Medical science has made exciting progress in recent years in treating desmoid tumors (also known as aggressive fibromatosis). Thanks to clinical trials, new therapies are available for people with these rare tumors. Clinical trials are research studies with volunteer participants that help determine if a new treatment may be more effective than existing ones while minimizing side effects.

Every new drug and treatment method that is approved by the U.S. Food and Drug Administration (FDA) must undergo a clinical trial to determine if it is safe and effective. New drugs, new drug combinations, new ways to use an existing drug, and other types of therapies are all studied in clinical trials. Therapies that are studied in clinical trials are known as investigative drugs or investigative treatments.

If you have a desmoid tumor and are curious about whether a clinical trial may be right for you, here’s some important information that you can discuss in more detail with your doctor.

1. Clinical Trials Are Leading to New Desmoid Tumor Treatment

Desmoid tumors are a rare disease in which benign (noncancerous) tumors form in the body’s soft tissues. Thanks to clinical trials, new treatments for desmoid tumors have emerged that have expanded the range of therapy options for the condition.

In November 2023, the FDA approved the first medication for desmoid tumors, a targeted therapy called nirogacestat (Ogsiveo). Participants in a clinical trial who took the drug showed significant improvement in disease activity and progression-free survival (PFS). PFS refers to the length of time during and after treatment that a person lives with the disease without it worsening.

In some cases, clinical trials have shown that existing cancer drugs such as sorafenib tosylate (Nexavar) can be effective for the treatment of desmoid tumors. This drug is a type of targeted therapy called a multikinase inhibitor. Similar to tyrosine kinase inhibitors (TKIs), it targets specific proteins known as tyrosine kinases that play a role in desmoid tumor growth — along with other proteins. It’s currently used off-label to treat desmoid tumors. An off-label drug is a treatment that has been approved by the FDA for certain conditions, but may be prescribed for another disease if there is evidence that it might be an effective treatment.

A number of other potential therapies for desmoid tumors are currently being studied in clinical trials, including:

• New drugs, such as AL102 and tegavivint — These drugs target proteins involved in desmoid tumor growth, aiming to disrupt cellular pathways driving tumor proliferation​​​.
• Nirogacestat with cryoablation — Cryoablation therapy uses very cold temperatures to destroy tumors. Nirogacestat, a gamma-secretase inhibitor, is also being explored in other clinical trials.
• High-intensity focused ultrasound (HIFU) ablation — HIFU uses precise sound waves to heat and destroy tumor tissue, offering a noninvasive option for treating small intra-abdominal (inside the abdomen) desmoid tumors​​.

The U.S. government has a database that lists current clinical trials for people with desmoid tumors.

2. Clinical Trials Potentially Provide Access to New Treatments

In clinical trials, people with specific conditions receive investigational treatments to evaluate their effectiveness. These new therapies, not yet available to the public, may be more effective than current options.

However, there is no guarantee the treatment will work, and all medications carry the risk of side effects.

It’s also important to know that some people who participate in a clinical trial may not receive the new treatment. Some people are given a placebo. A placebo, sometimes called a “sugar pill,” is designed to look like the treatment being studied but doesn’t have any active ingredients. To test the effectiveness of an investigational treatment, clinical trials may assign a group of participants a placebo in order to compare results. This is known as a placebo-controlled study.

Notably, in some clinical trials, participants who initially receive a placebo may later receive the actual treatment.

3. Clinical Trial Participants Are Told if They Might Receive a Placebo

In every clinical study, participants are informed if there is a chance they might receive a placebo, rather than the treatment under study. This information may affect whether or not you want to participate in a clinical trial. In some cases, participants may be given an existing treatment rather than the investigational treatment.

To avoid bias, researchers decide at random which participants receive the investigational treatment versus the placebo group or existing treatment. This process is called randomization. Many research studies are also double-blind (or double-masked), meaning neither the participants nor the researchers know which treatment each participant is receiving until the trial is over, a participant needs to switch treatments, or a participant leaves the trial for any reason.

In some clinical trials, if the investigational treatment shows significantly better results than the placebo or comparison treatment, it may be offered to all participants during the study. When considering a clinical trial, it's important to ask about the study design and whether you could access the investigational treatment if it proves effective.

4. Clinical Trials Are Designed To Protect Participants’ Safety

Clinical trials are voluntary, and participants may leave a clinical study at any time for any reason. In the U.S., clinical trials are carefully regulated by the federal government to minimize risk. Before any trial begins, researchers conduct extensive laboratory and animal tests to ensure a new treatment’s safety and effectiveness. Only if these tests show promising results can a study proceed to a clinical trial.

Institutional Review Boards Oversee Clinical Trials

Most trials are reviewed by an institutional review board (IRB), which includes scientists, doctors, and other experts. The IRB ensures that potential benefits outweigh the risks. If a trial becomes unsafe, it will be stopped immediately.

Informed Consent

Before joining a clinical trial, participants must undergo an informed consent process, where researchers explain the study, its risks, and procedures. You’ll have time to ask questions and consult with your family, doctor, or advisers before deciding. Throughout the study, you will receive updates and information.

Your doctor can help you determine the types of questions you should ask when considering participating in a clinical study, including if you will undergo procedures such as biopsy, and if there are any costs you may incur for travel or other expenses.

Phases of Clinical Trials

Clinical trials are conducted in different phases, which are staggered to ensure participant safety while evaluating the treatment's effectiveness and potential risks. The phases are as follows:

• Phase 1 focuses on determining the highest dose of a new drug that can be administered safely. These studies involve small groups of participants, usually around 20 to 100 people.
• Phase 2 trials test the drug’s effectiveness on a specific disease and monitor side effects. They involve larger groups of participants than phase 1 trials, around 100 to 300.
• Phase 3 and 4 trials compare the new treatment to standard ones and evaluate additional benefits, such as quality of life improvements. During these phases, there may be several hundred or thousands of participants.

Early trial phases may last several months to two years, while later phases can continue for four years or longer.

5. People Have Different Reasons for Participating in a Clinical Trial

Participating in a desmoid tumor clinical study is a personal decision. Some people participate because they have a chance of receiving a new therapy that may improve their condition and quality of life. There may also be financial considerations as well because the sponsors of clinical trials — universities, drug companies, foundations, or individuals — may cover the costs of the investigational treatment. Health insurance may also cover some of the costs.

Others participate in clinical trials because they want to contribute to research that may lead to new breakthroughs in desmoid tumor treatment options. Some are motivated by the additional care, monitoring, and follow-up provided in clinical studies, along with the opportunity to gain fresh insights into their condition.

6. Each Clinical Trial Has Eligibility Requirements

Every clinical trial has specific criteria that determine who can participate. Factors including age, gender, disease stage, and medical history may impact eligibility.

The Desmoid Tumor Research Foundation (DTRF) has more information on clinical trials. You can talk to your doctor to find out if a clinical trial may be appropriate for you.

Find Your Team

On MyDesmoidTumorTeam — the online social network for people with desmoid tumors and their loved ones — more than 300 members come together to ask questions, give advice, and share their stories with others who understand life with desmoid tumors.

Have you participated in a clinical trial? Do you have any tips for people who are thinking about joining a clinical study? Share your experience in the comments below, or start a conversation by posting on your Activities page.