Follow-up 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: Active, not recruiting; Phase 3

Conditions: X-Linked Retinitis Pigmentosa

Treatments: Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose; Biological: Genetic: AAV5-hRKp.RPGR Low Dose

Study type: Interventional

Funder types: Industry

Identifiers

NCT04794101

2020-002255-37 (EudraCT Number)

MGT-RPGR-022

Details and patient eligibility

About

A clinical trial of AAV5-hRKp.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

• None

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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97 participants in 3 patient groups

Deferred Treatment From MGT-RPGR-021 of Intermediate Dose

Experimental group

Description:

Deferred treatment

Treatment:

Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose

Already Treated in MGT-RPGR-021

Experimental group

Description:

Already treated

Treatment:

Biological: Genetic: AAV5-hRKp.RPGR Low Dose

Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose

Deferred Treatment From MGT-RPGR-021 Low Dose

Experimental group

Description:

Deferred treatment

Treatment:

Biological: Genetic: AAV5-hRKp.RPGR Low Dose