A Phase 1/2 Multicenter, Open-label, Dose Escalation, Safety and Efficacy Study of Subretinal Administration of Dual AAV8.MYO7A, AAVB-081 in Participants with Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-518489-29-00

Acronym

081-101

Enrollment

Registered

2024-11-19

Start date

2024-07-01

Completion date

Unknown

Last updated

2025-06-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Usher Syndrome Type 1B (USH1B) Retinitis Pigmenotsa

Brief summary

The incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment emergent adverse events (TEAEs) and dose limiting toxicities (DLTs) at 24 months., The incidence and nature of DLTs at each dose level at 24 months

Interventions

DRUGAAVB-081

DRUGPREDNISONE

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
The incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment emergent adverse events (TEAEs) and dose limiting toxicities (DLTs) at 24 months., The incidence and nature of DLTs at each dose level at 24 months	—

Countries

Italy

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026