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REP1 Gene Replacement Therapy for Choroideremia

NCT02407678 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2021-08-05

No results posted yet for this study

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.

Conditions

  • Choroideremia

Interventions

GENETIC

AAV-mediated REP1 gene replacement

AAV vector carrying human REP1 gene is delivered into the treated eye by subretinal injection

Sponsors & Collaborators

  • Moorfields Eye Hospital NHS Foundation Trust

    collaborator OTHER

  • University College, London

    collaborator OTHER

  • University of Oxford

    lead OTHER

Principal Investigators

  • Robert E MacLaren, MB ChB DPhil · University of Oxford

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
90 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-08-16
Primary Completion
2021-07-23
Completion
2021-07-23

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02407678 on ClinicalTrials.gov