REP1 Gene Replacement Therapy for Choroideremia

Choroideremia Clinical Trial

— REGENERATE  

Official title:

An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02407678
• Other study ID #: REGEN2015
• Status: Completed
• Phase: Phase 2
• Start date: August 16, 2016
• Est. completion date: July 23, 2021

Study information

• Verified date: July 2021
• Source: University of Oxford
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: July 23, 2021
• Est. primary completion date: July 23, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 90 Years

Eligibility

Inclusion Criteria:

1. Candidate is 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 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 period of 45 days.

5. Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery.

6. Participation in another research study involving an investigational product in the preceding 12 weeks.

Related Conditions & MeSH terms

• Choroideremia

Intervention

Genetic:
• AAV-mediated REP1 gene replacement
AAV vector carrying human REP1 gene is delivered into the treated eye by subretinal injection

Locations

Country	Name	City	State
United Kingdom	Moorfields Eye Hospital NHS Foundation Trust	London
United Kingdom	Oxford University Hospitals NHS Foundation Trust	Oxford

Sponsors (3)

Lead Sponsor	Collaborator
University of Oxford	Moorfields Eye Hospital NHS Foundation Trust, University College, London

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in best corrected visual acuity in the treated eye		2 years
Secondary	Change from baseline in the central visual field in the treated eye as determined by microperimetry		2 years
Secondary	Change from baseline in the area of surviving retinal pigment epithelium 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		2 years