Status:

COMPLETED

Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)

Lead Sponsor:

MeiraGTx UK II Ltd

Collaborating Sponsors:

Janssen, LP

Conditions:

Retinitis Pigmentosa

Eligibility:

All Genders

5+ years

Brief Summary

The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone pho...

Detailed Description

X-linked Retinitis Pigmentosa (XLRP) is a severe form of RP with early onset of nyctalopia and progression to legal blindness by the 3rd to 4th decade. Most affected males show symptomatic night blind...

Eligibility Criteria

Inclusion

  • Males \& Females aged 5 years or older
  • Have RPGR-associated retinal dystrophy
  • Are able to give informed consent or assent, with the guidance of their parent/guardian where appropriate
  • Are able to undertake age-appropriate clinical assessments as specified in the protocol
  • Have genetic mutation within the RPGR gene confirmed by an accredited lab or research lab.

Exclusion

  • Are unable or unwilling to undertake consent or clinical testing

Key Trial Info

Start Date :

December 19 2017

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

April 19 2024

Estimated Enrollment :

140 Patients enrolled

Trial Details

Trial ID

NCT03349242

Start Date

December 19 2017

End Date

April 19 2024

Last Update

June 20 2024

Active Locations (8)

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Page 1 of 2 (8 locations)

1

Shiley Eye Institute - UCSD

La Jolla, California, United States, 92093

2

Stanford University, Spencer Center for Vision Research

Stanford, California, United States, 94303

3

Emory Eye Centre

Atlanta, Georgia, United States, 30322

4

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, United States, 02114