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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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