Gene Therapy for Blindness Caused by Choroideremia

Summary

\- 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.

Conditions

• Choroideremia

Interventions

DRUG

rAAV2.REP1

Single subretinal injection of rAAV2.REP1 vector suspension containing 10e12 genome particles per ml. Dose 1 = dose containing approximately 10e10 rAAV2.REP1 genome particles. Dose 2 = dose containing approximately 10e11 rAAV2.REP1 genome particles.

Sponsors & Collaborators

• Oxford University Hospitals NHS Trust

  collaborator OTHER

• Moorfields Eye Hospital NHS Foundation Trust

  collaborator OTHER

• University College, London

  collaborator OTHER

• Manchester University NHS Foundation Trust

  collaborator OTHER_GOV

• University of Manchester

  collaborator OTHER

• University Hospital Southampton NHS Foundation Trust

  collaborator OTHER

• University of Southampton

  collaborator OTHER

• University of Oxford

  lead OTHER

Principal Investigators

• Robert E MacLaren, MB ChB DPhil · University of Oxford, Oxford Radcliffe Hospitals NHS Trust and Moorfields Eye Hospital

• Miguel C Seabra, MD PhD · Imperial College London

• Andrew R Webster, MD · UCL Institute of Ophthalmology and Moorfields Eye Hospital

• Susan M Downes, MD · Oxford University Hospitals NHS Trust

• Graeme C Black, MB BCh DPhil · University of Manchester and Central Manchester University Hospitals NHS Foundation Trust

• Andrew J Lotery, MD · University of Southampton and Southampton University Hospitals Trust

• Len W Seymour, PhD · University of Oxford

• Tanya Tolmachova, PhD · Imperial College London

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Sex

MALE

Healthy Volunteers

No

Timeline & Regulatory

Start

2011-10-31

Primary Completion

2017-10-31

Completion

2017-10-31

Countries

• United Kingdom

Study Locations