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

  • STATUS
    Recruiting
  • participants needed
    15
  • sponsor
    4D Molecular Therapeutics
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Updated on 19 February 2024
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Summary

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.

Details
Condition Choroideremia
Age 18-100 years
Treatment 4D-110
Clinical Study IdentifierNCT04483440
Sponsor4D Molecular Therapeutics
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

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)

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)
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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