A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)

Janssen (J&J Innovative Medicine)

Status and phase: Enrolling; Phase 2

Conditions: X-Linked Retinitis Pigmentosa

Treatments: Biological: AAV5-hRKp.RPGR; Other: No intervention (Follow-Up assessment)

Study type: Interventional

Funder types: Industry

Identifiers

NCT06646289

74765340RPG2001

Details and patient eligibility

About

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.

Enrollment: 39 estimated patients

Sex: Male

Ages: 5+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• There are no specific exclusion criteria to enroll in this study

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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39 participants in 3 patient groups

Cohort 1

Experimental group

Description:

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.

Treatment:

Biological: AAV5-hRKp.RPGR

Cohort 2

Experimental group

Description:

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.

Treatment:

Biological: AAV5-hRKp.RPGR

Cohort 3

Other group

Description:

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.

Treatment:

Other: No intervention (Follow-Up assessment)