Clinical Trials for Alveolar Rhabdomyosarcoma

There are currently 2 ongoing clinical trials for alveolar rhabdomyosarcoma, a rare and aggressive type of cancer that develops in skeletal muscle tissue. These trials are testing new treatment approaches including personalized peptide vaccines and combination therapies for patients whose cancer has spread or did not respond to standard treatments. Both trials are being conducted in Germany.

Clinical trial locations

• Germany
  • Study of Personalized Peptide Vaccine with PERVI-FUS, PERVI-NEO, and 11902A for Children and Young Adults with Metastatic Fusion-Driven Sarcomas
  • Study of Trabectedin alone versus Trabectedin with tTF-NGR combination therapy in adults with metastatic or refractory soft tissue sarcoma who failed first-line treatment

Study of Personalized Peptide Vaccine with PERVI-FUS, PERVI-NEO, and 11902A for Children and Young Adults with Metastatic Fusion-Driven Sarcomas

This trial is designed specifically for children and young adults with fusion-driven sarcomas that have spread to other parts of the body. The study focuses on three specific types of sarcomas: Ewing sarcoma, alveolar rhabdomyosarcoma, and synovial sarcoma.

Who can participate: Patients must have one of the three types of fusion-driven metastatic sarcoma and be in their first or second complete remission (meaning no signs of cancer) or partial response (cancer has shrunk but is still present). Participants must have finished their standard chemotherapy and local therapy. Special genetic testing of the cancer cells, including whole exome sequencing and RNA sequencing, must be available. A personalized vaccine must have been successfully created for the patient. Both male and female patients who are children or young adults can participate.

Who cannot participate: Patients who do not have fusion-driven metastatic sarcoma, are not in the specified remission or response state, or fall outside the required age range cannot join the study.

What the trial tests: The trial examines an individualized peptide vaccine made up of specific proteins (PERVI-FUS, PERVI-NEO, and 11902A). This vaccine is custom-designed for each patient based on their unique cancer characteristics. The main goal is to see if this personalized vaccine can help the body’s immune system, particularly T-cells (a type of white blood cell), recognize and attack the cancer cells more effectively. The study compares the vaccine to a placebo (an inactive substance) and monitors participants for up to 113 days of treatment, measuring immune response, side effects, and overall health.

Investigational drug: The individualized peptide vaccine is an emulsion for injection containing small proteins called peptides that are unique to each patient’s cancer cells. The vaccine aims to boost the immune system’s ability to recognize and attack cancer cells after standard treatment has been completed.

Study of Trabectedin alone versus Trabectedin with tTF-NGR combination therapy in adults with metastatic or refractory soft tissue sarcoma who failed first-line treatment

This trial focuses on soft tissue sarcomas, including alveolar rhabdomyosarcoma, that have either spread to other parts of the body or did not respond well to previous treatments. The study compares two treatment approaches in adults.

Who can participate: Adults between 18 and 75 years old with confirmed high-grade soft tissue sarcoma (grade 2-3) of specific types, including alveolar rhabdomyosarcoma, liposarcoma, fibrosarcoma, leiomyosarcoma, angiosarcoma, synovial sarcoma, and undifferentiated sarcoma. Participants must have advanced or metastatic disease that either did not respond to previous treatment with anthracycline medications or where patients cannot take anthracyclines for medical reasons. The tumor must test positive for CD13 (a specific protein marker) and patients must have at least one measurable tumor that has not been previously treated with radiation. Participants must have a life expectancy of at least 3 months and be able to carry out daily activities. Women who can become pregnant must use effective birth control during the study and for 3 months after, and men must use birth control for 5 months after treatment.

Who cannot participate: People younger than 18 or older than 75 years, those without confirmed soft-tissue sarcoma, patients who test negative for CD13, pregnant or breastfeeding women, those with no measurable disease, patients who have previously received trabectedin, and those with severe heart, liver, or kidney problems. Patients with active uncontrolled infections, other active cancers requiring treatment, or allergic reactions to study medications are also excluded.

What the trial tests: The study aims to determine if adding tTF-NGR to standard trabectedin treatment helps keep the cancer under control for longer. One group receives only trabectedin, while another receives both trabectedin and tTF-NGR. Both medications are given through intravenous infusion directly into the bloodstream. The experimental drug tTF-NGR is designed to concentrate the chemotherapy medication inside the tumor, potentially making the treatment more effective. Treatment continues for up to 360 days, with regular medical examinations and imaging tests to monitor how the cancer responds.

Investigational drugs: Trabectedin is a cancer medication that interferes with cancer cell growth and division, used to treat soft tissue sarcoma when other treatments have not worked effectively. tTF-NGR is an experimental therapy designed to work together with trabectedin. It is a protein that targets blood vessels in tumors and helps trap the cancer medication inside the tumor, aiming to keep the medication concentrated where it is most needed.

Summary

Both ongoing clinical trials for alveolar rhabdomyosarcoma are being conducted in Germany, representing different approaches to treating this rare and aggressive cancer. The first trial focuses specifically on children and young adults with metastatic disease, using a highly personalized vaccine approach tailored to each patient’s unique cancer characteristics. The second trial addresses adult patients and includes alveolar rhabdomyosarcoma among several soft tissue sarcoma types, testing whether combining an experimental drug with standard chemotherapy can improve outcomes.

These trials reflect the challenges of treating rare cancers and the need for innovative approaches beyond standard chemotherapy. The personalized vaccine approach represents an immunotherapy strategy aimed at harnessing the body’s own immune system, while the combination therapy trial seeks to improve the effectiveness of existing treatments. Both studies require specific characteristics in participants, such as genetic testing results and tumor markers, highlighting the increasingly targeted nature of modern cancer treatment research.