An Open-label, Multicenter, Phase 2 Follow-on Study for Second Eye Treatment of Patients Previously Treated With a Recombinant Adeno-associated Virus Vector (AAV5 hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Other, Biological
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 5
Healthy Volunteers: f
View:
• Have been treated with AAV5-hRKp.RPGR in study MGT009 and have completed or is currently enrolled in Study MGT010
• Must sign an informed consent form indicating that they understand the purpose and procedures of the study and is willing to participate in the study
• Willing to adhere to the protocol and long-term follow-up
Locations
United States
Massachusetts
Massachusetts Eye and Ear Infirmary
RECRUITING
Boston
Michigan
University of Michigan Kellogg Eye Center
RECRUITING
Ann Arbor
Other Locations
United Kingdom
Moorfields Eye Hospital
RECRUITING
London
Contact Information
Primary
Study Contact
Participate-In-This-Study1@its.jnj.com
844-434-4210
Time Frame
Start Date: 2024-10-10
Estimated Completion Date: 2030-10-24
Participants
Target number of participants: 42
Treatments
Experimental: Cohort 1
Participants will receive a dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 depending on the dosage administered in study MGT009 (NCT03252847) in the past. After receiving the treatment, participants will be assessed for safety.
Experimental: Cohort 2
Participants will receive the treatment dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 in the second eye once the safety will be determined in Cohort 1.
Other: Cohort 3
The participants who are not willing to undergo surgery or are not eligible for surgery will be assessed in this cohort. They will be assessed yearly until 5 years after their initial eye surgery in previous study MGT009.
Related Therapeutic Areas
Sponsors
Leads: Janssen Research & Development, LLC