A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT

Recruiting now · Phase 2

Conditions studied: Central Nervous System Nongerminomatous Germ Cell Tumor, Choriocarcinoma, Embryonal Carcinoma, Immature Teratoma, Malignant Teratoma, Mixed Germ Cell Tumor, Pineal Region Germ Cell Tumor, Pineal Region Immature Teratoma, Pineal Region Yolk Sac Tumor, Suprasellar Germ Cell Tumor

In brief

This phase II trial studies the best approach to combine chemotherapy and radiation therapy (RT) based on the patient's response to induction chemotherapy in patients with non-germinomatous germ cell tumors (NGGCT) that have not spread to other parts of the brain or body (localized). This study has 2 goals: 1) optimizing radiation for patients who respond well to induction chemotherapy to diminish spinal cord relapses, 2) utilizing higher dose chemotherapy followed by conventional RT in patients who did not respond to induction chemotherapy. Chemotherapy drugs, such as carboplatin, etoposide, ifosfamide, and thiotepa, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays or high-energy protons to kill tumor cells and shrink tumors. Studies have shown that patients with newly-diagnosed localized NGGCT, whose disease responds well to chemotherapy before receiving radiation therapy, are more likely to be free of the disease for a longer time than are patients for whom the chemotherapy does not efficiently eliminate or reduce the size of the tumor. The purpose of this study is to see how well the tumors respond to induction chemotherapy to decide what treatment to give next. Some patients will be given RT to the spine and a portion of the brain. Others will be given high dose chemotherapy and a stem cell transplant before RT to the whole brain and spine. Giving treatment based on the response to induction chemotherapy may lower the side effects of radiation in some patients and adjust the therapy to a more efficient one for other patients with localized NGGCT.

Key facts

Study ID: NCT04684368
Run by: Children's Oncology Group
People needed: 160
Starts: 2021-07-13
Expected to finish: 2029-12-21
Last updated by the study team: 2026-05-05

Who can join

Age: 3 and older, up to 29. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Patients must be >= 3 years and < 30 years at the time of study enrollment
Patients must be newly diagnosed with localized primary CNS NGGCT of the suprasellar and/or pineal region by pathology and/or serum or cerebrospinal fluid (CSF) elevation of AFP above institutional normal or > 10 ng/mL or human chorionic gonadotropin (hCG) beta > 100 mIU/mL as confirmed by Rapid Central Marker Screening Review on APEC14B1-CNS. Suprasellar, pineal and bifocal tumors are included. (CSF tumor markers and cytology must be within 31 days prior to enrollment and start of protocol therapy [repeat if necessary]. Serum tumor markers, AFP and hCGbeta must be within 7 days prior to enrollment and start of protocol therapy [repeat if necessary]). Basal ganglia or other primary sites are excluded
Patients with any of the following pathological elements are eligible: endodermal sinus (yolk sac), embryonal carcinoma, choriocarcinoma, malignant/immature teratoma and mixed germ cell tumor (GCT) (i.e., may include some pure germinoma) if malignant elements listed above are present. Patients with only mature teratoma are excluded. Patients with pure germinoma admixed with mature teratoma are excluded (would be eligible for pure germinoma protocols)
Patients must have a cranial MRI with and without gadolinium at diagnosis/prior to enrollment. If surgical resection is performed, patients must have pre-operative and post operative brain MRI with and without gadolinium. The post operative brain MRI should be obtained within 72 hours of surgery. If patient has a biopsy only, post-operative brain MRI is recommended but not required (within 31 days prior to study enrollment and start of protocol therapy )
Patients must have a spine MRI with gadolinium obtained at diagnosis/prior to enrollment. Spine MRI with and without gadolinium is recommended (within 31 days prior to study enrollment and start of protocol therapy)
Lumbar CSF must be obtained prior to study enrollment unless medically contraindicated. If a patient undergoes surgery and lumbar CSF cytology cannot be obtained at the time of surgery, then it should be performed at least 10 days following surgery and prior to study enrollment. False positive cytology can occur within 10 days of surgery
Patients must have RAPID CENTRAL TUMOR MARKER REVIEW CSF tumor markers obtained prior to enrollment unless medically contraindicated. Ventricular CSF obtained at the time of CSF diversion procedure (if performed) is acceptable for tumor markers but lumbar CSF is preferred. In case CSF diversion and biopsy/surgery are combined, CSF tumor markers should be collected first
Peripheral absolute neutrophil count (ANC) >= 1000/uL (within 7 days prior to enrollment)
Platelet count >= 100,000/uL (transfusion independent) (within 7 days prior to enrollment)
Hemoglobin >= 8.0 g/dL (may receive red blood cell [RBC] transfusions) (within 7 days prior to enrollment)
Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/gender as follows (within 7 days prior to enrollment):
Age: Maximum serum creatinine (mg/dL)
3 to < 6 years: 0.8 (male), 0.8 (female)
6 to < 10 years: 1 (male), 1 (female)
10 to < 13 years: 1.2 (male), 1.2 (female)
13 to < 16 years: 1.5 (male), 1.4 (female)
>= 16 years: male (1.7), 1.4 (female)
Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment)
Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (within 7 days prior to enrollment)
Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L
Central nervous system function defined as:
Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
Patients must not be in status epilepticus, coma or assisted ventilation prior to study enrollment
Protocol therapy must begin within 31 calendar days of definitive surgery or clinical diagnosis, whichever is later. If a biopsy only was performed, the biopsy date will be considered the date of definitive surgery. For patients who have a biopsy or incomplete resection at diagnosis followed by additional surgery, the date of the last resection will be considered the date of definitive surgery.
All patients and/or their parents or legal guardians must sign a written informed consent

