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AAV2-hCHM · 1 trial · 2 indications
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were AEs that occurred on or after the day of study drug administration. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: AAV2-hCHM Dose 1 | EXPERIMENTAL | Single, unilateral subretinal administration of a single low dose range of AAV2-hCHM. |
| Cohort 2: AAV2-hCHM Dose 2 | EXPERIMENTAL | Single, unilateral subretinal administration of a single high dose range of AAV2-hCHM. |
| Cohort 3 (Expansion Cohort): AAV2-hCHM Dose 2 | EXPERIMENTAL | Single, unilateral subretinal administration of a single high dose range of AAV2-hCHM. |
| Name | Type | Description |
|---|---|---|
| AAV2-hCHM | BIOLOGICAL | Comparison of different dosages of AAV2-hCHM |
Inclusion Criteria: * Male at least 18 years of age diagnosed with CHM gene mutation * Central visual field (VF) \<30° in any of the 24 meridians (using Goldmann perimetry III4e isopter) in the eye to be injected * Any evidence of functioning outer retinal cells within the central 10° Exclusion Cr...
AAV2-hCHM is an investigational gene therapy being developed by Roche Holding AG for choroideremia, a rare inherited retinal disorder. It is designed to deliver a functional copy of the CHM gene to retinal cells. It has been studied in a single Phase 1 clinical trial and is not approved for any indication.
AAV2-hCHM is being studied as a potential treatment for choroideremia, a rare genetic eye disease that causes progressive vision loss. It is intended to address the underlying CHM gene mutations. The therapy remains investigational and has not been approved for clinical use.
AAV2-hCHM is being developed by Roche Holding AG, which trades on the OTC market under the ticker RHHBY. Roche is the sponsor of the clinical program evaluating this gene therapy for choroideremia.
AAV2-hCHM is in Phase 1 clinical development. The only reported trial was a Phase 1 safety and dose-escalation study that has been completed. The therapy is investigational and has not received FDA approval.
AAV2-hCHM has been evaluated in one clinical trial, NCT02341807, titled "Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations." The Phase 1 trial enrolled 15 male participants aged 18 years and older in the United States and is now completed.