Neoplasms

All Conditions

Rare Diseases

Medulloblastoma

Sarcoma, Ewing

Neuroectodermal Tumors, Primitive, Peripheral

Ependymoma

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Glioma

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neuroepithelioma

Medulloblastoma, Childhood

Stefan Rutkowski, Prof.

The Register is based on the study HIT 2000 that recruited patients until 31.12.2011. All german patients with intracranial medulloblastoma, CNS-PNET, ependymoma can be included in the register. Object of the register is to maintain the quality of diagnostic standard by using of central review (Neuroradiology, Pathology, and cranio spinal fluid (CSF) cytology).

Furthermore, the register should enable to continue the collection of epidemiologic data and biological material (tumor material, CSF, and blood) for associated studies.

The Register is based on the study HIT 2000 that recruited patients until 31.12.2011. All german patients with intracranial medulloblastoma, CNS-PNET, ependymoma can be included in the register. Object of the register is to maintain the quality of diagnostic standard by using of central review (Neuroradiology, Pathology, and cranio spinal fluid (CSF) cytology).

Furthermore, the register should enable to continue the collection of epidemiologic data and biological material (tumor material, CSF, and blood) for associated studies.

The register gives recommendations for the therapy of the patients, but these are not mandatory.

Multicenter Register for Children and Young Adults With Intracranial Localized Medulloblastoma, CNS-PNET or Ependymoma

Multicenter Register for Children and Young Adults With Intracranial Localized Medulloblastoma, Central Nervous System (CNS)- Primitive Neuroectodermal Tumour (PNET), or Ependymoma

event free survival probability rates will be calculated

Universitätsklinikum Hamburg-Eppendorf

Nervous System Diseases

Symptoms and General Pathology

Ataxia

Cerebellar Ataxia

Infratentorial Neoplasms

Dyskinesias

Neurologic Manifestations

Each child in this group will receive the selected physical therapy program which include mobility exercises, strengthening exercises, balance exercises, gait training exercises, and exercises to improve physical conditioning for one-hour session three times weekly for 8 successive weeks

Each child in this group will perform one-hour session consist of two tasks (cognitive and balance task) in addition to the selected physical therapy program as control group three times weekly for 8 successive weeks.

Fifteen patients will receive Dual Task Training Program. Total Period: 8 Weeks.

Dual Task Training Program Group

Each child in this group will receive the selected physical therapy program which include mobility exercises, strengthening exercises, balance exercises, gait training exercises, and exercises to improve physical conditioning for one-hour session three times weekly for 8 successive weeks.

Control Group

Fatma Mostafa Sedeek, BSc

Medulloblastoma is a rapidly-growing tumor of the cerebellum, this area controls balance, posture and sophisticated motor functions like finer hand movements, speech, and swallowing. With the goal of, complete resection, major complications during tumor removal are usually caused by damage to the brain stem and injury to the lower cranial nerves.It has been reported that those children present Ataxia after resection. So the purpose of this study is to investigate the effectiveness of using a selected dual-task- training program to improve postural stability in those Children.

Most patients present with symptoms of hydrocephalus due to obstruction by tumor at the level of the fourth ventricle or cerebral aqueduct, and ataxia (Martin et al., 2014). If the tumor is large enough, the lesion itself can also cause mass effect and increased intracranial pressure. Furthermore, direct structural damage from the children can thus present with nausea, vomiting, ataxia,diplopia.

Ataxia in children is a common clinical sign of various origins consisting of impaired coordination of movement and balance with a lack of muscle control during voluntary activity. Ataxia is most frequently caused by dysfunction of the complex circuitry connecting the basal ganglia, cerebellum and cerebral cortex, and this type of involvement is recorded as "cerebellar ataxia.

Recently, the evidence for procedural memory training has advanced sophistication to suggest and ultimately prove that automaticity of a primary (motor task can be developed through exposure to massive repetitions practice) and the forced subconscious processing using dual task interference (Bermúdez, 2017).

The Objectives of the Study:

1- Aim of the Study: Determining the efficacy of "Dual Task Training in children with ataxia after Medulloblastoma Resection regarding "Static Balance" and "Dynamic Balance" as indicators of Postural Control

2-Specific Objectives:

1. Assess static balance by measuring center of pressure using force plate mode in the humac balance system.
2. Assess dynamic balance by using tilt mode in the humac balance system .
3. Evaluate the improvement on functional status and independence in ADL activities by functional independence measurement.
4. Determine the more effective program for rehabilitation of children with ataxia

3-Secondary Objectives:

1. Assess the degree of ataxia in children after medulloblastoma resection by SARA scale.
2. Detect the effect of cognitive interference on postural stability and balance.
3. Detect if there is improvement in cognitve function after dual task training.

