Neoplasms

All Conditions

Nervous System Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Digestive System Diseases

Diseases and Abnormalities at or Before Birth

Musculoskeletal Diseases

Skin and Connective Tissue Diseases

Symptoms and General Pathology

Rare Diseases

Carcinoma

Glioma

Meningioma

Osteosarcoma

Neuroblastoma

Sarcoma

Carcinoma, Renal Cell

Liver Neoplasms

Wilms Tumor

Medulloblastoma

Sarcoma, Ewing

Neuroectodermal Tumors, Primitive, Peripheral

Ependymoma

Craniopharyngioma

Adamantinoma

Rhabdomyosarcoma

Rhabdoid Tumor

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Sarcoma, Clear Cell

Neoplasms, Neuroepithelial

Choroid Plexus Neoplasms

Ameloblastoma

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Kidney Neoplasms

Urologic Neoplasms

Urogenital Neoplasms

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Kidney Diseases

Urologic Diseases

Male Urogenital Diseases

Neoplasms, Germ Cell and Embryonal

Neoplasms, Nerve Tissue

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Myosarcoma

Neoplasms, Vascular Tissue

Meningeal Neoplasms

Central Nervous System Neoplasms

Nervous System Neoplasms

Syndrome

Neoplastic Syndromes, Hereditary

Genetic Diseases, Inborn

Bone Neoplasms

Bone Diseases

Cerebral Ventricle Neoplasms

Brain Neoplasms

Brain Diseases

Central Nervous System Diseases

Renal Cell Carcinoma

Soft Tissue Sarcoma

Clear Cell Renal Cell Carcinoma

Germ Cells Tumors

Wilms' Tumor

Ewing Sarcoma

Neuroepithelioma

Dysembryoplastic Neuroepithelial Tumor

A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Guided Therapy

Giselle Sholler, MD

The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup. This technology includes a genomic report based on DNA exomes and RNA sequencing that will be used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.

Molecular-Guided Therapy for Childhood Cancer

Molecular-guided Therapy for the Treatment of Patients With Relapsed and Refractory Childhood Cancers

The definition of feasibility is: Enrollment onto study, genomic profile, analysis and report generation completed, tumor board held with treatment decision, treatment review completed, start of treatment, and completion of 1 cycle of therapy.

To determine the safety of allowing a molecular tumor board to determine individualized treatment plans

To determine the activity of treatments chosen based on Overall response rate (ORR) using RESIST criteria. The assessment of response will include the initial measurable targets and will be performed after cycle 2, then after every other cycle. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT or MRI imaging and/or by MIBG or PET scans: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Progressive Disease, At least a 20% increase in the sum of the disease measurements for measurable lesions, Stable Disease, Neither sufficient decrease to qualify for PR or sufficient increase to qualify for PD from study entry. Overall Response (OR) = CR + PR.

Time to progression (PFS), defined as the period from the start of the treatment until the criteria for progression are met taking as reference the screening measurements. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as at least a 20% increase in the sum of the disease measurements for measurable lesions, taking as reference the smallest disease measurement recorded since the start of treatment (nadir), and minimum 5 mm increase over the nadir or the appearance of one or more new lesions or appearance of positive bone marrow.

Translational Genomics Research Institute

Dell, Inc.

Giselle Sholler

Study Chair

A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Guided Therapy: A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Age, Categorical

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Tumor Type

Subjects that started at least one dose of study therapy

Subjects on study with a diagnosis of Neuroblastoma

Subjects on study with a diagnosis of a CNS Tumor which included, but was not limited to, Ependymoma, Glioblastoma, Diffuse Midline Glioma, and Other CNS Tumors.

CNS Tumors

Subjects on study with a diagnosis of a rare solid tumor which included, but was not limited to, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, and Other Rare Tumors.

Rare Tumors

Days to Treatment Will be Used in Order to Determine Feasibility of Using Tumor Samples to Assess Genomic Sequencing Using Predictive Modeling to Make Real-time Treatment Decisions for Children With Relapsed/Refractory Cancers.

All subjects that received at least one dose of study therapy.

Number of Participants With an Unexpected Adverse Events as a Measure of Safety

Complete Response

No Evidence of Disease

Partial Response

Stable Disease

Progressive Disease

Having at least one disease evaluation following completion of at least one cycle of molecular-guided therapy

Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans.

Time on study was measured in days from the date of initial biopsy to date of last administration of study therapy. For subjects with \>1 biopsies and \>1 MGT regimens, the off-therapy date from their last treatment was used.

Progression Free Survival (PFS) Interval Will be Measured by Days and Compared to the PFS of Previous Chemotherapy Regimens Since Relapse for Each Patient.

Overall Study

Desmoplastic Small Round Cell Tumor

All Drugs and Chemicals

Hypoglycemic Agents

Antibodies

Antibodies, Monoclonal

Insulin

Insulin, Globin Zinc

Immunoglobulins

Mitogens

If a total of two or more responses (partial and complete) in EFTs/DSRCTs are documented in this or the ongoing phase 1 study (20050118), then the study will allow enrollment of up to 10 additional EFT/DSRCT subjects who have been exposed to prior anti-IGF-1R targeting therapy.

Subjects with relapsed Ewing's Family Tumors (EFTs) and Desmoplastic Small Round Cell Tumors (DSRCTs) who have not received prior anti-IGF-1R therapy will receive AMG 479 at 12mg/kg.

AMG 479 is a fully human monoclonal antibody (IgG1) against the insulin-like growth factor receptor-1 (IGF-1R). IGF-1R signaling has been shown to play a critical role in the survival of cancer cells. AMG 479 inhibits the binding of both IGF-1 and IGF-2 to IGF-1R, thus inhibiting ligand-dependent receptor activation. Inhibition of IGF-1R signaling with AMG 479 provides a potential mechanism for inhibiting tumor growth and survival.

AMG 479

Single-arm, open-label study of AMG 479 in up to 35 subjects with Ewing's Family Tumors (EFTs) and Desmoplastic Small Round Cell Tumors (DSRCTs) who have progressed or recurred after at least one prior chemotherapy regimen. An exploratory cohort of an additional up to 10 subjects with prior exposure to anti-IGF-1R therapy and who have progressed or recurred after at least one prior chemotherapy regimen will also be assessed.

QUILT-3.025: A Phase 2 Study of AMG 479 in Relapsed or Refractory Ewing's Family Tumor and Desmoplastic Small Round Cell Tumors

A Phase 2 Study of AMG 479 in Relapsed or Refractory Ewing's Family Tumor and Desmoplastic Small Round Cell Tumors

NantCell, Inc.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Molecular-Guided Therapy for Childhood Cancer	NCT02162732	Neuroblastoma Medulloblastoma Glioma Ependymoma Choroid Plexus ... Craniopharyngio... Dysembryoplasti... Meningioma Primitive Neuro... Germ Cell Tumor... Rhabdomyosarcom... Non-rhabdomyosa... Ewings Sarcoma Osteosarcoma Wilms Tumor Renal Cell Carc... Malignant Rhabd... Clear Cell Sarc... Liver Tumors	Guided Therapy	13 Months - 21 Years	Milton S. Hershey Medical Center
QUILT-3.025: A Phase 2 Study of AMG 479 in Relapsed or Refractory Ewing's Family Tumor and Desmoplastic Small Round Cell Tumors	NCT00563680	Askin's Tumors Desmoplastic Sm... Estraosseous Ew... Ewing's Family ... Ewing's Sarcoma Primitive Neuro... Sarcoma	AMG 479	16 Years -	NantCell, Inc.

Spots Global Cancer Trial Database for primitive neuroectodermal tumors (pnets)