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The trial is taking place at:
C

Children's Hospital Los Angeles | Neurology Research

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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) logo

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

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

Trial contacts and locations

29

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Central trial contact

Study Contact

Data sourced from clinicaltrials.gov

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