Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||1 Year - 21 Years|
- - Patients must have a recurrent, progressive, or refractory CNS malignancy for which there are not known curative options.
- - Tumor must be measureable, defined as a tumor that can be accurately measured in two perpendicular dimensions on MRI.
- - Patients with metastatic disease are eligible but must have at least one target lesions which is measurable.
- - Patients must have available archival (formalin-fixed paraffin embedded) or fresh tumor tissue for correlative studies.
- - Patients must be >1yrs and <21 years of age.
- - Must have recovered from all surgical interventions prior to the start of the Radiation and Chemotherapy Phases.
- - Patients must have recovered from the acute effects of prior therapy.
- - There is a maximum of 3 previous myelosuppressive therapy regimens.
- - Patients must have received their last dose of known myelosuppressive therapy at least three (3) weeks prior to receipt of SGT-53.
- - Patients must have received their last dose of biological agent >7 days prior to receipt of SGT-53.
- - Patients must be far enough from previous irradiation that in the opinion of a radiation oncologist using standard fractionation is deemed to be reasonable from a clinical standard of care perspective.
- - Patients who are receiving dexamethasone or other corticosteroids must be on a stable or decreasing dose for at least one (1) week prior to enrollment.
- - Patients must have received their last dose of any short acting growth factor at least one week prior to treatment, for long acting or pegylated growth factors, the last dose must be at least two (2) weeks prior to start of treatment.
- - Patients with neurologic deficits must have deficits that have been stable in grade for a minimum of one week prior to enrollment.
- - Performance status (Karnofsky PS for >16yrs, or Lansky PS for <16yrs) assessed within two weeks must be >50.
- - Patients must have normal organ and marrow function.
- - All patients of childbearing or child fathering potential must be willing to use an acceptable form of birth control while being treated on this study.
- - Female patients must not be pregnant or nursing.
- - Patient and/or guardian have the ability to understand and the willingness to sign a written informed consent document according to institutional guidelines.
- - Patients with any clinically significant unrelated systemic illness (serious infection or significant cardiac, pulmonary, hepatic, or other organ dysfunction) that is likely to interfere with ability to tolerate study therapy or study procedure results.
- - Patients with low-grade gliomas, craniopharyngioma, or extracranial tumors with CNS metastases.
- - Patients who are receiving any other investigational drug therapy.
- - Patients who require therapeutic anti-coagulation.
- - Patients who in the opinion of the investigator cannot adhere to protocol requirements.
- - Patients with history of clinically significant clot or hemorrhage are eligible but will not receive bevacizumab during chemotherapy regimen.
- - Unavailability of the chemotherapy due to insurance coverage or other logistical issues is an ineligibility criterion.
- - Patients may not be on immunosuppressive therapy, including corticosteroids (with the exception of physiologic replacement, defined as 0.75mg/m2/day) at time of enrollment.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
|Early Phase 1|
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|SynerGene Therapeutics, Inc.|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Eugene Hwang, MD|
|Principal Investigator Affiliation||Children's National Research Institute|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
|Overall Status||Not yet recruiting|
The disease, disorder, syndrome, illness, or injury that is being studied.
|Childhood CNS Tumor|
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.
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.
Genetic: - SGT-53
2.1 mg DNA/m2 or 2.8 mg DNA/m2 twice weekly
Radiation: - Radiation
Standard radiation plan
Drug: - Irinotecan
50mg/m2/dose IV daily for five days in a 4-week cycle
Drug: - Temozolomide
100mg/m2 PO daily for five days in a 4-week cycle
Drug: - Bevacizumab
10mg/kg IV every two weeks in a 4-week cycle
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.