Title

Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia
  • Phase

    Phase 1
  • Study Type

    Interventional
  • Status

    Recruiting
  • Intervention/Treatment

    4D-110
  • Study Participants

    15
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).
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.
Study Started
Jun 02
2020
Primary Completion
Nov 30
2021
Anticipated
Study Completion
May 31
2023
Anticipated
Last Update
Sep 23
2021

Biological 4D-110

4D-110 drug product developed for gene therapy which comprises an AAV capsid variant (4D-R100) carrying a transgene encoding a codon-optimized human CHM gene.

4D-110 Dose 1 Experimental

4D-110 IVT injection

4D-110 Dose 2 Experimental

4D-110 IVT injection

Criteria

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)
No Results Posted