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
ACTIVE_NOT_RECRUITING
Boston
Michigan
University of Michigan Kellogg Eye Center
ACTIVE_NOT_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