NCT06131983

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 4 doses of ARO-DUX4 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose MRI-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
60

participants targeted

Target at P75+ for phase_1

Timeline
7mo left

Started Feb 2024

Typical duration for phase_1

Geographic Reach
8 countries

17 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress80%
Feb 2024Dec 2026

First Submitted

Initial submission to the registry

November 8, 2023

Completed
7 days until next milestone

First Posted

Study publicly available on registry

November 15, 2023

Completed
3 months until next milestone

Study Start

First participant enrolled

February 22, 2024

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2026

Last Updated

February 6, 2026

Status Verified

February 1, 2026

Enrollment Period

2.8 years

First QC Date

November 8, 2023

Last Update Submit

February 4, 2026

Conditions

Facio-Scapulo-Humeral Dystrophy

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)

    Part 1: Up to Day 90; Part 2: Up to Day 360

Secondary Outcomes (10)

  • Pharmacokinetics (PK) of ARO-DUX4: Maximum Observed Plasma Concentration (Cmax)

    Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose

  • PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)

    Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose

  • PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)

    Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose

  • PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time from Zero to Infinity (AUCinf)

    Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose

  • PK of ARO-DUX4: Terminal Elimination Half-Life (t1/2)

    Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose

  • +5 more secondary outcomes

Study Arms (2)

ARO-DUX4

EXPERIMENTAL

ARO-DUX4 for Injection

Drug: ARO-DUX4 for Injection

Placebo

PLACEBO COMPARATOR

(0.9%NaCl)

Drug: Placebo

Interventions

PlaceboDRUG

calculated volume to match active treatment by IV infusion

Placebo
ARO-DUX4 for InjectionDRUG

single or multiple doses of ARO-DUX4 by intravenous (IV) infusion

ARO-DUX4

Eligibility Criteria

Age16 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Genetically confirmed FSHD1 based on Screening evaluation or source verifiable medical record
  • Clinical severity score between 3 and 8 (scale, 0 to 10)
  • Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader
  • A 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety in the study
  • Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 12 weeks following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 12 weeks following the end of study or last dose of study medication, whichever is later.

You may not qualify if:

  • Human Immunodeficiency Virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at Screening
  • Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening
  • Uncontrolled hypertension
  • Severe cardiovascular disease
  • History of thrombolic events
  • Platelet count less that the lower limit of normal at Screening
  • History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.
  • Any contraindication to muscle biopsy or MRI

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Research Site 2

Liverpool, New South Wales, 2170, Australia

RECRUITING

Research Site 3

Auchenflower, Queensland, 4066, Australia

RECRUITING

Research Site 1

Birtinya, Queensland, 4575, Australia

RECRUITING

Research Site 4

Melbourne, Victoria, 3004, Australia

RECRUITING

Research Site 2

Calgary, Alberta, T2N4Z6, Canada

NOT YET RECRUITING

Research Site 3

Edmonton, Alberta, T6G2G4, Canada

RECRUITING

Research Site 1

Montreal, Quebec, H3A2B4, Canada

RECRUITING

Research Site 2

München, 80336, Germany

RECRUITING

Research Site 1

Ulm, 89081, Germany

RECRUITING

Research Site 1

Milan, 20162, Italy

RECRUITING

Research Site 2

Roma, 00168, Italy

RECRUITING

Research Site 1

Leiden, 2333, Netherlands

RECRUITING

Research Site 1

Auckland, 1010, New Zealand

RECRUITING

Research Site 3

Barcelona, 08035, Spain

RECRUITING

Research Site 2

Madrid, 28034, Spain

RECRUITING

Research Site 1

Valencia, 46026, Spain

RECRUITING

Research Site

Bangkok, 10700, Thailand

WITHDRAWN

MeSH Terms

Conditions

Muscular Dystrophy, Facioscapulohumeral

Interventions

Injections

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Drug Administration RoutesDrug TherapyTherapeutics

Central Study Contacts

Medical Monitor

CONTACT

626-304-3400ARO_DUX4@arrowheadpharma.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 8, 2023

First Posted

November 15, 2023

Study Start

February 22, 2024

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

December 1, 2026

Last Updated

February 6, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations

CA(3)DE(2)ES(3)NZ(1)TH(1)NL(1)IT(2)AU(4)