Study on Trastuzumab Deruxtecan for Treating Newly Diagnosed, Recurrent, or Hard-to-Treat Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
Sponsor: National Cancer Institute (NCI)
ClinicalTrials ID:NCT04616560
This clinical trial is testing a new treatment called trastuzumab deruxtecan for patients with certain types of cancer, including osteosarcoma, Wilms tumor, and desmoplastic small round cell tumor. The study aims to see if this treatment can help patients whose cancer is newly diagnosed, has returned, or hasn't responded to previous treatments.
Patient Parameters
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Program Overview
This study is exploring the effects of a new cancer treatment called trastuzumab deruxtecan (DS-8201a) in patients with osteosarcoma, Wilms tumor, or desmoplastic small round cell tumor. These cancers may be newly diagnosed, have come back after treatment, or not responded to previous treatments. Trastuzumab deruxtecan is a type of medication known as an antibody-drug conjugate, which combines a targeted antibody with a chemotherapy drug to attack cancer cells more effectively.
Description
The study is divided into two phases. In Phase I, researchers will determine the safest dose of trastuzumab deruxtecan for children aged 2 to 12 with Wilms tumor or desmoplastic small round cell tumor that has returned or not responded to treatment. They will also monitor any side effects. In Phase II, the study will evaluate how well the treatment works in children and young adults with these cancers.
Participants will receive the treatment through an IV infusion every 21 days for up to 35 cycles, as long as the cancer does not progress and side effects are manageable. Throughout the study, patients will undergo various tests, including CT and MRI scans, heart function tests, and blood sample collections.
After completing the treatment, patients will have follow-up visits at 6 months to monitor their health and any long-term effects of the treatment.
Eligibility Criteria
Inclusion Criteria
- Phase 1: Children aged 2 to less than 12 years with recurrent or refractory Wilms tumor or desmoplastic small round cell tumor.
- Phase 2: Wilms tumor patients under 18 years old and desmoplastic small round cell tumor patients aged 2 to 39 years.
- Must have a confirmed diagnosis of Wilms tumor or desmoplastic small round cell tumor.
- Must have measurable disease according to specific medical criteria.
- Must have received standard initial therapy for their cancer.
- Must have a good performance status, meaning they are fully active or have some restrictions but can still perform light work.
- Must have recovered from any side effects of previous cancer treatments.
- Must meet specific blood count and organ function criteria.
- Must not have received prior HER2-targeted therapies.
- Must be at least 14 days from the last surgery.
- Must have adequate kidney and liver function.
- Must have a normal heart function and oxygen levels.
- Must not have any active infections or uncontrolled medical conditions.
- Must be willing to sign informed consent and comply with study requirements.
Exclusion Criteria
- Pregnant or breastfeeding women cannot participate.
- Participants must agree to use effective birth control during the study and for a period after the last dose.
- Cannot be receiving other investigational drugs or anti-cancer agents.
- Cannot have received a live vaccine within 30 days before starting the study.
- Cannot have a history of severe heart disease or congenital heart defects.
- Cannot have uncontrolled infections or non-healing surgical sites.
- Cannot have a history of severe allergic reactions to the study drug or similar medications.
- Cannot have significant lung diseases or conditions affecting the lungs.
- Cannot have active hepatitis B or C with detectable viral load.
- Cannot have autoimmune or inflammatory disorders affecting the lungs.
- Must not have an active primary immunodeficiency.
Locations
| FACILITY | ZIP | CITY | STATE |
|---|---|---|---|
| Children's Hospital of Alabama | 35233 | Birmingham | Alabama |
| Children's Hospital Los Angeles | 90027 | Los Angeles | California |
| Children's Hospital of Orange County | 92868 | Orange | California |
| Lucile Packard Children's Hospital Stanford University | 94304 | Palo Alto | California |
| UCSF Medical Center-Mission Bay | 94158 | San Francisco | California |
| Children's Hospital Colorado | 80045 | Aurora | Colorado |
| Children's National Medical Center | 20010 | Washington D.C. | District of Columbia |
| Johns Hopkins All Children's Hospital | 33701 | St. Petersburg | Florida |
| Children's Healthcare of Atlanta - Arthur M Blank Hospital | 30329 | Atlanta | Georgia |
| Lurie Children's Hospital-Chicago | 60611 | Chicago | Illinois |
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