Featured Clinical Trials

This page highlights some of the clinical trials that are currently enrolling participants with rare tumors. Clinical trials are research studies that involve human volunteers. The data we gain from clinical trials are important for learning more about rare tumors and finding new treatments that work. Learn more about the different types of clinical trials.

NCI-Sponsored Clinical Trials

Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors

This study follows patients with rare solid tumors over time to learn about how rare tumors progress and how they affect quality of life. This is a MyPART study. Learn more here.

Testing Tiragolumab and Atezolizumab in SMARCB1 or SMARCA4 Deficient Tumors

This trial, led by MyPART physician Dr. Mary Frances Wedekind, will test if the immunotherapy drugs tiragolumab and atezolizumab have any effect on tumors that are missing or have changes in the genes SMARCB1 or SMARCA4 and have either come back (relapsed) or do not respond to other therapies. This trial is also testing if taking tiragolumab with atezolizumab is safe in the pediatric population. Tumors that are missing or have changes in SMARCB1 or SMARCA4 are often aggressive tumors that are difficult to treat. This trial is open to patients 12 months and older, through adulthood. Participants can enroll at the NIH Clinical Center, or at PEP-CTN core sites and non-core sites throughout the US. The trial is also open for enrollment in Quebec, Canada and Queensland, Australia. Learn more about this clinical trial here.

Testing Atezolizumab in People with Advanced Alveolar Soft Part Sarcoma

A Phase II clinical study testing the ability of the anti-PD-L1 antibody, atezolizumab, in treating patients with ASPS that has spread to other parts of the body and cannot be removed by surgery. This trial is open at many sites across the US for patients ages 14 and older. Patients age 2 and older can enroll at the NIH Clinical Center. We are currently seeking to enroll more pediatric patients at the NIH Clinical Center. Please contact Dr. Mary Frances Wedekind Malone to enroll patients age 2 through 17 by email: maryfrances.wedekindmalone@nih.gov.

Now Open to Patients Age 12 and Older: Trial for Relapsed Solid Tumors

A phase 1 study entitled "Combination Nilotinib and Paclitaxel in Adults With Relapsed Solid Tumors" is testing the safety of using both drugs, nilotinib and paclitaxel, to treat patients with solid tumors that do not show any response to treatment. In pre-clinical studies, nilotinib was found to show greater anti-tumor activity when used in combination with paclitaxel. This trial is open to people age 12 and older. To be eligible to participate in this study, patients must have completed any radiation or surgery treatments 3 weeks (or longer) prior to starting this study. To enroll patients ages 12-17, please contact MyPART physician, Dr. Mary Frances Wedekind Malone, at maryfrances.wedekindmalone@nih.gov.

KOMET Study: Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

A Phase III study enrolling adult patients ages 18 and older with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas to test the safety and efficacy of selumetinib. Selumetinib was approved in 2020 for children ages 2 years and older with NF1 and inoperable plexiform neurofibromas. The clinical trial leading to Selumetinib’s approval in the pediatric NF setting showed the drug shrank plexiform neurofibromas in 70% of pediatric patients, with many patients also reporting improvements in pain, function, and quality of life. The KOMET study is enrolling patients in the US in Florida, Maryland, Missouri, and New York, and is also enrolling patients in Australia, Brazil, Canada, China, France, Germany, Italy, Japan, Poland, Spain, and the United Kingdom. Learn more about this study here.

Other Rare Tumor Clinical Trials

TCF-001 TRACK (Target Rare Cancer Knowledge) Study (TRACK)

A study sponsored by MyPART advocacy partner TargetCancer Foundation to determine whether rare cancer patients benefit from DNA sequencing that may help point to a potential precision therapy.