Long-term Follow-up Gene Therapy Study for RPGR- XLRP

Janssen (J&J Innovative Medicine)

Status: Completed

Conditions: X-Linked Retinitis Pigmentosa

Treatments: Biological: AAV5-hRKp.RPGR

Study type: Observational

Funder types: Industry

Identifiers

NCT04312672

MGT010

2018-000425-31 (EudraCT Number)

Details and patient eligibility

About

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

Enrollment: 42 patients

Sex: Male

Ages: 5+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Received AAV5-hRKp.RPGR in the MGT009 Study

Exclusion criteria

• Unwilling or unable to meet with the requirements of the study

Trial design

42 participants in 1 patient group

Follow up cohort

Description:

no intervention follow up study

Treatment:

Biological: AAV5-hRKp.RPGR