MANOEUVRE - Roche
TRIAL NAME: A Study to Evaluate RO7204239 in Participants with Facioscapulohumeral Muscular Dystrophy
STATUS: Some Sites Enrolling
Who is conducting the clinical trial?
Hoffman La-Roche (also referred t as Roche or Roche Genentech) is conducting the study.
What is the purpose of the clinical trial?
MANOEUVRE is a Phase 2 study of a medicine called RO7204239 (also called GYM329). The purpose of the study is to:
- Test whether RO7204239 can help muscles grow
- Evaluate how safe RO7204239 is and what the side effects of taking it might be.
How does the medicine work?
FSHD causes muscle cells to die. RO7204239 blocks a protein in the muscles called myostatin. Myostatin controls muscle growth. Therefore, blocking myostatin may help muscles grow. RO7204239 belongs to a class of medicines called antibody therapies.
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?
RO7204239 and the placebo will be given as an injection under your skin, using a small needle in your abdomen. You will receive an injection every 4 weeks for 1 year.
How long is the clinical trial?
Patients will complete a 4-week pre-treatment period to collect movement data with a wearable device. Then, patients will take the medicine (or placebo) for 1 year. At this time, you will have the option to take part in an active treatment extension. If you do not take part in the extension, you part in the study would take about 13 months.
Please see “Will the medicine be offered to patients once the clinical trial is over?” for more information about the extension.
What are the criteria to be included in the clinical trial?
- have FSHD1 or FSHD2
- have a genetic test confirming your FSHD
- be between 18 and 65 years old
- be able walk unassisted
- be able to undergo an MRI (magnetic resonance imaging)
- agree to not change your exercise routines during the study.
The study team will assess you for:
- the severity of your FSHD, using the Ricci Clinical Severity Scale.
What criteria would exclude me from the clinical trial?
You will not be able to take part if you:
- Have certain medical conditions, such as cardiovascular disease or history of cancer
- Take certain medications, such as creatine, growth hormones, or steroids
- 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 team may be able to help you get one. However, the study will not pay for your genetic test.
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 will likely give priority to patients who are already tested, or who have already started the process. You can learn more about genetic testing HERE
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 the study. This screening process can take up to 4 weeks.
WHAT TO EXPECT IF YOU ENROLL
What activities will I be asked to do?
- Provide blood samples
- Undergo 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
- Wear a digital device for periods of 4 weeks at a time to measure movement
How many study visits are there? Are they in-person or virtual?
You will attend a study visit every 4 weeks to receive your medicine. You will also attend longer study visits every 6 months. Sometimes, these longer visits can be broken down to several shorter visits over a couple days. This will depend on your study site and what you prefer.
Will I have to stay overnight for any visits?
An overnight stay is not required for the study. However, if you are traveling from a long distance or prefer to break a longer visit into several shorter visits, you may decide to stay overnight in a hotel near your study site. You should talk to your study team about your travel needs.
Will the medicine be offered to patients once the clinical trial is over?
Yes. After 4 weeks of pre-treatment and 52 weeks of treatment, you will have the option to take part in an active treatment extension. This is an additional period where all patients receive the medicine, even if you were previously on the placebo. You can decide whether you want to do the active treatment 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 Hoffman La-Roche.
Will I be reimbursed for travel costs?
Yes, you will be reimbursed for travel costs needed to attend study visits. However, this will depend on local regulations. You should talk to your study team about your travel needs. Roche is committed to helping patients with travel costs.
Who is paying for the clinical trial?
Hoffman La-Roche is paying for the study.
HOW TO SIGN UP
How many volunteers are needed?
The study needs 48 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 a few locations in Denmark, Italy, the United Kingdom, and the United States. To find specific sites, visit the Roche For Patients website or clinicaltrials.gov and click on the triangle shape to open up a list of 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 the Roche For Patients website or 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 MANOEUVRE 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.