Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia
Overview
- Phase
- Phase 1
- Intervention
- 4D-110
- Conditions
- Choroideremia
- Sponsor
- 4D Molecular Therapeutics
- Enrollment
- 13
- Locations
- 2
- Primary Endpoint
- Frequency and severity of ocular and systemic adverse events (AEs)
- Status
- Active, not recruiting
- Last Updated
- 11 months ago
Overview
Brief 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.
Investigators
Eligibility Criteria
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)
Arms & Interventions
4D-110 Dose 1
4D-110 IVT injection
Intervention: 4D-110
4D-110 Dose 2
4D-110 IVT injection
Intervention: 4D-110
4D-110 Dose 3
4D-110 IVT injection
Intervention: 4D-110
Outcomes
Primary Outcomes
Frequency and severity of ocular and systemic adverse events (AEs)
Time Frame: 24 months
Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.