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THOR - Tübingen Choroideremia Gene Therapy Trial

NCT02671539 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2020-10-27

No results posted yet for this study

Summary

An open label monocentric phase II trial in adult males with a clinical phenotype of choroideremia and a confirmed molecular diagnosis of a null mutation in the gene encoding REP1 to assess the anatomical and functional outcomes, as well as the safety of a single subretinal injection of rAAV2.REP1 in 6 subjects with genetically confirmed choroideremia for up to 24 months.

Conditions

  • Choroideremia

Interventions

GENETIC

rAAV2.REP1

single subretinal injection

Sponsors & Collaborators

  • University Hospital Tuebingen

    collaborator OTHER

  • STZ eyetrial

    lead OTHER

Principal Investigators

  • Manuel D Fischer, MD, PhD · Centre for Ophthalmology, University Hospital Tübingen, Germany

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-31
Primary Completion
2018-02-28
Completion
2018-02-28

Countries

  • Germany

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 NCT02671539 on ClinicalTrials.gov