REP1 Gene Replacement Therapy for Choroideremia (REGENERATE)

University of Oxford

Status and phase

Completed

Phase 2

Conditions

Choroideremia

Treatments

Genetic: AAV-mediated REP1 gene replacement

Study type

Interventional

Funder types

Other

Identifiers

NCT02407678

REGEN2015

Details and patient eligibility

About

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.

Enrollment

30 estimated patients

Sex

Male

Ages

18 to 90 years old

Volunteers

No Healthy Volunteers

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.