An early phase 1 for pediatric patients with recurrent or progressive CNS malignancies
This clinical trial is a early phase 1, open label, single center, single arm study of the combination of intravenously administered SGT-53 and irradiation and/or chemotherapy in pediatric patients with recurrent or progressive CNS malignancies. The objective of the study is to establish the safety and feasibility of administration of SGT-53 in conjunction with conventional radiotherapy and/or chemotherapy in children with recurrent or refractory CNS malignancies.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
2.1 mg DNA/m2 or 2.8 mg DNA/m2 twice weekly
Standard radiation plan
50mg/m2/dose IV daily for five days in a 4-week cycle
Children's National Medical Center
Washington D.C., District of Columbia, United States
Incidence of Adverse Events
An adverse event (AE) was any untoward medical occurrence that began or worsened in grade after the start of study drug through 30 days after the last dose. The safety will be assessed by the number and severity of any AE or serious adverse events (SAE) experienced by the patients, and by their relationship to the study drug SGT-53 (e.g. definitely, probably, possibly, unlikely or unrelated). Severity will be graded according to NCI CTCAE version 4.0.
Time frame: up to 13 months
Response Rate
The response rate will be calculated from the percentage of patients whose cancer shrinks or disappears after treatment.
Time frame: 36 months
Duration of Response
The duration of response will be the time calculated from documentation of tumor response as indicated by response criteria, to disease progression.
Time frame: 36 months
Overall Survival
Overall survival will be calculated from date of original diagnoses to death and also from the date of study registration to death.
Time frame: 36 months
Progressive-Free Survival (PFS)
PFS will be calculated at all times during follow-up, with particular interest in the 6-month time point. Progression free survival will be calculated from date of first treatment to the date of first observation of progressive disease, non-reversible neurological progression or increasing steroid requirements (applies to stable disease only), death due to any cause, or early discontinuation of treatment.
Time frame: 36 months
Characterization of Phenotype of Patients
Tissue will be obtained from each enrolling patient for subsequent testing for TP53 pathway functionality, including assessment of mdm2, p21, and other mutation or expression alterations. This analysis will also include measures of commonly assessed polymorphisms, including MGMT methylation assessment, ATRX mutation among others.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
100mg/m2 PO daily for five days in a 4-week cycle
10mg/kg IV every two weeks in a 4-week cycle
Time frame: 4-5 days
Feasibility of Droplet PCR Assays to Monitor for Tumor Burden
Droplet PCR will be used to assess for the presence of circulating tumor DNA
Time frame: 12 months