SGT-53 in Children With Recurrent or Progressive CNS Malignancies
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03554707|
Recruitment Status : Not yet recruiting
First Posted : June 13, 2018
Last Update Posted : August 5, 2019
|Condition or disease||Intervention/treatment||Phase|
|Childhood CNS Tumor||Genetic: SGT-53 Radiation: Radiation Drug: Irinotecan Drug: Temozolomide Drug: Bevacizumab||Early Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study of SGT-53 in Conjunction With Irradiation and Chemotherapy in Children With Recurrent or Progressive CNS Malignancies|
|Estimated Study Start Date :||December 2019|
|Estimated Primary Completion Date :||December 2020|
|Estimated Study Completion Date :||December 2021|
Experimental: SGT-53 with radiation or drugs
Radiation phase: SGT-53 will be given at 2.1 mg DNA/m2 twice weekly for the first week of radiation therapy, and then increase to 2.8 mg DNA/m2 twice weekly. Radiation therapy will be administered as per clinical care, with a target of fifteen (15) fractions, but patients with other clinically-determined radiation plans will be allowed.
Chemotherapy phase: SGT-53 will be administered at the highest tolerated dose given during radiation phase. Irinotecan will be given at a dose of 50mg/m2/dose IV daily for five days in a 4-week cycle. Temozolomide will be given at a dose of 100mg/m2 PO daily for five days in a 4-week cycle and bevacizumab will be given at a dose of 10mg/kg IV every two weeks in a 4-week cycle.
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
100mg/m2 PO daily for five days in a 4-week cycle
Other Name: Temodar
10mg/kg IV every two weeks in a 4-week cycle
Other Name: Avastin
- Incidence of Adverse Events [ Time Frame: up to 13 months ]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.
- Response Rate [ Time Frame: 36 months ]The response rate will be calculated from the percentage of patients whose cancer shrinks or disappears after treatment.
- Duration of Response [ Time Frame: 36 months ]The duration of response will be the time calculated from documentation of tumor response as indicated by response criteria, to disease progression.
- Overall Survival [ Time Frame: 36 months ]Overall survival will be calculated from date of original diagnoses to death and also from the date of study registration to death.
- Progressive-Free Survival (PFS) [ Time Frame: 36 months ]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.
- Characterization of Phenotype of Patients [ Time Frame: 4-5 days ]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.
- Feasibility of Droplet PCR Assays to Monitor for Tumor Burden [ Time Frame: 12 months ]Droplet PCR will be used to assess for the presence of circulating tumor DNA
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03554707
|Contact: Sabrina Malik||202-476-5115||SaMalik@childrensnational.org|
|United States, District of Columbia|
|Children's National Medical Center||Not yet recruiting|
|Washington, District of Columbia, United States, 20010|
|Contact: Sabrina Malik 202-476-5115 SaMalik@childrensnational.org|
|Principal Investigator: Eugene Hwang, MD|
|Principal Investigator:||Eugene Hwang, MD||Children's Research Institute|