Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Janssen (J&J Innovative Medicine)

Status and phase: Completed; Phase 3

Conditions: X-Linked Retinitis Pigmentosa

Treatments: Biological: Genetic: AAV5-hRKp.RPGR

Study type: Interventional

Funder types: Industry

Identifiers

NCT04671433

2020-002873-88 (EudraCT Number)

MGT-RPGR-021

CR109258

Details and patient eligibility

About

A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Enrollment: 97 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female
• 3 years of age or older
• Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory

Exclusion criteria

• Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration
• Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
• Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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97 participants in 3 patient groups

Experimental - Immediate Treatment

Experimental group

Description:

Intermediate dose.

Treatment:

Biological: Genetic: AAV5-hRKp.RPGR

Biological: Genetic: AAV5-hRKp.RPGR

Deferred Treatment

Other group

Description:

Deferred Treatment

Treatment:

Biological: Genetic: AAV5-hRKp.RPGR

Biological: Genetic: AAV5-hRKp.RPGR

Experimental Immediate Treatment

Experimental group

Description:

Low dose.

Treatment:

Biological: Genetic: AAV5-hRKp.RPGR

Biological: Genetic: AAV5-hRKp.RPGR