Gene Therapy for Blindness Caused by Choroideremia

Choroideremia Clinical Trial

Official 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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01461213
• Other study ID #: CHM09/01
• Secondary ID: 2009-014617-27
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: October 21, 2011
• Last updated: November 16, 2017
• Start date: October 2011
• Est. completion date: October 2017

Study information

• Verified date: November 2017
• Source: University of Oxford
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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: www.thelancet.com/journals/lancet/article/PIIS0140-6736(13)62117-0/abstract ; http://dx.doi.org/doi:10.1016/S0140-6736(13)62117-0

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: October 2017
• Est. primary completion date: October 2017
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Choroideremia

Intervention

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.

Locations

Country	Name	City	State
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

Sponsors (8)

Lead Sponsor	Collaborator
University of Oxford	Central Manchester University Hospitals NHS Foundation Trust, Moorfields Eye Hospital NHS Foundation Trust, Oxford University Hospitals NHS Trust, University College, London, University Hospital Southampton NHS Foundation Trust, University of Manchester, University of Southampton

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Visual acuity	Best corrected visual acuity, following cataract surgery if indicated	6 months
Secondary	Microperimetry, OCT and fundus autofluorescence	Structure function correlations at the margins of the retinal degeneration	24 months