Ongoing Clinical Trials for Desmoplastic Small Round Cell Tumour
There are currently 2 ongoing clinical trials investigating new treatment options for desmoplastic small round cell tumour. These studies are being conducted in Germany, Spain, and Italy, and are testing different medications aimed at slowing disease progression and improving outcomes for patients with this rare and aggressive cancer.
Clinical trial locations
- Germany
- Italy
- Spain
Study on Pasireotide for Patients with Synovial Sarcoma and Desmoplastic Small Round Cell Tumor
This trial is investigating pasireotide, a medication given as a monthly injection into the muscle, as a maintenance treatment. The study is taking place in Germany and is expected to continue until October 2028.
Main inclusion criteria: Patients must be between 13 and 50 years old, weigh at least 30 kg, and have a confirmed diagnosis of desmoplastic small round cell tumour. They must have completed at least 2 cycles of chemotherapy within the last 8 weeks and show stable disease or improvement after standard treatment. The tumor must have high levels of specific markers called SSTR2/3/5, which are tested through RNA sequencing. Patients must have adequate bone marrow, kidney, and liver function. Those who can have children must agree to use two forms of birth control during the study and for 3 months afterward. Patients must have no curative treatment options available and be able to perform daily activities relatively well.
Main exclusion criteria: Patients without locally advanced or metastatic disease cannot participate. Those whose tumors do not express the SSTR2/3/5 proteins are also excluded. The study has specific age range requirements, and patients outside these limits are not eligible.
Study focus: The trial aims to determine whether pasireotide can help patients live longer without their disease getting worse. It measures progression-free survival from the start of treatment until disease progression or death. The medication is administered once monthly through deep intramuscular injection. Patients are monitored regularly with imaging tests to check for disease progression throughout the study period.
Investigational drug: Pasireotide is a somatostatin analog that works by binding to somatostatin receptors on tumor cells. This helps inhibit the release of growth factors that promote tumor growth. The medication is being tested specifically in tumors that express SSTR2, SSTR3, and SSTR5 receptors.
Study of lurbinectedin and irinotecan in adults and young adults with advanced desmoplastic small round cell tumor
This clinical trial is testing the combination of two medications, lurbinectedin and irinotecan, given through intravenous infusion. The study is being conducted in Spain and Italy and is designed for patients whose cancer has continued to grow despite previous treatments.
Main inclusion criteria: Patients must be at least 15 years old and have a confirmed diagnosis with the specific EWSR1-WT1 genetic translocation. They must have advanced cancer that cannot be completely removed by surgery or has spread to other parts of the body. The cancer must be measurable on scans. Patients should have received between 1 and 3 previous chemotherapy treatments, including at least one containing anthracyclines. They must have recovered from most side effects of previous treatments, except for mild tiredness, hair loss, or skin problems. Adequate blood cell counts, liver function, kidney function, and blood clotting are required. Heart function must be good, with an ejection fraction of at least 50%. Women who can become pregnant must have a negative pregnancy test within 7 days before starting treatment. All participants who can have children must use effective birth control during the study and for several months after the last treatment.
Main exclusion criteria: Patients younger than 15 years old cannot participate. Those without the confirmed EWSR1-WT1 genetic mutation are excluded. Patients who have not received anthracyclines or have received more than 4 previous treatment lines are not eligible. Those with no evidence of disease progression, active infections, pregnancy or breastfeeding, or known allergies to the study medications cannot join. Patients with other serious medical conditions or those currently in another clinical trial are also excluded.
Study focus: The main goal is to see how well tumors respond to the combination of lurbinectedin and irinotecan. The study also tracks overall survival, progression-free survival, and monitors side effects. Patients complete questionnaires about their quality of life and pain levels throughout treatment. Regular blood tests, imaging scans, and health assessments are performed during treatment cycles.
Investigational drugs: Lurbinectedin is a synthetic medication that interferes with DNA processes in cancer cells by binding to DNA and disrupting cell division. It targets transcription and DNA repair mechanisms in aggressive tumors. Irinotecan is a chemotherapy drug that belongs to the topoisomerase inhibitor class. It works by interfering with cancer cell DNA replication, preventing tumor cells from dividing and multiplying. Both medications are administered intravenously and are being tested together to determine if the combination is more effective than single treatments.
Summary
Currently, 2 clinical trials are actively recruiting patients with desmoplastic small round cell tumour across three European countries. One trial is being conducted in Germany, while the other spans both Spain and Italy. The studies represent two different therapeutic approaches: one testing a single maintenance therapy with pasireotide, and another investigating a combination chemotherapy approach using lurbinectedin and irinotecan.
Notably, both trials target patients with advanced disease who have already undergone previous treatments. The pasireotide study focuses on maintaining disease stability after initial treatment response, while the lurbinectedin and irinotecan study specifically targets patients whose disease has progressed despite prior therapy. Both studies require specific genetic or molecular characteristics in the tumors, reflecting the increasing importance of personalized medicine in treating this rare cancer.
The trials also differ in their age requirements, with the pasireotide study accepting younger patients from age 13, while the combination therapy study requires patients to be at least 15 years old. Both studies emphasize the need for adequate organ function and recovery from previous treatment side effects before enrollment.
