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New treatment option for Facio-Scapulo-Humeral Dystrophy
Official title Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
ClinicalTrials.gov ID: NCT06131983
What this study is testing
What is ARO-DUX4 for Injection?
ARO-DUX4 for Injection is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for facio-scapulo-humeral dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 16 to 70
You may be able to join 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...
You likely can't join 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
See the full eligibility 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Liverpool, New South Wales, Australia
- Auchenflower, Queensland, Australia
- Birtinya, Queensland, Australia
- Melbourne, Victoria, Australia
- Calgary, Alberta, Canada
- Edmonton, Alberta, Canada
- Montreal, Quebec, Canada
- München, Germany
- Ulm, Germany
- Milan, Italy
- Roma, Italy
- Leiden, Netherlands
- Auckland, New Zealand
- Barcelona, Spain
- Madrid, Spain
- Valencia, Spain
- Bangkok, Thailand
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 16 years to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Liverpool, New South Wales, Australia; Auchenflower, Queensland, Australia; Birtinya, Queensland, Australia; Melbourne, Victoria, Australia; Calgary, Alberta, Canada; Edmonton, Alberta, Canada and 11 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.