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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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
See also
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Withdrawn NCT05045703 - The Dark-Adapted Retinal Function Response in Choroideremia (DARC) Study N/A
Recruiting NCT01866371 - High Resolution Retinal Imaging
Completed NCT01603576 - Pilot Study of a Suprachoroidal Retinal Prosthesis N/A
Enrolling by invitation NCT03584165 - Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa Phase 3
Completed NCT03359551 - Natural History of the Progression of Choroideremia Study
Recruiting NCT02435940 - Inherited Retinal Degenerative Disease Registry
Active, not recruiting NCT04483440 - Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia Phase 1
Completed NCT02671539 - THOR - Tübingen Choroideremia Gene Therapy Trial Phase 2
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Completed NCT02341807 - Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations Phase 1/Phase 2
Completed NCT03406416 - Study of a Suprachoroidal Retinal Prosthesis N/A
Enrolling by invitation NCT05158049 - Longitudinal Study of a Bionic Eye
Terminated NCT02994368 - "Natural History" Study of Choroideremia
Terminated NCT01654562 - The Short-term Effects of Simvastatin on the Vision of Males Affected by Choroideremia Phase 1/Phase 2
Active, not recruiting NCT00427180 - IRIS PILOT - Extended Pilot Study With a Retinal Implant System N/A
Completed NCT04750785 - A Study to Assess Choroideremia (CHM) Health Outcomes