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Trial record 1 of 2 for:    NCT01461213
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Gene Therapy for Blindness Caused by Choroideremia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01461213
Recruitment Status : Completed
First Posted : October 28, 2011
Last Update Posted : November 17, 2017
Sponsor:
Collaborators:
Oxford University Hospitals NHS Trust
Moorfields Eye Hospital NHS Foundation Trust
University College, London
Manchester University NHS Foundation Trust
University of Manchester
University Hospital Southampton NHS Foundation Trust
University of Southampton
Information provided by (Responsible Party):
University of Oxford

Study Description
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Brief 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.


Condition or disease Intervention/treatment Phase
Choroideremia Drug: rAAV2.REP1 Phase 1 Phase 2

Detailed 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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Study Start Date : October 2011
Actual Primary Completion Date : October 2017
Actual Study Completion Date : October 2017

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Dose 1
Dose 1 = single subretinal injection of vector suspension containing approximately 10e10 rAAV2.REP1 genome particles. Six patients have now received Dose 1.
 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.
Other Name: Adeno-associated viral vector
Experimental: Dose 2
Dose 2 = single subretinal injection of vector suspension containing approximately 10e11 rAAV2.REP1 genome particles. Three patients thus far have received Dose 2.
 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.
Other Name: Adeno-associated viral vector


Outcome Measures
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Primary Outcome Measures :
  1. Visual acuity [ Time Frame: 6 months ]
    Best corrected visual acuity, following cataract surgery if indicated


Secondary Outcome Measures :
  1. Microperimetry, OCT and fundus autofluorescence [ Time Frame: 24 months ]
    Structure function correlations at the margins of the retinal degeneration


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01461213


Locations
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United Kingdom
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom, EC1V 2PD
St Mary's Hospital, Central Manchester University Hospitals NHS Foundation Trust
Manchester, United Kingdom, M13 9WL
Oxford Radcliffe Hospitals NHS Trust
Oxford, United Kingdom, OX3 9DU
Eye Unit, Southampton University Hospitals NHS Trust
Southampton, United Kingdom, SO16 6YD
Sponsors and Collaborators
University of Oxford
Oxford University Hospitals NHS Trust
Moorfields Eye Hospital NHS Foundation Trust
University College, London
Manchester University NHS Foundation Trust
University of Manchester
University Hospital Southampton NHS Foundation Trust
University of Southampton
Investigators
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Study Chair: Robert E MacLaren, MB ChB DPhil University of Oxford, Oxford Radcliffe Hospitals NHS Trust and Moorfields Eye Hospital
Principal Investigator: Miguel C Seabra, MD PhD Imperial College London
Principal Investigator: Andrew R Webster, MD UCL Institute of Ophthalmology and Moorfields Eye Hospital
Principal Investigator: Susan M Downes, MD Oxford University Hospitals NHS Trust
Principal Investigator: Graeme C Black, MB BCh DPhil University of Manchester and Central Manchester University Hospitals NHS Foundation Trust
Principal Investigator: Andrew J Lotery, MD University of Southampton and Southampton University Hospitals Trust
Principal Investigator: Len W Seymour, PhD University of Oxford
Principal Investigator: Tanya Tolmachova, PhD Imperial College London
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: University of Oxford
ClinicalTrials.gov Identifier: NCT01461213    
Other Study ID Numbers: CHM09/01
2009-014617-27 ( EudraCT Number )
First Posted: October 28, 2011    Key Record Dates
Last Update Posted: November 17, 2017
Last Verified: November 2017
Keywords provided by University of Oxford:
Tapetoretinal degeneration, choroideraemia, X-linked
retinitis pigmentosa
Additional relevant MeSH terms:
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Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
 Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked