Phase I Trial of the Safety and Efficacy of Deflexifol for Refractory or Recurrent Paediatric Central Nervous System (CNS) Tumours

ACTRN 12623000104651

Brief Summary

This is a multicentre Phase I clinical trial to establish if Deflexifol is safe and effective in children, adolescents and young adults with recurrent or refractory brain tumours.

Intervention/Treatment

• Drug: Deflexifol.

Inclusion Criteria

1. Written informed consenta process in which a patient receives detailed information about a procedure or treatment, including its potential risks, benefits, and alternatives. The patient then has the opportunity to understand the information and voluntarily agree to the procedure or treatment..
2. Patients must be greater than 12 months and less than or equal to 21 years of age at the time of study enrolment.
3. Patient must have histologic confirmation of CNS tumoura tissue mass that forms from groups of unhealthy cells that is refractory or recurrent based on radiologic or tissuea group of cells that work together to perform a function diagnosisthe process of identifying a disease based on signs and symptoms, patient history and medical test results; and for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of lifethe overall well-being of a person diagnosed with cancer, encompassing both physical and emotional aspects of health.
4. Patient must have measurable disease as per RAPNO criteria and/or evaluable disease. For patients who have received prior radiation therapya treatment that uses controlled doses of radiation to damage or kill cancer cells, the irradiated lesion needs to have progressed since cessation of radiotherapy.
5. Karnofsky performance status greater than or equal to 50 for patients above 16 years old;; or Lansky performance status greater than or equal to 50 for patients less than or equal to 16 years old. Patients who are not able to walk due to paralysis, but who are sitting up in a wheelchair, are considered ambulatoryable to walk or move around unassisted for the purpose of performance score assessment.
6. Life expectancy of greater than 6 weeks.
7. Patients receiving corticosteroidsa type of anti-inflammatory medication that is used to treat inflammation will need to be on a stable, or reducing, dosethe amount of medication taken of steroids for at least one week before enrolment.
8. Fully recovered from acutenew, recent, comes with an urgent or significant sense, is sudden, sharp toxic effects of all prior anti-cancer therapy:
   • Must not have received myelosuppressive chemotherapya cancer treatment that uses drugs to kill or slow the growth of cancer cells, while minimising damage to healthy cells within 21 days of study entry (42 days if prior nitrosourea or mitomycin-C).
   • At least 7 days after the last dose of anti-cancer agents not known to be myelosuppressive. For agents that have known adverse events occurring beyond 7 days after last administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.
   • At least 42 days after the completion of any type of immunotherapya treatment that uses a person's immune system to fight cancer, e.g. tumour vaccines.
   • At least 21 days after the last dose of a monoclonal antibodya protein made by the immune system to fight against harmful substances (antigens), such as bacteria or viruses, and toxicities related to prior antibody therapy have recovered to less than or equal to gradea description of how abnormal cancer cells and tissue look under a microscope when compared to healthy cells 1.
   • At least 6 weeks after the completion of radiation therapy (including CSI). If palliative radiotherapy has been administered, then 2 weeks must have lapsed before the patient can commence on this trial, and this lesion cannot be used as the target lesion for assessment of treatment response.
   • 14 days or more from last long-acting haematopoietic growth factors (e.g. pegfilgrastim) or 7 days for short acting growth factor.
9. Adequate function in bone marrowsoft, spongy tissue found in bones that makes blood cells, renal and liver. Bloodthe red bodily fluid that transports oxygen and other nutrients around the body pressure within normal range for age.
10. Patient must be or have been previously enrolled in the ZERO Childhood Cancera disease where abnormal cells split without control and spread to other nearby body tissue and/or organs (ZERO) precision medicine trial or program.
11. Female patient of childbearing potential must have a negative serum or urine pregnancy test at screeningtesting for cancer or conditions that can lead to cancer before symptoms appear, also known as cancer screening and prior to the beginning of each cycle. They must agree to use an effective method of contraception during the study period and for six months after treatment discontinuation.
12. Fertile male patients must use an effective method of contraception during the study period and for six months after treatment discontinuation.