Gene therapy for treatment of choroideremia

Not Applicable

Completed

• Conditions: Choroideremia; Eye Diseases; Hereditary choroidal dystrophy

• Registration Number: ISRCTN15602229
• Lead Sponsor: IHR Evaluation, Trials and Studies Coordinating Centre (NETSCC)

Brief Summary

2024 Results article in https://doi.org/10.3310/WARA5730 (added 05/06/2024)

Detailed Description

Not available

Study Timeline

• Study Start: 2016-04-28
• Study Completion: 2021-07-31
• Last Update Posted: 18 June 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Male
• Target Recruitment: 30

Inclusion Criteria

1. Willing and able to give informed consent for participation in the study
2. Male aged 18 years or above
3. Genetic or molecular confirmed diagnosis of choroideremia (REP1 protein deficiency)
4. Active disease visible clinically within the macula region
5. 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

Exclusion Criteria

1. Any female, or a male aged below 18 years
2. An additional cause for sight loss (e.g. amblyopia) in the eye to be treated
3. 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
4. Inability to take systemic prednisolone for a minimum of 3 weeks
5. Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery, if relevant
6. Participation in another research study involving an investigational product in the preceding 12 weeks

Study & Design

• Study Type: Interventional
• Study Design: Not specified