase 2 Dose ExpansionExperimental Treatment1 Intervention
Phase 1/2 Dose Escalation: male subjects at least 18 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug.
Phase 2 Dose Expansion: male subjects at least 8 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug. This will be the low dose group for the phase 2 expansion.
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 site
Who is running the clinical trial?
Applied Genetic Technologies CorpLead Sponsor
10 Previous Clinical Trials
312 Total Patients Enrolled
2 Trials studying Retinitis Pigmentosa
77 Patients Enrolled for Retinitis Pigmentosa
Matthew Feinsod, MDStudy Director
Applied Genetics Technologies Corporation2 Previous Clinical Trials
77 Total Patients Enrolled
2 Trials studying Retinitis Pigmentosa
77 Patients Enrolled for Retinitis Pigmentosa
Media Library
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.
Patient
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.
Patient
What questions have other patients asked about this trial?
Does it match the type of X-linked RP that I have?
Patient
How responsive is this trial?
Average response time
- < 1 Day
Typically responds via
Email
Most responsive sites:
- Retina Foundation of the Southwest: < 24 hours