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 1Lead Sponsor
4D Molecular TherapeuticsStudy Type
InterventionalStatus
Active, not recruitingIndication/Condition
ChoroideremiaIntervention/Treatment
4D-110Study Participants
13This 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.
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)
