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Conditions: Brain and Central Nervous System Tumors, Neuroblastoma
Interventions: enzastaurin hydrochloride; Drug
Lead sponsor: Pediatric Brain Tumor Consortium; Network
Eligibility: Up to 21 Years
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2010
U.S. locations: 8
States / cities: San Francisco, California • Washington D.C., District of Columbia • Chicago, Illinois + 5 more

Conditions: Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Choriocarcinoma, Childhood Central Nervous System Embryonal Tumor, Childhood Central Nervous System Germinoma, Childhood Central Nervous System Mixed Germ Cell Tumor, Childhood Central Nervous System Teratoma, Childhood Central Nervous System Yolk Sac Tumor, Childhood Choroid Plexus Tumor, Childhood Craniopharyngioma, Childhood Ependymoblastoma, Childhood Grade I Meningioma, Childhood Grade II Meningioma, Childhood Grade III Meningioma, Childhood High-grade Cerebellar Astrocytoma, Childhood High-grade Cerebral Astrocytoma, Childhood Infratentorial Ependymoma, Childhood Low-grade Cerebellar Astrocytoma, Childhood Low-grade Cerebral Astrocytoma, Childhood Medulloepithelioma, Childhood Mixed Glioma, Childhood Oligodendroglioma, Childhood Supratentorial Ependymoma, Extra-adrenal Paraganglioma, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Central Nervous System Embryonal Tumor, Recurrent Childhood Cerebellar Astrocytoma, Recurrent Childhood Cerebral Astrocytoma, Recurrent Childhood Ependymoma, Recurrent Childhood Medulloblastoma, Recurrent Childhood Pineoblastoma, Recurrent Childhood Spinal Cord Neoplasm, Recurrent Childhood Subependymal Giant Cell Astrocytoma, Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor, Recurrent Childhood Visual Pathway and Hypothalamic Glioma
Interventions: vorinostat, temozolomide, diagnostic laboratory biomarker analysis, pharmacological study; Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 1 Year to 21 Years
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2010
U.S. locations: 7
States / cities: Chicago, Illinois • Ann Arbor, Michigan • Minneapolis, Minnesota + 4 more

Conditions: Brain Tumor, Central Nervous System Tumors, Cachexia, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions: cyproheptadine hydrochloride, megestrol acetate; Drug
Lead sponsor: University of South Florida; Other
Eligibility: 2 Years to 20 Years
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2007
U.S. locations: 40
States / cities: Oakland, California • Washington D.C., District of Columbia • Gainesville, Florida + 33 more

Conditions: Advanced Solid Tumors
Interventions: Elimusertib (BAY1895344), Pembrolizumab (Keytruda®); Drug
Lead sponsor: Bayer; Industry
Eligibility: 18 Years and older
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 8
States / cities: Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more

Conditions: Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor, Childhood Craniopharyngioma, Childhood Ependymoblastoma, Childhood Grade I Meningioma, Childhood Grade II Meningioma, Childhood Grade III Meningioma, Childhood High-grade Cerebellar Astrocytoma, Childhood High-grade Cerebral Astrocytoma, Childhood Infratentorial Ependymoma, Childhood Low-grade Cerebellar Astrocytoma, Childhood Low-grade Cerebral Astrocytoma, Childhood Medulloepithelioma, Childhood Mixed Glioma, Childhood Oligodendroglioma, Childhood Supratentorial Ependymoma, Recurrent Childhood Brain Tumor, Recurrent Childhood Cerebellar Astrocytoma, Recurrent Childhood Cerebral Astrocytoma, Recurrent Childhood Ependymoma, Recurrent Childhood Medulloblastoma, Recurrent Childhood Pineoblastoma, Recurrent Childhood Subependymal Giant Cell Astrocytoma, Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor, Recurrent Childhood Visual Pathway and Hypothalamic Glioma
Interventions: lenalidomide, perfusion-weighted magnetic resonance imaging, diffusion-weighted magnetic resonance imaging, laboratory biomarker analysis; Drug · Procedure · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: Up to 21 Years
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2004
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Pancreatic Ductal Adenocarcinoma, Pancreatic Cyst, Chronic Pancreatitis, Fatty Pancreas, Genetic Pancreatic Cancer, Genetic Pancreatitis, BRCA Mutation, Lynch Syndrome, FAP, Familial Atypical Multiple Mole-Melanoma, PALB2 Gene Mutation, Peutz-Jeghers Syndrome, Ataxia Telangiectasia
Interventions: Data collection; Other
Lead sponsor: H. Lee Moffitt Cancer Center and Research Institute; Other
Eligibility: 18 Years and older
Enrollment: 317 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2026
U.S. locations: 1
States / cities: Tampa, Florida

