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Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

Recruiting
18 years and older
Male
Phase 1

Overview

This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).

Description

This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single IVT injection of 4D-110 at two dose levels in male patients with genetically-confirmed CHM.

Eligibility

Key Inclusion Criteria:

  • Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing
  • Both eyes must have 34 ETDRS letters (~20/200)

Key Exclusion Criteria:

  • Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye
  • Patient has previously received any AAV treatment
  • Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)

Study details
    Choroideremia

NCT04483440

4D Molecular Therapeutics

5 August 2020

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