CompletedPhase 2

REP1 Gene Replacement Therapy for Choroideremia

United Kingdom30 participantsStarted 2016-08-16

Plain-language summary

The assessment of the efficacy (with respect to preservation of visual function and retinal structure) and safety of a single subretinal injection of AAV2.REP1 in participants with a confirmed diagnosis of choroideremia, as evaluated by various functional and anatomical outcomes measured over a number of time points up to 24 months post-treatment.

Who can participate

Age range18 Years – 90 Years

SexMALE

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Inclusion criteria

✓. 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 better than or equal to 6/60 (20/200; Decimal 0.1; LogMAR 1.0) in the study eye.

Exclusion criteria

✕. 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 period of 45 days.
✕. Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery.
✕. Participation in another research study involving an investigational product in the preceding 12 weeks.

What they're measuring

Change from baseline in best corrected visual acuity in the treated eye

Timeframe: 2 years

Trial details

NCT IDNCT02407678

SponsorUniversity of Oxford

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2021-07-23

View on ClinicalTrials.gov More Choroideremia trials