Metronomic therapy for high-risk pediatric solid tumors

Metronomic Therapy for Pediatric Patients With Solid Tumors at High Risk of Recurrence: A Multi-Institutional Study

This study is testing a new way of giving lower doses of cancer drugs continuously to help kids in remission from high-risk solid tumors stay cancer-free longer.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 12 Months to 31 Years
• Sex: All
• Sponsor: Miller Children's & Women's Hospital Long Beach (Academic / other)
• Drugs / interventions: bevacizumab, chemotherapy, cyclophosphamide
• Locations: 2 sites (Long Beach, California and 1 other locations)
• Trial ID: NCT02446431 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to improve outcomes for pediatric patients with solid tumors at high risk of recurrence by implementing metronomic therapy, which involves lower dose continuous drug exposure. The study will treat patients who are in remission after completing front-line therapy, using agents that target disrupted signaling pathways and epigenetic abnormalities. The treatment will be administered in an outpatient setting, and the trial will also analyze the financial impact of this therapy on families and the healthcare system. The goal is to initiate treatment when patients have minimal disease burden to enhance efficacy.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients aged 12 months to 31 years with specific solid tumors who are in remission after front-line therapy.

Not a fit: Patients with solid tumors that do not meet the eligibility criteria or those who have not completed front-line therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could lead to improved outcomes and reduced recurrence rates for high-risk pediatric patients with solid tumors.

How similar studies have performed: While metronomic therapy has shown success in pediatric leukemias, its application in solid tumors has been limited and largely untested, making this approach novel.

Eligibility criteria

Inclusion Criteria:

1. The following solid tumors will be studied: rhabdomyosarcoma, osteosarcoma, Ewing sarcoma, other soft tissue sarcomas
2. Other solid tumors fulfilling the remainder of eligibility criteria and available historical data to determine time to tumor progression
3. Expected time to progression of \< 2 years, based on historical data
4. All patients will have completed front-line therapy
5. All patients will be in remission from their primary diagnosis
6. All patients will start metronomic therapy within 6 weeks of completion of front-line treatment
7. All patient will have recovered from previous toxicities
8. All patients or their parents/legal guardian will have signed an informed consent document
9. All institutional eligibility criteria will be meet
10. Age: Patients must be ≥ 12 months and \<31 years of age at the time of study entry
11. Patients must have had histologic verification of malignancy at original diagnosis
12. Patients must have a Lansky or Karnofsky performance status score of ≥ 50, corresponding to ECOG categories 0, 1 or 2.
13. Adequate renal function defined as: Normal serum creatinine
14. Normal liver tests (ALT/AST/total bilirubin/triglycerides/cholesterol)
15. Recovered from all surgical procedures for at least 7 days (minor procedures) or 28 days (major procedures)
16. Adequate cardiac function defined as: Shortening fraction of ≥ 27% by echocardiogram, or ejection fraction of ≥ 50% by radionuclide angiogram
17. Platelet count 100,000K/uL (transfusion independent), hemoglobin 8.0 g/dL
18. Adequate bone marrow function: Peripheral absolute neutrophil count (ANC) 1,000K/uL
19. Signed Informed Consent document and/or Assent document

Exclusion Criteria:

1. Female patients who are pregnant
2. Lactating females are not eligible unless they have agreed to discontinue breastfeeding
3. Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained
4. Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of study participation
5. Any primary central nervous system tumor
6. Any patient who has experienced relapsed or refractory disease or a second malignancy.
7. Any patient not in remission

Where this trial is running

Long Beach, California and 1 other locations

• Miller Children's and Women's Hospital Long Beach — Long Beach, California, United States (Recruiting)
• Children's Hospital Orange County — Orange, California, United States (Recruiting)

Study contacts

• Principal investigator: Ted Zwerdling, MD — Miller Children's and Women's Hospital Long Beach
• Study coordinator: Ted Zwerdling, MD

Conditions Solid Tumor; Metronomic Drug Therapy