findmyKOL
Clinical trialNCT01461213CompletedPhase 1/PHASE2Interventional

Gene Therapy for Blindness Caused by Choroideremia

SponsorUniversity of OxfordEnrollment14Choroideremia
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

\- Primary objective: To assess the safety and tolerability of the AAV.REP1 vector, administered at two different doses to the retina in 12 patients with a diagnosis of choroideremia. \- Secondary Objective: To identify any therapeutic benefit as evidenced by a slowing down of the retinal degeneration assessed by functional and anatomical methods in the treated eye compared to the control eye 24 months after gene delivery.
clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

  • Andrew J Lotery, MD

    University of Southampton and Southampton University Hospitals Trust

    Principal Investigator
    clinicaltrials.gov ↗
  • Andrew R Webster, MD

    UCL Institute of Ophthalmology and Moorfields Eye Hospital

    Principal Investigator
    clinicaltrials.gov ↗
  • Graeme C Black, MB BCh DPhil

    University of Manchester and Central Manchester University Hospitals NHS Foundation Trust

    Principal Investigator
    clinicaltrials.gov ↗
  • Len W Seymour, PhD

    University of Oxford

    Principal Investigator
    clinicaltrials.gov ↗
  • Miguel C Seabra, MD PhD

    Imperial College London

    Principal Investigator
    clinicaltrials.gov ↗
  • Robert E MacLaren, MB ChB DPhil

    University of Oxford, Oxford Radcliffe Hospitals NHS Trust and Moorfields Eye Hospital

    Study Chair
    clinicaltrials.gov ↗
  • Susan M Downes, MD

    Oxford University Hospitals NHS Trust

    Principal Investigator
    clinicaltrials.gov ↗
  • Tanya Tolmachova, PhD

    Imperial College London

    Principal Investigator
    clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

4 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United Kingdom
  • Moorfields Eye Hospital Nhs Foundation Trust

    London, United Kingdom

  • St Mary'S Hospital, Central Manchester University Hospitals Nhs Foundation Trust

    Manchester, United Kingdom

  • Oxford Radcliffe Hospitals Nhs Trust

    Oxford, United Kingdom

  • Eye Unit, Southampton University Hospitals Nhs Trust

    Southampton, United Kingdom

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

RAAV2.REP1
Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
31 Oct 2011
Primary completion
31 Oct 2017
Completion
31 Oct 2017
Last update posted
17 Nov 2017
Registered enrollment
14
Registry id
NCT01461213

Cross-registrations

EU-CTR / CTIS (EudraCT)2009-014617-27
clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.