Conditions: Brain and Central Nervous System Tumors
Interventions: laromustine; Drug
Lead sponsor: Pediatric Brain Tumor Consortium; Network
Eligibility: Up to 21 Years
Enrollment: 42 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2008
U.S. locations: 10
States / cities: San Francisco, California • Washington D.C., District of Columbia • Chicago, Illinois + 7 more

Conditions: Cowden's Disease, Cowden's Syndrome, Lhermitte-Duclos Disease, Cerebellum Dysplastic Gangliocytoma, Bannayan Zonana Syndrome, Myhre Riley Smith Syndrome, Riley Smith Syndrome, PTEN Hamartoma Tumor Syndrome, Bannayan Riley Ruvalcaba Syndrome
Interventions: Everolimus blinded oral capsules, Placebo; Drug
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 5 Years to 45 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 3
States / cities: Stanford, California • Boston, Massachusetts • Cleveland, Ohio

Conditions: Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor, Childhood Craniopharyngioma, Childhood Ependymoblastoma, Childhood Grade I Meningioma, Childhood Grade II Meningioma, Childhood Grade III Meningioma, Childhood High-grade Cerebellar Astrocytoma, Childhood High-grade Cerebral Astrocytoma, Childhood Infratentorial Ependymoma, Childhood Low-grade Cerebellar Astrocytoma, Childhood Low-grade Cerebral Astrocytoma, Childhood Medulloepithelioma, Childhood Mixed Glioma, Childhood Oligodendroglioma, Childhood Supratentorial Ependymoma, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Brain Tumor, Recurrent Childhood Cerebellar Astrocytoma, Recurrent Childhood Cerebral Astrocytoma, Recurrent Childhood Ependymoma, Recurrent Childhood Medulloblastoma, Recurrent Childhood Pineoblastoma, Recurrent Childhood Subependymal Giant Cell Astrocytoma, Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor, Recurrent Childhood Visual Pathway and Hypothalamic Glioma
Interventions: cilengitide, laboratory biomarker analysis; Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: Up to 21 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2003
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Brain and Central Nervous System Tumors, Kidney Cancer, Liver Cancer, Retinoblastoma, Sarcoma
Interventions: filgrastim, busulfan, etoposide, ifosfamide, melphalan, thiotepa, stem cell transplantation, tomotherapy, total marrow irradiation, Mesna, Whole lung radiation; Biological · Drug · Procedure + 1 more
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 70 Years
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2016
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Pancreatic Neoplasms, Peutz-Jegher's Syndrome, BRCA1 Gene Mutation, BRCA2 Gene Mutation, Ataxia Telangiectasia, Familial Atypical Mole-Malignant Melanoma Syndrome, Colorectal Neoplasms, Hereditary Nonpolyposis, Hereditary Pancreatitis
Interventions: Pancreatic Cancer Screening Pathway 1, Pancreatic Cancer Screening Pathway 2; Other
Lead sponsor: University of Arkansas; Other
Eligibility: 18 Years to 99 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 1
States / cities: Little Rock, Arkansas

Conditions: Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer, Liver Cancer, Lymphoma, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions: carboplatin, dasatinib, etoposide phosphate, ifosfamide, microarray analysis, western blotting, immunohistochemistry staining method, laboratory biomarker analysis, therapeutic conventional surgery, radiation therapy; Drug · Genetic · Other + 2 more
Lead sponsor: City of Hope Medical Center; Other
Eligibility: 1 Year to 25 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2022
U.S. locations: 1
States / cities: Duarte, California

