ARO-DUX4 for Muscular Dystrophy

Recruiting at 19 trial locations

Overseen By Medical Monitor

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Arrowhead Pharmaceuticals

Must not be taking: Hormone-based contraceptives

Disqualifiers: HIV, Hepatitis B/C, Hypertension, Cardiovascular disease, others

Compensation: Varies

Number of Visits: 5

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ARO-DUX4 for individuals with facioscapulohumeral muscular dystrophy Type 1 (FSHD1), a condition that weakens muscles, particularly in the face, shoulders, and upper arms. Researchers aim to determine the treatment's safety and behavior in the body. Participants will receive either the treatment or a placebo (a harmless, inactive substance) at different stages of the trial. Suitable candidates for this trial have a confirmed diagnosis of FSHD1 and can undergo muscle biopsies. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that ARO-DUX4 is likely to be safe for humans?

Research shows that ARO-DUX4 has undergone early testing to assess its safety in humans. Lab studies produced promising results in targeting the DUX4 protein, linked to facioscapulohumeral muscular dystrophy (FSHD). The goal is to reduce this protein's production to help treat the disease.

Currently, ARO-DUX4 is in the early stages of human testing. This phase focuses on safety and tolerability, as researchers evaluate how well people tolerate the treatment and monitor for side effects. Since the treatment is in early trials, scientists continue to gather detailed safety information.

In summary, while initial tests suggest promise, more data is needed to fully understand its safety in people with FSHD. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for muscular dystrophy?

ARO-DUX4 is unique because it targets the root cause of muscular dystrophy by silencing the DUX4 gene expression, which is not addressed by existing treatments. Unlike traditional options that focus on managing symptoms, ARO-DUX4 uses a novel RNA interference mechanism. This approach aims to tackle the disease at a genetic level, potentially offering more effective and lasting relief for patients. Researchers are excited because this method could revolutionize how muscular dystrophy is treated, offering hope for a more durable solution.

What evidence suggests that ARO-DUX4 might be an effective treatment for muscular dystrophy?

Research has shown that ARO-DUX4, which participants in this trial may receive, may help treat facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In studies with mice, this treatment lowered levels of DUX4 mRNA, a key factor in the disease. This reduction is crucial because DUX4 is linked to the muscle damage seen in FSHD1. The studies also noted improvements in muscle condition, such as reduced muscle wasting and better overall muscle health. These findings suggest that ARO-DUX4 could help manage or improve symptoms in people with FSHD1. ^{1 2 5 6 7}

Are You a Good Fit for This Trial?

Adults and adolescents with genetically confirmed Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1) can join this trial. They should have a moderate clinical severity score, no serious heart issues on an ECG, and a muscle eligible for biopsy. Participants must use effective contraception and not donate sperm during the study.

Inclusion Criteria

My condition's severity is moderate, rated between 3 and 8.

Your heart test at screening should not show any abnormal results that could put your safety at risk during the study.

A specialist has confirmed I can have a muscle biopsy based on my MRI results.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Participants receive one dose of ARO-DUX4 or placebo

1 week

1 visit (in-person)

Treatment Part 2

Participants receive 4 doses of ARO-DUX4 or placebo

4 weeks

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• ARO-DUX4

Trial Overview

The trial is testing ARO-DUX4, which participants will receive either once or four times depending on the part of the study they're in. There's also a placebo group for comparison. The drug's safety, how it affects the body, and its movement through the body are being studied.

How Is the Trial Designed?

Group I: ARO-DUX4 Experimental Treatment 1 Intervention

ARO-DUX4 for Injection

Group II: Placebo Placebo Group 1 Intervention

(0.9%NaCl)

Who Is Running the Clinical Trial?

Arrowhead Pharmaceuticals

Lead Sponsor

Trials

45

Recruited

6,200+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06131983

NCT06131983 | Study of ARO-DUX4 in Adult and ...

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with ...

2.

arrowheadpharma.com

arrowheadpharma.com/news-press/arrowhead-presents-preclinical-data-on-aro-dux4-at-fshd-society-international-research-congress/

Arrowhead Presents Preclinical Data on ARO-DUX4 at ...

Importantly, in a transgenic mouse model of FSHD, treatment with ARO-DUX4 prevented and reversed tamoxifen-induced increases in DUX4 and DUX4 target gene ...

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11354670/

Oligonucleotide Therapies for Facioscapulohumeral Muscular ...

As a result, outcome measures concerning treatment efficacy tend to vary between studies. While DUX4 mRNA levels are usually measured to determine post- ...

4.

afm-telethon.fr

afm-telethon.fr/sites/default/files/media/documents/Advances_FSHD.pdf

Advances 2025 in facioscapulohumeral muscular dystrophy

In mouse models of FSHD, treatment with. ARO-DUX4 reduced DUX4 mRNA and DUX4 target gene expression. Their weight, muscle atrophy and muscle ...

5.

mdaquest.org

mdaquest.org/simply-stated-updates-in-facioscapulohumeral-muscular-dystrophy-fshd/

Simply Stated: Updates in Facioscapulohumeral Muscular ...

In November 2024, Sarepta acquired the rights to ARO-DUX4 and will lead its future phase 3 development and eventual commercialization.

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8699294/

DUX4 Role in Normal Physiology and in FSHD Muscular ...

Here, we summarize current knowledge on the cellular and molecular processes regulated by DUX4 with a special emphasis on FSHD muscular dystrophy.

7.

musculardystrophynews.com

musculardystrophynews.com/news/early-data-experimental-dm1-therapy-srp-1003-coming-soon/

Early data for experimental DM1 therapy SRP-1003 ...

Arrowhead has also developed another siRNA therapy called ARO-DUX4, which is designed to knock down production of the DUX4 protein in skeletal ...