FORTITUDE - Avidity

TRIAL NAME: Phase 1/2 Study of Del-brax in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)  

ABBREVIATION: FORTITUDE™

STATUS: Recruiting

See Study Website
See on ClinicalTrials.gov

THE BASICS 

Who is conducting the clinical trial?
Avidity Biosciences is conducting the study. 

What is the purpose of the clinical trial? 
FORTITUDE is a Phase 1/2 study of a medicine called Del-brax. The purpose of the study is to: 

  • Understand how Del-brax acts in the human body 
  • Evaluate how safe Del-brax is and what the side effects of taking it might be 
  • Test whether Del-brax can slow or stop the progression of FSHD 

How does the medicine work?
FSHD is caused by expression of the DUX4 gene in muscles. Del-brax reduces the expression of DUX4. Del-brax 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?
Del-brax 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. You will receive 5 infusions over 9 months.  

How long is the clinical trial? 
Patients will take the medicine (or placebo) for 9 months, then do follow-up for up to 3 months. At this time, you will have to option to take part in an open-label extension. If you do not take part in the extension, you would do an additional 6 months of follow-up. Your part in the study would take about 1.5 years. 

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

ELIGIBILITY  

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

  • have FSHD1 or FSHD2  
  • be between 16 and 70 years old 
  • be able walk 10 meters (with or without assistive devices) 
  • have weakness in both your upper and lower body 
  • be able to undergo an MRI.  

 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 Early-Onset 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 this for 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?
Yes, maybe. After 9 months of treatment and 3 months of follow-up, you may be able to take part in an open-label extension. This is an additional period where all patients receive the medicine, even if you were previously on the placebo. The study team will let you know if this is an option for you. You can decide whether you want to do the open-label extension.   

 

COSTS AND PAYMENTS 

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

Will I be reimbursed for travel costs?
Yes. Avidity 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?
Avidity Biosciences is paying for the study. 

 

HOW TO SIGN UP 

How many volunteers are needed? 
The study needs 72 patients. However, many more volunteers are needed because not everyone who volunteers will be eligible and able to take part. 

Where are the study sites that are conducting the clinical trial? 
The study is conducted across multiple locations in the United States, as well as one site in Canada and one in the Netherlands. To find specific sites, visit clinicaltrials.gov and go to "Contacts and Locations." More locations may open as the study progresses.   

How do I contact my study site to learn more and sign up? 
To find specific sites, visit clinicaltrials.gov and click on the triangle shape to open up a list of current locations.  

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 FORTITUDE 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