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Protocol No. ADVL1622

Principal Investigator DeSantes, Kenneth

Phase II

Age Group Both

Scope National

Sponsor Type National

Title Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors

Objective The purpose of the research study: The purpose of this research study is to find out if we can improve the treatment for patients with sarcomas, Wilms tumor or other rare tumors, that have either not responded to therapy, recurred or have no known curative therapy available. This study is looking at the effectiveness of XL184 in treating these types of cancers.

Treatment The treatment involves the experimental drug XL184, also called cabozantinib. XL184 is a type of drug called a small molecule inhibitor and works by reducing tumor growth and tumor blood vessel growth. We are using XL184 because it seems to work against cancer in test tubes and animals and in some adults. XL184 has been Food and Drug Administration (FDA) approved for the treatment of adults with progressive, metastatic medullary thyroid cancer and adults with advanced renal cell carcinoma who have received prior anti-angiogenic therapy. However, the use of XL184 in children and young adults with the types of cancer included in this trial has not yet been approved by the FDA and is considered experimental.

Description In this study you will get XL184 daily during each cycle. A cycle lasts 28 days (4 weeks). There is not a set length of therapy on this study. You may continue to receive cycles of daily XL184 unless you develop serious side effects or your tumor worsens. You will get 2 cycles of treatment and then there will be an evaluation. An evaluation means that you will have tests and imaging studies (scans) done to see if the tumor has changed. If the tumor does not get larger and you do not have bad side effects, your doctor may suggest you continue with treatment. You will have further evaluations at the end of Cycles 4, 6, and every 3rd cycle thereafter. You will stop getting treatment on this study if the tumor gets larger or comes back, or if side effects from the treatment are too severe. If that happens, your doctor will discuss other treatment options with you.

Key Eligibility
  • Age: Patients must be ≥ 2 and ≤ 30 years of age at the time of study entry for all strata except upper age limit of ≤ 18 years of age for medullary thyroid carcinoma (MTC),renal cell carcinoma (RCC) and hepatocellular carcinoma (HCC)
  • Patients must have a body surface area ≥ 0.35 m2
  • Diagnosis: Patients must have recurrent or refractory disease, or newly diagnosed disease with no known curative therapy or therapy proven to prolong survival with an acceptable quality of life
  • Patients must have had histologic verification of one of the malignancies listed at original diagnosis or at relapse: ewing sarcoma, rhabdomyosarcoma (RMS), non-rhabdomyosarcoma soft tissue sarcomas (STS), wilms tumor, or rare tumors
  • Patients must have radiographically measurable disease
  • Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study
  • Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies
  • Patients who have an uncontrolled infection are not eligible
  • Patients with active bleeding are not eligible
  • Patients who have had significant traumatic injury within 28 days prior to enrollment are not eligible
  • Patients with any medical or surgical conditions that would interfere with gastrointestinal absorption of the study drug are not eligible

Applicable Disease Sites Anal; Bladder; Brain/Central Nervous System; Breast; Cervix; Colon and Rectum; Endocrine cancers; Esophagus; Gastrointestinal cancers, other; Genitourinary cancers, other; Head and Neck; Ill-Defined Sites; Kidney; Liver; Lung; Melanoma/Skin cancer; Ovary; Pancreas; Prostate; Sarcoma; Stomach; Thyroid; Uterus

Therapies Involved Immunotherapy

Drugs Involved XL184 (Cabozantinib)

Status Open

Participating Institutions Gundersen Health System; UW Hospital and Clinics