Phase 2 clinical trial of a gene therapy for choroideremia
- Conditions
- Choroideremia - a currently incurable retinal degeneration caused by null mutations in the CHM gene encoding REP1 protein located on the X chromosome. The condition causes blindness in males by the third or fourth decade.MedDRA version: 18.1Level: LLTClassification code 10008791Term: ChoroideremiaSystem Organ Class: 100000004853Therapeutic area: Diseases [C] - Eye Diseases [C11]
- Registration Number
- EUCTR2015-001383-18-GB
- Lead Sponsor
- niversity of Oxford
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Male
- Target Recruitment
- 30
• Candidate is willing and able to give informed consent for participation in the study.
• Male aged 18 years or above.
• Genetic or molecular confirmed diagnosis of choroideremia (REP1 protein deficiency).
• Active disease visible clinically within the macula region.
• Best corrected visual acuity equal to or worse than 6/6 (20/20; Decimal 1.0; LogMAR 0) but better than or equal to 6/60 (20/200; Decimal 0.1; LogMAR 1.0) in the study eye.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0
• Any female, or a male aged below 18 years.
• An additional cause for sight loss (e.g. amblyopia) in the eye to be treated.
• Any other significant ocular and non-ocular disease or disorder which, in the opinion of the investigator, may put the participants at risk because of participation in the study.
• Inability to take systemic prednisolone for a minimum of 3 weeks.
• Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery, if relevant.
• Participation in another research study involving an investigational product in the preceding 12 weeks.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the effectiveness (with respect to preservation of visual function and retinal structure) and safety of a single injection of the gene therapy product into the back of the eyes of patients with a confirmed diagnosis of choroideremia. The effectiveness and safety of the gene therapy product will be evaluated by various tests conducted regularly for a period of 2 years following treatment.;Secondary Objective: ;Primary end point(s): Change from baseline in best corrected visual acuity in the treated eye.;Timepoint(s) of evaluation of this end point: Regular tests over a period of 24 months following gene therapy.
- Secondary Outcome Measures
Name Time Method Timepoint(s) of evaluation of this end point: Regular tests over a period of 24 months following gene therapy.;Secondary end point(s): Change from baseline in the central visual field in the treated eye as determined by microperimetry.<br><br>Change from baseline in the area of surviving RPE in the treated eye as measured by fundus autofluorescence, compared to the untreated fellow eye (control eye) after randomisation of treatment to one eye or the other.<br><br>Change from baseline in other functional and anatomical outcomes in the treated eye pertaining to vector efficacy and safety, and safety of the subretinal injection procedure.<br><br>Change from baseline in immunological and physiological outcomes pertaining to vector safety.