Gene Therapy for Blindness Caused by Choroideremia | Recruiting
Gene Therapy for Blindness Caused by Choroideremia

Trial Source

There is no location for this trial

Location data is sourced from multiple external providers and UKCTG is not responsible for and cannot guarantee the accuracy of data.

Health Conditions
  • Choroideremia
Primary Contact Details
Recruitment Status
Primary Trial ID Number
- 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.
Research Details
  • Detailed description may be found in the following scientific publication: Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial, The Lancet, Volume 383, Issue 9923, Pages 1129 - 1137 (29 March 2014). Links: ;
Phase 1/Phase 2
Study Design
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Study Type
Drug : rAAV2.REP1

Study Arm Groups : Dose 1, Dose 2

Intervention Type
See Interventions above
Primary Outcome Measures
  • Visual acuity; 6 months
Secondary Outcome Measures
  • Microperimetry, OCT and fundus autofluorescence; 24 months
Sorry, this information is not available
Result Reports
This is available on the website
Age Range
18 Years - N/A
Who Can Participate
Number of Participants
Participant Inclusion Criteria
  • Inclusion Criteria:
  • - Participant is willing and able to give informed consent for participation in the
  • study,
  • - Male aged 18 years or above,
  • - Diagnosed with choroideraemia and in good health,
  • - Active disease with SLO changes visible within the macula region,
  • - Willing to allow his or her General Practitioner and consultant, if appropriate, to
  • be notified of participation in the study,
  • - Vision at least 6/60 or better in the study eye.
  • Exclusion Criteria:
  • - Female and child participants (under the age of 18),
  • - Men unwilling to use barrier contraception methods, if relevant,
  • - Previous history of retinal surgery or ocular inflammatory disease (uveitis),
  • - Grossly asymmetrical disease or other ocular morbidity which might confound use of
  • the fellow eye as a long-term control,
  • - Any other significant disease or disorder which, in the opinion of the Investigator,
  • may either put the participants at risk because of participation in the study, or may
  • influence the result of the study, or the participant's ability to participate in the
  • study,
  • - Participants who have participated in another research study involving an
  • investigational product in the previous 12 weeks.
Participant Exclusion Criteria
This is in the inclusion criteria above
Trial Location(s)
University of Oxford
Southampton General Hospital
SO16 6YD
Manchester Royal Infirmary
M13 9WL
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
Trial Contact(s)
Primary Trial Contact
Robert E MacLaren
Other Trial Contacts
Sorry, this information is not available
Countries Recruiting
United Kingdom
Scientific Title
An Open Label Dose Escalation Phase 1 Clinical Trial of Retinal Gene Therapy for Choroideraemia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)
EudraCT Number
Not available for this trial
  • Oxford University Hospitals NHS Trust
  • Moorfields Eye Hospital NHS Foundation Trust
  • University College, London
  • Central Manchester University Hospitals NHS Foundation Trust
  • University of Manchester
  • University Hospital Southampton NHS Foundation Trust.
  • University of Southampton
Other Study ID Numbers
University of Oxford
Key Dates

Recruitment Start Date

Oct 2011

Recruitment End Date

Sep 2015

Trial Start Date
Date Not Available
Trial End Date
Date Not Available
Date Assigned

21 Oct 2011

Last Updated

05 Mar 2015