Conditions: Brain and Central Nervous System Tumors
Interventions: MOPP Regimen, Leucovorin Calcium, Mechlorethamine Hydrochloride, Methotrexate, Prednisone, Procarbazine Hydrochloride, Vincristine Sulfate, Conventional Surgery; Drug · Procedure
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: Up to 3 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1989 – 2008
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Ataxia-Telangiectasia
Interventions: vinblastine, vincristine, prednisone, daunorubicin, doxorubicin, methotrexate, cyclophosphamide, L-asparaginase, etoposide, cytarabine, mercaptopurine, dexamethasone, procarbazine, chemotherapy, intrathecal chemotherapy, steroid therapy; Drug · Procedure
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 10 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2013
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Neoplasms, Brain, Glioblastoma Multiforme, Glioblastoma of Cerebellum, Neoplasms, Astrocytoma, Astrocytoma, Cerebellar, Neuroectodermal Tumors, Neuroectodermal Tumors, Primitive, Cerebellar PNET, Childhood, Cerebellar Neoplasms, Cerebellar Neoplasms, Primary, Cerebellar Neoplasm, Malignant, Cerebellar Neoplasm Malignant Primary, Neoplasm Metastases, Neoplasm Malignant, Neoplasms, Neuroepithelial, Neoplasms, Germ Cell and Embryonal, Neoplasms by Histologic Type, Neoplasms, Glandular and Epithelial, Neoplasms, Nerve Tissue, Central Nervous System Neoplasms, Primary, Central Nervous System Neoplasms, Malignant, Nervous System Neoplasms, Neoplasms by Site, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Medulloblastoma Recurrent, HSV, Virus, Pediatric Brain Tumor, Nervous System Cancer, Primitive Neuroectodermal Tumor (PNET) of Cerebellum
Interventions: G207; Biological
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: 3 Years to 21 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2027
U.S. locations: 3
States / cities: Birmingham, Alabama • St Louis, Missouri • Houston, Texas

Conditions: Brain and Central Nervous System Tumors, Metastatic Cancer
Interventions: iodine I 131 monoclonal antibody 81C6; Radiation
Lead sponsor: Duke University; Other
Eligibility: 3 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1993 – 2010
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Brain and Central Nervous System Tumors
Interventions: VEGF inhibitor PTC299; Drug
Lead sponsor: Pediatric Brain Tumor Consortium; Network
Eligibility: 3 Years to 21 Years
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2015
U.S. locations: 8
States / cities: San Francisco, California • Washington D.C., District of Columbia • Chicago, Illinois + 5 more

Conditions: Brain and Central Nervous System Tumors, Cognitive/Functional Effects, Long-term Effects Secondary to Cancer Therapy in Children, Neurotoxicity, Psychosocial Effects of Cancer and Its Treatment, Radiation Toxicity
Interventions: Donepezil; Drug
Lead sponsor: Wake Forest University Health Sciences; Other
Eligibility: 8 Years to 17 Years
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2010
U.S. locations: 2
States / cities: Boston, Massachusetts • Winston-Salem, North Carolina

