Pediatric patients 6-21 years of age with supratentorial recurrent, refractory, or progressive pediatric ependymoma and high-grade glioma (HGG) will be included in this study of treatment with Rhenium-186 Nanoliposome (186RNL). Phase 1 of the study will look to determine the maximum tolerated dose (MTD) of 186RNL in this patient population. Phase 2 of the study will use the recommended dose determined in Phase 1 to continue to look at overall response rate and progression-free survival following 186RNL treatment.
6 years to 21 years* of age.
Phase 1a/b only: A single lesion (less than or equal to) ≤3.5 cm (longest axis) and volume of (less than or equal to) ≤22.4 mL as the largest tumor (subsequent to individual Cohort lesion size requirements).
Phase 2a only: A single lesion or any number of multiple lesions separated by (less than or equal to) ≤3 cm; each lesion (less than or equal to) ≤3.5 cm (longest axis) and volume of (less than or equal to) ≤22.4 mL as the largest tumor.
Karnofsky Performance Status ≥ 60. For subjects <16 years of age, Lansky score ≥ 60.
Spinal disease.
Infratentorial location of tumor.
Involvement of the leptomeninges.
Hypertension (two or more blood pressure readings performed at screening of systolic blood pressure (SBP) or diastolic blood pressure (DBP) above 95th percentile for age) despite optimal treatment.
Sequential
Rhenium-186 Nanoliposome
DrugRhenium (186Re) Obisbemeda (Rhenium-186 NanoLiposome, 186RNL), BMEDA-chelated-186rhenium encapsulated within liposomes, allows the 186Re to be directly delivered to the site of the tumor through CED and maintain localization at the site of infusion.
Maximum Tolerated Dose (MTD)
Determine the maximum tolerated dose (MTD) of 186RNL administered by convection-enhanced delivery (CED) in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG.
Overall Response Rate (ORR) by RANO in Ependymoma
Determine the best overall response rate (ORR) by Radiographic Assessment in Neuro-Oncology (RANO) criteria following 186RNL administration in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma.
Progression-Free Suvival at 12 months (PFS12) in HGG
Determine progression-free survival at 12 months (PFS12) in subjects with supratentorial recurrent, refractory, or progressive pediatric HGG.
Safety of 186RNL Dose
Assess the safety of single dose 186RNL administered by CED in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG, as determined by the most recent version of the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE).
Dose Distribution of 186RNL
Assess the dose distribution of 186RNL administered by CED in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG.
Neuropsychologic Outcome
Assess the neuropsychologic outcomes of subjects exposed to 186RNL at 1 year in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG using the following assessments. Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V; ages 6-16:11) OR Wechsler Adult Intelligence Scale, Fourth Edition (WAIS-5; age \>17): Vocabulary, Block Design, Symbol Search, Coding, Digit Span These provide an IQ score on the standard scale with a higher score indicating a better outcome
Progression-Free Survival at 24 Months (PFS24) in Ependymoma
Determine the progression-free survival at 24 months (PFS24) in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma.
Overall Response Rate (ORR) by RAPNO
Assess overall response rate (ORR) by Response Assessment in Pediatric Neuro-Oncology (RAPNO).
Tissue Sample from Standard of Care Surgical Resection of Tumor
In Phase 2a only: tissue sample collected from SOC surgical resection of tumor for microscopic activity 4 weeks after receiving treatment.
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Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Collaborator
