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Recruiting NCT06131983

Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1

Phase I / Phase II Interventional Facio-Scapulo-Humeral Dystrophy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ARO-DUX4 for Injection, Placebo.
Who it may be relevant to
Registry conditions: Facio-Scapulo-Humeral Dystrophy. Basic parameters: 16 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Australia, Canada, Germany, Italy, Netherlands +3
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1

Overview

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.

Interventions

  • Drug ARO-DUX4 for Injection
    single or multiple doses of ARO-DUX4 by intravenous (IV) infusion
  • Drug Placebo
    calculated volume to match active treatment by IV infusion

Primary outcome measures

  • Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS) [Time frame: Part 1: Up to Day 90; Part 2: Up to Day 360]
Secondary outcome measures (10)
  • Pharmacokinetics (PK) of ARO-DUX4: Maximum Observed Plasma Concentration (Cmax) [Time frame: 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) [Time frame: 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) [Time frame: 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) [Time frame: 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) [Time frame: 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: Systemic Clearance (CL) [Time frame: 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: Volume of Distribution (Vss) [Time frame: 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: Recovery of Unchanged Drug in Urine Over 0-24 Hours (Amount Excreted: Ae) [Time frame: 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: Fraction of Drug Excreted in Urine as Percent of Intravenous (IV) Dose (Fe) [Time frame: 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: Renal Clearance (CLr) [Time frame: 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]

Eligibility criteria

Inclusion criteria

  • 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.

Exclusion criteria

  • 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

Note: additional inclusion/exclusion criteria may apply per protocol

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

Australia · 4 centers
  • Research Site 2 — Liverpool
  • Research Site 3 — Auchenflower
  • Research Site 1 — Birtinya
  • Research Site 4 — Melbourne
Canada · 3 centers
  • Research Site 2 — Calgary
  • Research Site 3 — Edmonton
  • Research Site 1 — Montreal
Spain · 3 centers
  • Research Site 3 — Barcelona
  • Research Site 2 — Madrid
  • Research Site 1 — Valencia
Germany · 2 centers
  • Research Site 2 — München
  • Research Site 1 — Ulm
Italy · 2 centers
  • Research Site 1 — Milan
  • Research Site 2 — Roma
Netherlands · 1 center
  • Research Site 1 — Leiden
New Zealand · 1 center
  • Research Site 1 — Auckland
Thailand · 1 center
  • Research Site — Bangkok

Identifiers

NCT: NCT06131983 · ARODUX4-1001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