Conditions: Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome, Prenatal Benign Hypophosphatasia, Perinatal Lethal Hypophosphatasia, Odontohypophosphatasia, Adult Hypophosphatasia, Childhood-onset Hypophosphatasia, Infantile Hypophosphatasia, Hypophosphatasia, Kabuki Syndrome, Bohring-Opitz Syndrome, Narcolepsy Without Cataplexy, Narcolepsy-cataplexy, Hypersomnolence Disorder, Idiopathic Hypersomnia Without Long Sleep Time, Idiopathic Hypersomnia With Long Sleep Time, Idiopathic Hypersomnia, Kleine-Levin Syndrome, Kawasaki Disease, Leiomyosarcoma, Leiomyosarcoma of the Corpus Uteri, Leiomyosarcoma of the Cervix Uteri, Leiomyosarcoma of Small Intestine, Acquired Myasthenia Gravis, Addison Disease, Hyperacusis (Hyperacousis), Juvenile Myasthenia Gravis, Transient Neonatal Myasthenia Gravis, Williams Syndrome, Lyme Disease, Myasthenia Gravis, Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome), Isolated Klippel-Feil Syndrome, Frasier Syndrome, Denys-Drash Syndrome, Beckwith-Wiedemann Syndrome, Emanuel Syndrome, Isolated Aniridia, Axenfeld-Rieger Syndrome, Aniridia-intellectual Disability Syndrome, Aniridia - Renal Agenesis - Psychomotor Retardation, Aniridia - Ptosis - Intellectual Disability - Familial Obesity, Aniridia - Cerebellar Ataxia - Intellectual Disability, Aniridia - Absent Patella, Aniridia, Peters Anomaly - Cataract, Peters Anomaly, Potocki-Shaffer Syndrome, Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11, Silver-Russell Syndrome Due to Imprinting Defect of 11p15, Silver-Russell Syndrome Due to 11p15 Microduplication, Syndromic Aniridia, WAGR Syndrome, Wolf-Hirschhorn Syndrome, 4p16.3 Microduplication Syndrome, 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome, Autosomal Recessive Stickler Syndrome, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Stickler Syndrome, Mucolipidosis Type 4, X-linked Spinocerebellar Ataxia Type 4, X-linked Spinocerebellar Ataxia Type 3, X-linked Intellectual Disability - Ataxia - Apraxia, X-linked Progressive Cerebellar Ataxia, X-linked Non Progressive Cerebellar Ataxia, X-linked Cerebellar Ataxia, Vitamin B12 Deficiency Ataxia, Toxic Exposure Ataxia, Unclassified Autosomal Dominant Spinocerebellar Ataxia, Thyroid Antibody Ataxia, Sporadic Adult-onset Ataxia of Unknown Etiology, Spinocerebellar Ataxia With Oculomotor Anomaly, Spinocerebellar Ataxia With Epilepsy, Spinocerebellar Ataxia With Axonal Neuropathy Type 2, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 5, Spinocerebellar Ataxia Type 4, Spinocerebellar Ataxia Type 37, Spinocerebellar Ataxia Type 36, Spinocerebellar Ataxia Type 35, Spinocerebellar Ataxia Type 34, Spinocerebellar Ataxia Type 32, Spinocerebellar Ataxia Type 31, Spinocerebellar Ataxia Type 30, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 29, Spinocerebellar Ataxia Type 28, Spinocerebellar Ataxia Type 27, Spinocerebellar Ataxia Type 26, Spinocerebellar Ataxia Type 25, Spinocerebellar Ataxia Type 23, Spinocerebellar Ataxia Type 22, Spinocerebellar Ataxia Type 21, Spinocerebellar Ataxia Type 20, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 19/22, Spinocerebellar Ataxia Type 18, Spinocerebellar Ataxia Type 17, Spinocerebellar Ataxia Type 16, Spinocerebellar Ataxia Type 15/16, Spinocerebellar Ataxia Type 14, Spinocerebellar Ataxia Type 13, Spinocerebellar Ataxia Type 12, Spinocerebellar Ataxia Type 11, Spinocerebellar Ataxia Type 10, Spinocerebellar Ataxia Type 1 With Axonal Neuropathy, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia - Unknown, Spinocerebellar Ataxia - Dysmorphism, Non Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature, Spasticity-ataxia-gait Anomalies Syndrome, Spastic Ataxia With Congenital Miosis, Spastic Ataxia - Corneal Dystrophy, Spastic