Who is conducting the clinical trial?
Scholar Rock is conducting the study.
What is the purpose of the clinical trial?
FORGE is a Phase 2 study of an investigational drug called apitegromab (aka SRK-015) in participants living with FSHD. The purpose of the study is to:
How does the investigational drug work?
FSHD causes muscle cells to die. Apitegromab blocks a protein in the muscles called myostatin. Myostatin controls muscle growth. Therefore, blocking myostatin may help muscles grow. Apitegromab is known as a monoclonal antibody, which is a type of drug designed to target a specific protein in the body.
Will some people be given a placebo instead of the medicine?
Yes. This is a placebo-controlled study. Half of the participants will receive a placebo and not apitegromab. Placebo is administered via intravenous (IV) infusion at the same treatment schedule as apitegromab but does not contain any active ingredient.
Will I know whether I am receiving the medicine or the placebo?
No, participants will not know. This is a double-blinded study. The participants and the study team will not know who receives apitegromab and who receives the placebo.
How will the investigational drug be given?
Apitegromab and the placebo will be given as an intravenous (IV) infusion. This means apitegromab is given through a needle in a blood vein, usually in a participant’s arm. Each infusion will take 1-2 hours. Participants will receive 13 infusions over 52 weeks.
How long is the clinical trial?
Participants will receive 13 infusions of the investigational drug or placebo for 52 weeks. Following this period, participants will have the option to take part in an open-label extension. An open-label extension is an additional period where all participants receive the investigational drug, even if they were previously on the placebo. If participants do not take part in the extension, participants would do an additional 20 weeks of follow up visits to the study site.
Has apitegromab been studied before?
Yes. Before the FORGE study in FSHD, apitegromab was evaluated extensively in a clinical development program for spinal muscular atrophy (SMA).
Apitegromab has been studied for more than four years in children and adults living with SMA, representing more than 916 patient-years of clinical experience. In the Phase 3 SAPPHIRE trial, 99% of participants completed the study, and no participants discontinued treatment because of adverse events.
What are the criteria to be included in the clinical trial?
Participants must:
What criteria would exclude me from the clinical trial?
Participants will not be able to take part if participants:
Do I need to have a genetic test to be included? If so, will this be provided?
Yes, participants must have a genetic test.
We recommend participants work with their 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, so starting the process early may be relevant in the enrollment process. Learn more about genetic testing.
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 the study site will call participants. After this initial call, the coordinator may have additional pre-screening steps for participants to complete. If participants are a good fit, the coordinator will schedule an in-person screening visit.
The screening visit will take place at a study site. At this visit, participants will learn more about the study and provide their consent to proceed. Then, the study team will perform all the tests needed to determine if participants can take part in the study. This could take several hours, or participants may need multiple visits. After all test results come back, the study team will tell participants whether they can take part in the study. This screening process can take about 4 weeks.
What activities will participants be asked to do?
How many study visits are there? Are they in-person or virtual?
If enrolled, participants will attend a study visit every 4 weeks to receive the IV infusion. In addition to receiving the IV infusion, participants will also have tests and assessment done to monitor their health and response tothe IV infusion. Most study visits last 3 to 5 hours. Visits that include MRIs may take longer. The study team will give participants a schedule, so participants know what to expect at each visit.
Will I have to stay overnight for any visits?
Some visits may require overnight stays, and Scholar Rock will reimburse hotel stays and expenses for up to 1 night.
Will apitegromab be offered to patients once the clinical trial is over?
Yes, after 52 weeks of treatments, participants will have the option to take part in an open-label extension. This is an additional period where all patients receive the investigational drug, even if they were previously on the placebo. Participants can decide whether they 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, all costs are paid for by Scholar Rock.
Will I be reimbursed for travel costs?
Yes, Scholar Rock will provide travel and reimbursement for expenditures related to travel to the study sites.
Will I be paid a stipend for being in the clinical trial?
Clinical trial participants will not be provided stipends.
Who is paying for the clinical trial?
Scholar Rock is paying for the study.
How many volunteers are needed?
The study needs 60 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?
Check the FORGE study website (scholarrockforge.com) or clinicaltrials.gov for the location of participating sites and eligibility criteria.
Although all study locations are not yet operational, there are expected to be approximately 20 sites across North America and Europe. To stay up to date on the trial locations, visit clinicaltrials.gov and go to “Contacts and Locations.”
How do I contact a study site to learn more and sign up?
To find specific sites, visit clinicaltrials.gov and click on “Contacts and Locations” in the sidebar to open up a list of locations.
If a 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.” Interested individuals should email and call these contacts and let them know that they are interested in the FORGE study.
What will happen after I contact the 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 participants.
Study website:
Press releases and blog posts:
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