A Closer Look: Current and Upcoming Clinical Trials - Desmoid Tumor Research Foundation

A Closer Look: Current and Upcoming Clinical Trials

To view all clinical trials in desmoid tumors, visit here.
Below we’ve compiled additional information about a few open and upcoming clinical trials.



Open Clinical Trials

Phase II DART Immunotherapy

Despite their name, rare cancers make up more than 20 percent of cancers diagnosed worldwide. DART is a clinical trial testing new treatments for dozens of rare cancers. Desmoid tumor is one of the tumor types included in this trial.

The two drugs under study are new FDA-approved immunotherapies. Immunotherapies are a new class of cancer drugs designed to help a patient’s own immune system fight their cancer. DART is the first federally funded immunotherapy trial devoted to rare cancers. DART stands for Dual Anti-CTLA-4 & Anti-PD-1 blockade in Rare Tumors.

Rare cancers are those with less than a 6 in 100,000 incidences per year, according to the definition used for the DART trial. But only certain rare cancer patients can enroll in DART. Prospective patients submit tumor tissue for a genetic test, and doctors will use the test results to see if a patient’s type of cancer is covered under the DART trial. Doctors will also screen patients using other eligibility criteria, including the stage of their disease, their general health, and any prior treatment.

Once enrolled in DART, patients are treated with two immunotherapy drugs under the brand names Yervoy (ipilimumab) plus Opdivo (nivolumab). The FDA approved the combination to treat melanoma, and the combination is currently being tested on a variety of lung cancers. DART patients will receive both drugs. There is no placebo.

DART investigators want to determine if this combination, given in six-week cycles, can significantly shrink tumors. In addition, a basic science team will use patients’ tumor tissue samples to study how immune cells and genes respond to the drug combination, and see if there are any substances that can be measured in their tissue sample that may predict treatment response. Trial leaders plan to enroll 300 patients.

Rare cancer patients should discuss with their doctor whether DART is right for them. For study eligibility and locations, visit the link below. Please contact raretumors@crab.org with any questions.


MR-guided High Intensity Focused Ultrasound (HIFU) for Patients Under 30 Years Old

Children’s National Health System has an open clinical trial using magnetic resonance-guided high-intensity focused ultrasound (MR-HIFU), a noninvasive technology, to destroy solid tumors including desmoid tumors in children, adolescents, and young adults. To be eligible for the clinical study, patients need to be 30 years or less with refractory or relapsed solid tumors including desmoid tumors with measurable lesions that are located in bone or soft tissue in close proximity to bone. To learn more or to enroll in the trial, please see http://www.ignite4kids.org or contact Emily Stern, RN at 202-476-2802 (email:  EStern@childrensnational.org) or AeRang Kim, MD, PhD at 202-476-2800 (email: aekim@childrensnational.org).

More about HIFU clinical trials


Upcoming Clinical Trials

Phase III Trial for Nirogacestat


A new company named SpringWorks Therapeutics has been formed as a spinoff of Pfizer to focus on investigational therapies that hold 
significant promise for underserved populations. 

SpringWorks will now take the gamma-secretase inhibitor that showed promising results in treating desmoid tumors in Phase I and Phase II trials, into a Phase III trial. Formerly known as PF-03084014, the drug is now named Nirogacestat (pronounced “nye-roe-gay-se-stat”). 

We have been working for several years to bring this drug to patients. We will continue working collaboratively with SpringWorks Therapeutics and the investigator community as they design Phase III trials that serve both adult and pediatric desmoid patients.

No details have been announced about the trials or their timing. Rest assured that we will keep you informed as soon as any information is released in the upcoming months.

Please see the press release that provides further details about this exciting development.


Phase I Trial for Tegavivint 

At our 11th DTRF Annual Patient Meeting in Philadelphia, we were pleased to have Beta Cat Pharmaceuticals’ Jon Northrup, Chief Executive Officer, and Casey Cunningham, Chief Medical Officer, present an outline of the planned clinical trial of Tegavivint, a new drug that directly interferes with beta-catenin stabilization. Targeting beta-catenin in this way may hold promise for desmoid sufferers, as the science that has evolved shows that desmoid tumors are due to mutations in the beta-catenin gene, CTNNB1, which cause improper degradation of beta-catenin. Tegavivint has shown promise in preclinical studies of desmoid tumors and this trial will be the drug’s first trial in patients.

Beta Cat anticipates clinical sites at the Harvard Hospital system, Memorial Sloan Kettering in NYC, Northwell Hospital on Long Island, Princess Margaret in Toronto, Canada, University of Washington in Seattle, and MD Anderson in Houston.

We will keep you posted as more details are finalized for this trial. Until then, we recommend watching the video of Dr. Sunil Sharma (University of Utah’s Huntsman Cancer Institute) discussing the drug at last year’s Patient Meeting and perusing the slides from Beta Cat Pharmaceuticals.



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