Ataxia, Rare Hereditary Ataxia, Rare Ataxia, Recessive Mitochondrial Ataxia Syndrome, Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature, Posterior Column Ataxia - Retinitis Pigmentosa, Post-Stroke Ataxia, Post-Head Injury Ataxia, Post Vaccination Ataxia, Polyneuropathy - Hearing Loss - Ataxia - Retinitis Pigmentosa - Cataract, Muscular Atrophy - Ataxia - Retinitis Pigmentosa - Diabetes Mellitus, Non-hereditary Degenerative Ataxia, Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity, Olivopontocerebellar Atrophy - Deafness, NARP Syndrome, Myoclonus - Cerebellar Ataxia - Deafness, Multiple System Atrophy, Parkinsonian Type, Multiple System Atrophy, Cerebellar Type, Multiple System Atrophy, Maternally-inherited Leigh Syndrome, Machado-Joseph Disease Type 3, Machado-Joseph Disease Type 2, Machado-Joseph Disease Type 1, Leigh Syndrome, Late-onset Ataxia With Dementia, Infection or Post Infection Ataxia, GAD Ataxia, Hereditary Episodic Ataxia, Gliadin/Gluten Ataxia, Friedreich Ataxia, Fragile X-associated Tremor/Ataxia Syndrome, Familial Paroxysmal Ataxia, Exposure to Medications Ataxia, Episodic Ataxia With Slurred Speech, Episodic Ataxia Unknown Type, Episodic Ataxia Type 7, Episodic Ataxia Type 6, Episodic Ataxia Type 5, Episodic Ataxia Type 4, Episodic Ataxia Type 3, Episodic Ataxia Type 1, Epilepsy and/or Ataxia With Myoclonus as Major Feature, Early-onset Spastic Ataxia-neuropathy Syndrome, Early-onset Progressive Neurodegeneration - Blindness - Ataxia - Spasticity, Early-onset Cerebellar Ataxia With Retained Tendon Reflexes, Early-onset Ataxia With Dementia, Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia, Dilated Cardiomyopathy With Ataxia, Cataract - Ataxia - Deafness, Cerebellar Ataxia, Cayman Type, Cerebellar Ataxia With Peripheral Neuropathy, Cerebellar Ataxia - Hypogonadism, Cerebellar Ataxia - Ectodermal Dysplasia, Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss, Brain Tumor Ataxia, Brachydactyly - Nystagmus - Cerebellar Ataxia, Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia, Autosomal Recessive Syndromic Cerebellar Ataxia, Autosomal Recessive Spastic Ataxia With Leukoencephalopathy, Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay, Autosomal Recessive Spastic Ataxia - Optic Atrophy - Dysarthria, Autosomal Recessive Spastic Ataxia, Autosomal Recessive Metabolic Cerebellar Ataxia, Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine, Autosomal Recessive Ataxia, Beauce Type, Autosomal Recessive Ataxia Due to Ubiquinone Deficiency, Autosomal Recessive Ataxia Due to PEX10 Deficiency, Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia, Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency, Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency, Autosomal Recessive Congenital Cerebellar Ataxia, Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome, Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity, Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency, Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect, Autosomal Recessive Cerebellar Ataxia - Saccadic Intrusion, Autosomal Recessive Cerebellar Ataxia - Psychomotor Retardation, Autosomal Recessive Cerebellar Ataxia - Blindness - Deafness, Autosomal Recessive Cerebellar Ataxia, Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly, Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation, Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy, Autosomal Dominant Spastic Ataxia Type 1, Autosomal Dominant Spastic Ataxia, Autosomal Dominant Optic Atrophy, Ataxia-telangiectasia Variant, Ataxia-telangiectasia, Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy, Autosomal Dominant Cerebellar Ataxia Type 4, Autosomal Dominant Cerebellar Ataxia Type 3, Autosomal Dominant