Avidity’s FORTITUDE-3 Trial

¿Quién lleva a cabo el ensayo clínico?
Avidity Biosciences está llevando a cabo el estudio. 

What is the purpose of the clinical trial?
FORTITUDE is a Phase 3 study of a medicine called Del-brax. The purpose of the study is to: 

• Understand how Del-brax acts in the human body 
• Gather essential information on potential side effects and risks of Del-brax
• 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. Half of the patients will receive a placebo and not the medicine. 

¿Sabré si estoy recibiendo el medicamento o el 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. 

¿Cómo se administrará el medicamento?
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 13 infusions over 72 weeks. 

¿Cuánto tiempo dura el ensayo clínico?
Patients will take the medicine (or placebo) for 72 weeks, then do a follow up assessment after 6 weeks. At this time, you will have the option to take part in an open-label extension. If you do not take part in the extension, you would do an additional 3 months of follow up. 

Please see “What to Expect if You Enroll” for more information about the extension. 

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

• tener FSHD1 o FSHD2
• tener entre 16 y 70 años
• be able walk 10 meters at pre-specified walking speed (orthoses and ankle braces allowed)
• have sufficient total muscle strength, which the study team will assess

What criteria would exclude me from the clinical trial?
No podrás participar si: 

• Have high blood pressure (>140/90 mmHg at screening)
• Está embarazada o planea quedar embarazada.
• Hay otros factores que pueden excluirlo. Puede consultar más criterios de exclusión en ensayos clínicos.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.  

We recommend you work with your doctor to start the process of getting a genetic test before trying to join this clinical trial. Genetic testing can take many weeks to complete. Because of this, the study team may give priority to patients who are already tested, or who have already started the process. Más información sobre las pruebas genéticas. 

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 activities will I be asked to do?

• Proporcionar muestras de sangre y orina.
• Realice una serie de pruebas para evaluar su fuerza y función muscular.
• Responda cuestionarios sobre los síntomas relacionados con la FSHD y cómo se siente.
• 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.

¿Cuántas visitas de estudio hay? ¿Son presenciales o virtuales?
You will be asked to attend ~20 study visits. Some of these may be done virtually. 

Will I have to stay overnight for any visits?
No. 

Will the medicine be offered to patients once the clinical trial is over?
Yes, maybe. After 72 weeks of treatments and 12 weeks 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. 

Will I have to pay for any medical expenses? Will my insurance be billed for any costs? 
No, todos los gastos corren a cargo de Avidity Biosciences. 

Will I be reimbursed for travel costs?
Sí. Avidity se encargará de organizar y pagar los pasajes aéreos, el alojamiento y el transporte terrestre para usted y un cuidador. También se le pueden reembolsar los gastos razonables relacionados con el estudio, como comidas y estacionamiento. 

Will I be paid a stipend for being in the clinical trial?
Travel reimbursement is offered as part of this study for both the patient and caregiver. In addition to reimbursement for travel expenses, some study sites may have the ability to pay a per visit stipend payment. 

¿Quién paga el ensayo clínico?
Avidity Biosciences está financiando el estudio. 

¿Cuántos voluntarios se necesitan?
The study needs 200 participants. 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 will be conducted across multiple locations in the United States, United Kingdom, Canada, Japan, Italy, France, Germany, Spain, Denmark, and Netherlands. To find specific sites, visit ensayos clínicos.gov and go to “Contacts and Locations” or visit the study website and go to “Locations.” More locations may open as the study progresses. 

How do I contact my study site to learn more and sign up?
If you are interested in participating or if you have questions about the study, please have your physician contact medinfo@aviditybio.com. 

Additionally, if you know which study site(s) you would be able and willing to travel to, you could use a regular search engine (e.g. Google) to look up the name of the study site along with terms like “neuromuscular clinical trials” or “FSHD clinical trials” to try to find contact information for that site specifically. If there is more than one site that you could travel to, 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. 

• Cartera de productos de Avidity Biosciences para la FSHD
• Avidity comparte datos provisionales prometedores de su ensayo FORTITUDE.
• Avidity Biosciences finaliza la inscripción en la cohorte de biomarcadores del ensayo FORTITUDE™ de fase I/II.
• Resultados prometedores del ensayo clínico de Avidity sobre la distrofia miotónica
• Conoce a del-brax, el candidato terapéutico de Avidity para la FSHD.