You may not qualify if…

Patients with tumors located outside the ventricles (i.e., basal ganglia, thalamus)
Patients with only mature teratoma and non-elevated markers upon tumor sampling at diagnosis
Patients who have received any prior tumor-directed therapy for their diagnosis of NGGCT other than surgical intervention and corticosteroids
Patients with metastatic disease (i.e., MRI evaluation, lumbar CSF cytology or intraoperative evidence of dissemination)
Female patients who are pregnant, since fetal toxicities and teratogenic effects have been noted for several of the study drugs
Note: Serum and urine pregnancy tests may be falsely positive due to HCGbeta-secreting germ cell tumors. Ensure the patient is not pregnant by institutional standards
Lactating females who plan to breastfeed their infants
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation

Where it is running

Providence Alaska Medical Center — Anchorage, Alaska, United States (enrolling)
Nemours Children's Clinic-Jacksonville — Jacksonville, Florida, United States (enrolling)
Phoenix Childrens Hospital — Phoenix, Arizona, United States (enrolling)
Banner University Medical Center - Tucson — Tucson, Arizona, United States (enrolling)
UF Health Cancer Institute - Gainesville — Gainesville, Florida, United States (enrolling)
Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
Kaiser Permanente Downey Medical Center — Downey, California, United States (enrolling)
Loma Linda University Medical Center — Loma Linda, California, United States (enrolling)
Miller Children's and Women's Hospital Long Beach — Long Beach, California, United States (enrolling)
Children's Hospital Los Angeles — Los Angeles, California, United States (enrolling)
Banner Children's at Desert — Mesa, Arizona, United States (enrolling)
Cedars-Sinai Medical Center — Los Angeles, California, United States (enrolling)
Valley Children's Hospital — Madera, California, United States (enrolling)
Kaiser Permanente-Oakland — Oakland, California, United States (enrolling)
Children's Hospital of Orange County — Orange, California, United States (enrolling)
Lucile Packard Children's Hospital Stanford University — Palo Alto, California, United States (enrolling)
Rady Children's Hospital - San Diego — San Diego, California, United States (enrolling)
UCSF Medical Center-Mission Bay — San Francisco, California, United States (enrolling)
Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
Connecticut Children's Medical Center — Hartford, Connecticut, United States (enrolling)
Yale University — New Haven, Connecticut, United States (enrolling)
Alfred I duPont Hospital for Children — Wilmington, Delaware, United States (enrolling)
Children's National Medical Center — Washington D.C., District of Columbia, United States (enrolling)
Golisano Children's Hospital of Southwest Florida — Fort Myers, Florida, United States (enrolling)
University of Miami Miller School of Medicine-Sylvester Cancer Center — Miami, Florida, United States (enrolling)

Full record on ClinicalTrials.gov

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