Cerebellar Ataxia Type 2, Autosomal Dominant Cerebellar Ataxia Type 1, Autosomal Dominant Cerebellar Ataxia, Ataxia-telangiectasia-like Disorder, Ataxia With Vitamin E Deficiency, Ataxia With Dementia, Ataxia - Oculomotor Apraxia Type 1, Ataxia - Other, Ataxia - Genetic Diagnosis - Unknown, Acquired Ataxia, Adult-onset Autosomal Recessive Cerebellar Ataxia, Alcohol Related Ataxia, Multiple Endocrine Neoplasia, Multiple Endocrine Neoplasia Type II, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Multiple Endocrine Neoplasia, Type IV, Multiple Endocrine Neoplasia, Type 3, Multiple Endocrine Neoplasia (MEN) Syndrome, Multiple Endocrine Neoplasia Type 2B, Multiple Endocrine Neoplasia Type 2A, Atypical Hemolytic Uremic Syndrome, Atypical HUS, Wiedemann-Steiner Syndrome, Breast Implant-Associated Anaplastic Large Cell Lymphoma, Autoimmune/Inflammatory Syndrome Induced by Adjuvants (ASIA), Hemophagocytic Lymphohistiocytosis, Behcet's Disease, Alagille Syndrome, Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD), Lowe Syndrome, Pitt Hopkins Syndrome, 1p36 Deletion Syndrome, Jansen Type Metaphyseal Chondrodysplasia, Cockayne Syndrome, Chronic Recurrent Multifocal Osteomyelitis, CRMO, Malan Syndrome, Hereditary Sensory and Autonomic Neuropathy Type Ie, VCP Disease, Hypnic Jerking, Sleep Myoclonus, Mollaret Meningitis, Recurrent Viral Meningitis, CRB1, Leber Congenital Amaurosis, Retinitis Pigmentosa, Rare Retinal Disorder, KCNMA1-Channelopathy, Primary Biliary Cirrhosis, ZMYND11, Transient Global Amnesia, Glycogen Storage Disease, Alstrom Syndrome, White Sutton Syndrome, DNM1, EIEE31, Myhre Syndrome, Recurrent Respiratory Papillomatosis, Laryngeal Papillomatosis, Tracheal Papillomatosis, Refsum Disease, Nicolaides Baraitser Syndrome, Leukodystrophy, Tango2, Cauda Equina Syndrome, Rare Gastrointestinal Disorders, Achalasia-Addisonian Syndrome, Achalasia Cardia, Achalasia Icrocephaly Syndrome, Anal Fistula, Congenital Sucrase-Isomaltase Deficiency, Eosinophilic Gastroenteritis, Idiopathic Gastroparesis, Hirschsprung Disease, Rare Inflammatory Bowel Disease, Intestinal Pseudo-Obstruction, Scleroderma, Short Bowel Syndrome, Sacral Agenesis, Sacral Agenesis Syndrome, Caudal Regression, Scheuermann Disease, SMC1A Truncated Mutations (Causing Loss of Gene Function), Cystinosis, Juvenile Nephropathic Cystinosis, Nephropathic Cystinosis, Kennedy Disease, Spinal Bulbar Muscular Atrophy, Warburg Micro Syndrome, Mucolipidoses, Mitochondrial Diseases, Mitochondrial Aminoacyl-tRNA Synthetases, Mt-aaRS Disorders, Hypertrophic Olivary Degeneration, Non-Ketotic Hyperglycinemia, Fish Odor Syndrome, Halitosis, Isolated Congenital Asplenia, Lambert Eaton (LEMS), Biliary Atresia, STAG1 Gene Mutation, Coffin Lowry Syndrome, Borjeson-Forssman-Lehman Syndrome, Blau Syndrome, Arginase 1 Deficiency, HSPB8 Myopathy, Beta-Mannosidosis, TBX4 Syndrome, DHDDS Gene Mutations, MAND-MBD5-Associated Neurodevelopmental Disorder, Constitutional Mismatch Repair Deficiency (CMMRD), SPATA5 Disorder, SPATA5L1 Related Disorder, Acrodysostosis, Multi-systematic Smooth Muscle Dysfunction Syndrome, CRELD1 (Cysteine Rich With EGF Like Domains 1), GNB1 Syndrome, Pyruvate Dehydrogenase Complex Deficiency Disease, Beta Mannosidosis, Kbg Syndrome, Labrune Syndrome, Metachromatic Leukodystrophy (MLD), Moyamoya Disease, OPHN1 Syndrome, Oculopharyngeal Muscular Dystrophy (OPMD), TUBB3 Mutation, WOREE (WWOX-related Epileptic Encephalopathy, SCAR12, Skraban-Deardorff Syndrome, Hereditary Myopathy With Early Respiratory Failure
Interventions: Not listed
Lead sponsor: Sanford Health; Other
Eligibility: Not listed
Enrollment: 20,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2100
U.S. locations: 1
States / cities: Sioux Falls, South Dakota

