Actively Recruiting
A Phase 0/1 Study of cDNA for TP53, Checkpoint Inhibition and Radiation in Children With Recurrent, Progressive or Refractory CNS Malignancies.
Led by Children's National Research Institute · Updated on 2026-04-29
18
Participants Needed
1
Research Sites
123 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This clinical trial is studying a drug called SGT-53 along with radiation and another drug called Nivolumab. It's for children with brain tumors that have come back, gotten worse, or didn't get better with earlier treatments. The main questions it aims to answer are: What is the right dose of SGT-53 that children can safely receive when it is used with radiation and Nivolumab? This dose will be used in the second phase of the trial. What side effects are there of SGT-53 when it is used with radiation and Nivolumab? How does SGT-53 move through the body when given with radiation and Nivolumab? How much of the SGT-53 drug is found in the tumor tissue? This will be tested in a small group of patients? Participants will: For the first treatment cycle: Get SGT-53 twice per week Get Nivolumab every 2 weeks Receive radiation therapy during week 2 For Cycles 2-6: Get SGT-53 once per week during even cycles and twice per week during odd cycles Get Nivolumab every 2 weeks For Cycles 7+ Get both SGT-53 and Nivolumab every 2 weeks In the phase 0 part of the study, 4 participants will have genetic testing performed on their tumor tissue after receiving SGT-53. These samples will be compared to another sample taken from the skin.
CONDITIONS
Official Title
A Phase 0/1 Study of cDNA for TP53, Checkpoint Inhibition and Radiation in Children With Recurrent, Progressive or Refractory CNS Malignancies.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must have a recurrent, progressive, or refractory CNS malignancy with no known curative options.
- Eligible tumor types include medulloblastoma, ATRT, high-grade gliomas, pineoblastomas, embryonal tumors NOS, CNS sarcomas, ependymomas, and other high-grade malignancies that failed first-line therapies.
- Patients should have received radiation therapy as part of standard initial treatment.
- Patients must have received at least one line of standard care treatment.
- Evidence of clinical and/or radiological progressive disease as defined by RAPNO criteria.
- Patients with metastatic disease are eligible.
- Must have available archival or fresh tumor tissue for correlative studies.
- Initial 3 patients must be >7 to <22 years old; thereafter, patients must be ≥3 to <22 years old.
- Must have recovered from all surgical interventions before radiation and maintenance phases.
- Body surface area must be 0.6 m2 or more.
- Must have recovered from acute effects of prior therapy; last chemotherapy dose at least 2 weeks before SGT-53.
- Last biologic therapy dose at least 7 days before SGT-53.
- Eligible for hypofractionated irradiation clinically; timing restrictions apply based on prior radiation.
- Stable or decreasing corticosteroid dose for at least 1 week before enrollment; maximum dexamethasone dose 0.5 mg/m2/day.
- Last short-acting growth factor dose at least 1 week before treatment; long-acting growth factors at least 2 weeks prior.
- Neurologic baseline status for minimum 1 week before enrollment.
- Performance status (Karnofsky or Lansky) >50 assessed within 2 weeks.
- Normal organ and marrow function as specified (including blood counts, liver function, cardiac function, kidney function).
- Patients of childbearing potential must use acceptable birth control during and 5 months after treatment.
- Female patients must not be pregnant or nursing and have a negative pregnancy test at enrollment.
- Ability and willingness to provide informed consent.
You will not qualify if you...
- Low-grade glioma, craniopharyngioma, and other non-malignant CNS tumors.
- Diagnosis of diffuse midline gliomas (DMGs).
- Clinically significant unrelated systemic illness likely to interfere with study therapy.
- Pre-existing cardiac dysfunction with ejection fraction less than 35%.
- Pre-existing pulmonary fibrosis, interstitial lung disease, or significant pulmonary disease.
- Receiving any other investigational drug therapy.
- Inability to adhere to protocol requirements.
- History of autoimmune diseases requiring treatment within last 2 years.
- Receipt of live vaccine within 30 days before study treatment.
- Known HIV/AIDS or acute/chronic Hepatitis B or C infection.
- Prohibited from receiving radiation therapy due to maximum lifetime dose reached.
AI-Screening
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Trial Site Locations
Total: 1 location
1
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
Research Team
J
Julia Batarseh
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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