A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2022-502096-32-00

Acronym

AOC 1020-CS1

Enrollment

Registered

2023-07-25

Start date

Unknown

Completion date

2023-11-29

Last updated

2023-12-11

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

Conditions

Facioscapulohumeral muscular dystrophy (FSHD)

Brief summary

Incidence of treatment-emergent adverse events

Detailed description

Estimation of plasma PK parameters, including maximum plasma concentration, half-life, and area under the curve of AOC 1020, Concentration of siRNA component in skeletal muscle (Day 120)

Interventions

DRUGThe placebo for this study is saline (0.9% sodium chloride water for injection) for intravenous infusion administration and will be provided by the study site.

DRUGAOC 1020

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence of treatment-emergent adverse events	—

Secondary

Measure	Time frame
Estimation of plasma PK parameters, including maximum plasma concentration, half-life, and area under the curve of AOC 1020, Concentration of siRNA component in skeletal muscle (Day 120)	—

Countries

Italy, Netherlands

Outcome results

None listed

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