Conditions: Brain and Central Nervous System Tumors
Interventions: filgrastim, sargramostim, cyclophosphamide, thiotepa, autologous bone marrow transplantation, bone marrow ablation with stem cell support, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: Herbert Irving Comprehensive Cancer Center; Other
Eligibility: Not listed
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1997 – 2008
U.S. locations: 2
States / cities: Paterson, New Jersey • New York, New York

Conditions: Brain and Central Nervous System Tumors, Lymphoma, Neuroblastoma, Retinoblastoma
Interventions: filgrastim, carboplatin, etoposide, bone marrow ablation with stem cell support, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: Roswell Park Cancer Institute; Other
Eligibility: Up to 18 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2004
U.S. locations: 3
States / cities: St Louis, Missouri • Buffalo, New York • Houston, Texas

Conditions: Brain and Central Nervous System Tumors, Carcinoma of Unknown Primary, Leukemia, Lymphoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions: topotecan hydrochloride; Drug
Lead sponsor: Pediatric Brain Tumor Consortium; Network
Eligibility: 3 Years to 21 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2005
U.S. locations: 11
States / cities: San Francisco, California • Washington D.C., District of Columbia • Chicago, Illinois + 8 more

Conditions: Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Germ Cell Tumor, Childhood Cerebral Anaplastic Astrocytoma, Childhood Cerebral Astrocytoma, Childhood Grade I Meningioma, Childhood Grade II Meningioma, Childhood Grade III Meningioma, Childhood Infratentorial Ependymoma, Childhood Oligodendroglioma, Childhood Spinal Cord Neoplasm, Childhood Supratentorial Ependymoma, Recurrent Childhood Brain Neoplasm, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Cerebellar Astrocytoma, Recurrent Childhood Cerebral Astrocytoma, Recurrent Childhood Ependymoma, Recurrent Childhood Medulloblastoma, Recurrent Childhood Pineoblastoma, Recurrent Childhood Subependymal Giant Cell Astrocytoma, Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor, Recurrent Childhood Visual Pathway Glioma
Interventions: Cediranib Maleate; Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: Up to 21 Years
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2015
U.S. locations: 11
States / cities: San Francisco, California • Washington D.C., District of Columbia • Chicago, Illinois + 7 more

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Enzastaurin in Treating Young Patients With Refractory Primary CNS Tumors

Vorinostat and Temozolomide in Treating Young Patients With Relapsed or Refractory Primary Brain Tumors or Spinal Cord Tumors

Cyproheptadine and Megestrol in Preventing Weight Loss in Children With Cachexia Caused By Cancer or Cancer Treatment

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

Lenalidomide in Treating Young Patients With Recurrent, Progressive, or Refractory CNS Tumors

The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics

VNP40101M in Treating Young Patients With Recurrent, Progressive, or Refractory Primary Brain Tumors

Double-Blind Trial of Everolimus for Improving Social Abilities in PTEN Germline Mutations

Cilengitide in Treating Children With Refractory Primary Brain Tumors

MT2004-30: Tomotherapy for Solid Tumors

Pancreatic Cancer Screening of High-Risk Individuals in Arkansas

Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients With Metastatic or Recurrent Malignant Solid Tumors

Combination Chemotherapy in Treating Children With Astrocytomas and Primitive Neuroectodermal Tumors

Study for Treatment of Cancer in Children With Ataxia-telangiectasia

HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors

Radiolabeled Monoclonal Antibody Therapy in Treating Patients With Primary or Metastatic Brain Cancers

PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors

Donepezil in Treating Young Patients With Primary Brain Tumors Previously Treated With Radiation Therapy to the Brain

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Thiotepa Followed by Peripheral Stem Cell or Bone Marrow Transplant in Treating Patients With Malignant Glioma

Chemotherapy and Stem Cell Transplantation in Treating Children With Central Nervous System Cancer

Topotecan in Treating Young Patients With Neoplastic Meningitis Due to Leukemia, Lymphoma, or Solid Tumors

AZD2171 in Treating Young Patients With Recurrent, Progressive, or Refractory Primary CNS Tumors