Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

Launched by AUSTRALIAN & NEW ZEALAND CHILDREN'S HAEMATOLOGY/ONCOLOGY GROUP · Jan 5, 2024

Keywords

ClinConnect Summary

The clinical trial titled "Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer" is focused on finding new treatment options for children and young adults with specific types of cancer, such as solid tumors, brain tumors, and lymphoma, especially when standard treatments have not worked. This study is looking for patients up to 21 years old, or older patients with pediatric-type cancers, who have tumors that are difficult to treat. Participants will receive targeted therapies that are chosen based on the unique characteristics of their tumors, which is known as precision medicine.

To be eligible for this trial, patients should have a tumor that has progressed despite existing treatments or for which no effective treatment is available. They need to be in relatively good health, meaning they should be able to perform daily activities and have a life expectancy of at least six weeks. Participants will undergo regular health checks and tests, which may be done at their local hospital. It's important to note that patients must be able to comply with follow-up appointments and management of any side effects. If you or someone you know is facing these challenges with cancer, this trial could be an opportunity to explore new treatment options.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Patients must be diagnosed with a solid tumor, CNS tumor or lymphoma that has progressed despite standard therapy, or for which no effective standard therapy exists.
• • 2. Age \<21 years at inclusion; patients 21 years and older may be included after approval by the Study Chair if they have a pediatric type recurrent/refractory malignancy.
• • 3. Patients must be enrolled on a precision medicine study (i.e. PROFYLE, ZERO or equivalent as agreed with Study Chair.
• • 4. Patients enrolled in a Phase I cohort must have either evaluable or measurable disease.
• • 5. Patients enrolled in a Phase II cohort must have measurable disease. Evaluable and measurable disease are defined by standard imaging criteria for the patient's tumor type.
• • 6. Disease evaluations, laboratory tests, and other clinical assessments that are considered standard of care may be undertaken at the patient's local oncology treatment centre with results transferred to study site for evaluation.
• • 7. Performance status: Karnofsky performance status (for patients \> 16 years of age) or Lansky play score (for patients ≤ 16 years of age) ≥ 50%.
• • 8. Life expectancy ≥ 6 weeks.
• • 9. Patients must have fully recovered from the acute toxic effects of all prior anticancer therapy and must meet the following minimum duration from prior anticancer-directed therapy prior to enrolment.
• • 10. Adequate organ function.
• • 11. Able to comply with scheduled follow-up and with management of toxicity.
• • 12. Females of childbearing potential must have a negative serum or urine pregnancy test.
• • 13. Fertile males must agree to use adequate contraception during the study and following completion of treatment.
• • 14. Provide a signed and dated informed consent form.
• Exclusion Criteria:
• • 1. Patients with symptomatic CNS primary or metastatic tumors who are neurologically unstable or require increasing doses of corticosteroids or local CNS-directed therapy to control their CNS disease.
• • 2. Impairment of gastrointestinal (GI) function or GI disease that may significantly alter drug absorption of oral drugs.
• • 3. Clinically significant, uncontrolled heart disease.
• • 4. Known active viral hepatitis or human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
• • 5. Presence of any ≥Grade 2 treatment-related toxicity.
• • 6. Major surgery within 21 days of the first dose of investigational drug.
• • 7. Known hypersensitivity to any study drug or component of the formulation.
• • 8. Pregnant or nursing (lactating) females.
• • 9. Any other concomitant serious medical condition or organ dysfunction that in the opinion of the investigator would either compromise patient safety or interfere with the evaluation of the safety of the investigational drugs.