A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-509748-89-00

Acronym

ARODUX4-1001

Enrollment

Registered

2024-08-12

Start date

2025-02-19

Completion date

Unknown

Last updated

2026-01-26

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, frequency, and severity of treatment-emergent adverse events (TEAEs) in subjects with FSHD1 over time through end of study (EOS) at Day 90 (Part 1) or Day 360 (Part 2)

Detailed description

Plasma PK of ARO-DUX4 in subjects with FSHD1, Urinary excretion of ARO-DUX4 in subjects with FSHD1

Interventions

DRUGSODIUM CHLORIDE

DRUGARO-DUX4

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence, frequency, and severity of treatment-emergent adverse events (TEAEs) in subjects with FSHD1 over time through end of study (EOS) at Day 90 (Part 1) or Day 360 (Part 2)	—

Secondary

Measure	Time frame
Plasma PK of ARO-DUX4 in subjects with FSHD1, Urinary excretion of ARO-DUX4 in subjects with FSHD1	—

Countries

Germany, Italy, Netherlands, Spain

Outcome results

None listed

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