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NCT04312672Completed

Long-term Follow-up Gene Therapy Study for RPGR- XLRP

Long-term Follow-up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

Source: ClinicalTrials.gov · Last updated 25 Sept 2026

Brief summary (registry, in English)

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

Status (registry)
Completed
Study type
Observational
Enrollment
42 (actual)
First submitted
15 Jul 2019
Start date
31 Jul 2017
Primary completion date
17 Sept 2025
Lead sponsor
MeiraGTx Ocular UK Ltd
NCT number
NCT04312672
EudraCT number
2018-000425-31

What you commit to

  • Study type: Observational
  • Participants enrolled: 42
  • From 31 Jul 2017 to the primary completion date, 17 Sept 2025
  • 5 sites in 2 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Other

  • Unwilling or unable to meet with the requirements of the study

Who the study is looking for

  • Received AAV5-hRKp.RPGR in the MGT009 Study

Age in the registry: 5 years to - · Sex: Male · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (5)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Leeds Teaching Hospitals NHS Trust
Coordinator of this site? Confirm its status
LeedsUnited KingdomUnknown status
Moorfields Eye Hospital
Coordinator of this site? Confirm its status
LondonUnited KingdomUnknown status
Univ of Michigan Medical Center
Coordinator of this site? Confirm its status
Ann ArborUnited StatesUnknown status
Massachusetts Eye and Ear Infirmary
Coordinator of this site? Confirm its status
BostonUnited StatesUnknown status
University of Pittsburgh Medical Center
Coordinator of this site? Confirm its status
PittsburghUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.