ARO-DUX4 - Arrowhead

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

ABBREVIATION:

STATUS: Recruiting

See on ClinicalTrials.gov

THE BASICS 

Who is conducting the clinical trial?
Arrowhead Pharmaceuticals is conducting the study. 

What is the purpose of the clinical trial? 
This is a Phase 1/2a study of a medicine called ARO-DUX4. The purpose of the study is to:

  • Understand how ARO-DUX4 acts in the human body 
  • Evaluate how safe ARO-DUX4 is and what the side effects of taking it might be 

How does the medicine work?
FSHD is caused by expression of the DUX4 gene in muscles. ARO-DUX4 reduces the expression of DUX4. It belongs to a class of medicines called RNA therapeutics. 

Will some people be given a placebo instead of the medicine?
Yes. This is a placebo-controlled study. Some patients will receive a placebo and not the medicine. 

Will I know whether I am receiving the medicine or the placebo?
No, you will not know. This is a double-blinded study. The patients and the study team will not know who receives the medicine and who receives the placebo. 

How will the medicine be given?
ARO-DUX4 and the placebo will be given as an intravenous (IV) infusion. This means the medicine is given through a needle in a blood vein, usually in your arm. Each infusion could take 1 to 4 hours. In Part 1 of the study, participants will receive a single dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 2 or 4 doses of ARO-DUX4 or placebo.  

How long is the clinical trial? 
In Part 1, participants will be monitored for adverse events for 90 days. In Part 2, participants will be monitored for adverse events for 360 days.

Please see “Will the medicine be offered to patients once the clinical trial is over?” for more information about the extension study. 

ELIGIBILITY  

What are the criteria to be included in the clinical trial? 
You must: 

  • be between 18 and 70 years old 
  • be genetically confirmed FSHD1 based on screening evaluation or source verifiable medical record
  • have clinical severity score between 3 and 8 (scale, 0 to 10)
  • have an 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

 The study team will assess you for: 

  • muscle weakness in both your upper and lower body 
  • an area of muscle that can undergo a biopsy. 

What criteria would exclude me from the clinical trial?  

You will not be able to take part if you: 

  • Were diagnosed with infantile FSHD 
  • Have certain medical conditions, such as obesity, bleeding disorders, or other skin or muscle conditions 
  • Are pregnant or plan to become pregnant 
  • Other factors may exclude you. You can view more exclusion criteria on clinicaltrials.gov. 

Do I need to have a genetic test to be included? If so, will this be provided?
Yes, you must have a genetic test. If you don’t have one, the study will be able to provide the test at no cost to you. 

How will the study team determine whether I can take part in the study? 
The first step is usually a pre-screening phone call to talk through the eligibility criteria. A coordinator at your study site will call you. After this initial call, your coordinator may have additional pre-screening steps for you to complete. If you are a good fit, your coordinator will schedule an in-person screening visit.  

Your screening visit will take place at a study site. At this visit, you will learn more about the study and provide your consent to proceed. Then, the study team will perform all the tests needed to determine if you can take part in the study. This could take several hours, or you may need multiple visits. After all your test results come back, the study team will tell you whether you can take part in study. This screening process can take up to 6 weeks. 

 

WHAT TO EXPECT IF YOU ENROLL 

What activities will I be asked to do?  

  • Provide blood and urine samples 
  • Provide 2 muscle biopsy samples. This involves using a needle to draw a small piece of muscle, about the size of a piece of long-grain rice. The procedure is done with a local anesthetic 
  • Undergo whole-body MRI scans 
  • Perform a series of tests to assess your muscle strength and function  
  • Answer questionnaires about symptoms related to FSHD and how you are feeling 
  • Use highly effective contraception. If you have the ability to get pregnant, you will take regular pregnancy tests to confirm that you are not pregnant. 

How many study visits are there? Are they in-person or virtual?
You will be asked to attend ~20 study visits. Some of these can be done virtually.  

Will I have to stay overnight for any visits?
Some patients may be asked to stay overnight. Others may need to return to the study site 24 hours after their infusion for follow-up. 

Will the medicine be offered to patients once the clinical trial is over?
Possibly. 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.

 

COSTS AND PAYMENTS 

Will I have to pay for any medical expenses? Will my insurance be billed? 
No, all costs are paid for by Arrowhead Pharmaceuticals.

Will I be reimbursed for travel costs?
Yes. Arrowhead Pharmaceuticals will arrange and pay for airfare, lodging, and ground transportation for you and one caregiver. You may also be reimbursed for reasonable expenses related to the study, such as meals and parking. 

Who is paying for the clinical trial?
Arrowhead Pharmaceuticals is paying for the study. 

 

HOW TO SIGN UP 

How many volunteers are needed? 
The study seeks 52 volunteers with FSHD who meet the eligibility criteria.

Where are the study sites that are conducting the clinical trial? 
The study will be conducted in New Zealand. Additional global sites are being considered. Please check ClinicalTrials.gov for additional sites in the future.

How do I contact my study site to learn more and sign up? 

To find specific site contact information, visit clinicaltrials.gov.  

If your study site is marked as “Recruiting,” there should be an email and/or phone number listed for contact. Sometimes there is also contact information listed for sites that are “Not Yet Recruiting.” You should email and call these contacts and let them know that you are interested in the ARO-DUX4 study. If you are able and willing to travel to multiple different sites, you should contact each site individually. 

What will happen after I contact my study site? 
It can take several weeks or months to hear back, so be patient. Sites could still be in the process of getting ready for the study and may not be able to contact anyone just yet. Sometimes, sites are overloaded with more interested patients than they can enroll in the study. Make sure you provide your email and phone number to make it easier for the coordinators to contact you. You should also keep checking in on a monthly basis – this helps the study team know that you are committed to completing the clinical trial. 

 

MORE INFORMATION