NCT04483440

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

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: 4D Molecular Therapeutics · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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).

Detailed 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 three dose levels in male patients with genetically-confirmed CHM.

Conditions

• Choroideremia

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2020-06-02
• Primary completion: 2027-05-26
• Completion: 2027-08-31
• First posted: 2020-07-23
• Last updated: 2025-05-22

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study