Experimental controlled randomization using Block Stratified Randomized Software program, after obtaining informed consent from illegible patient or his caregiver.

Dual Task Training On Children With Ataxia After Medulloblastoma Resection

Efficacy Of Dual Task Training On Children With Ataxia After Medulloblastoma Resection

Will be assessed with Pediactris Balance Scale with scoring range with a scoring scale with minimum value (0, which means the child has no balance ) and maximum values (56, which means the child has a good balance score).

assessment of ADL (activity of daily living) with functional independence measurement questionnaire which the higher scores mean a better daily activity independence lower score means worse activity with dependency in function.

Determining the efficacy of "Dual-Task Training in children with ataxia after Medulloblastoma Resection regarding "Static Balance" and "Dynamic Balance" will be assessed with HUMAC Balance system as indicators of Postural Control

The degree of ataxia will be assessed in children by SARA (Scale of Assessment and Rating of Ataxia) with a scoring scale with minimum value (0, which means there is no Ataxia) and maximum values (40, high score of Ataxia).

Children's Cancer Hospital Egypt 57357

Recurrence

Brain Neoplasms

Brain Tumor, Childhood

Skeletally-immature children with refractory or recurrent medulloblastoma of the SHH group

Skeletally-mature subjects with refractory or recurrent medulloblastoma of the SHH group

Subjects who are eligible for the Phase I or Phase II arm of the trial and are candidates for surgery, may be enrolled in the surgical arm prior to initiation of the Phase I or Phase II treatment.

CX-4945 is supplied as 200 mg capsules delivered orally as a formulated API

CX 4945

Ralph Salloum, MD

This is a multi center, Phase I, Phase II and surgical study of the CX-4945 drug (silmitasertib sodium) for patients with recurrent SHH (Sonic Hedgehog) medulloblastoma

PRIMARY OBJECTIVES:

I. To estimate the maximum tolerated dose (MTD) and/or the recommended phase II dose (RP2D) of CX-4945 administered orally daily to skeletally-immature children with recurrent SHH (sonic hedgehog) medulloblastoma (Phase I) II. To describe the toxicity profile and define the dose-limiting toxicities (DLTs) of CX-4945 in skeletally-immature children with recurrent SHH (sonic hedgehog) medulloblastoma. (Phase I) III. To characterize the pharmacokinetics of CX-4945 administered orally daily to skeletally-immature children with recurrent SHH (sonic hedgehog) medulloblastoma. (Phase I) IV. To characterize the concentrations of CX-4945 in tumor after administration of CX-4945 and surgical resection (Surgical Study). V. To establish the safety and characterize the toxicity of 1000mg BID continuous dosing of CX-4945 in skeletally-mature patients with recurrent SHH medulloblastoma (Phase II). VI. To estimate the objective response rate associated with CX-4945 in skeletally-mature patients with recurrent SHH medulloblastoma

SECONDARY OBJECTIVES:

I. To document preliminary antitumor activity of CX-4945 in skeletally-immature children with recurrent SHH medulloblastoma (Phase I). II. To perform a genomic analysis within the confines of a Phase I study to investigate correlation between response to treatment and the presence of specific genomic alterations. and/or specific subgroups of disease (Phase I). III. To explore the ability of CX-4945 at the MTD/ RP2D to inhibit CK2-mediated signaling in tumor (Surgical Study). IV. To characterize the pharmacokinetics of CX-4945 in skeletally-mature patients with recurrent SHH medulloblastoma (Phase II). V. To perform a genomic analysis within the confines of a Phase II study to investigate correlation between response to treatment and the presence of specific genomic alterations and/or specific subgroups of disease (Phase II).

OUTLINE: Phase I component is a dose-escalation study. The Phase II component is to establish the safety of 1000mg BID given continuously.

The study will open with a safety cohort of 3 subjects who are considered skeletally-mature. The initial 3 subjects will be administered CX-4945 twice a day at the adult RP2D of 1000 mg BID or at its BSA adjusted equivalent; however, the dose will be given continuously. If there are not excessive toxicities in this cohort, the study will proceed following the Phase II design for subjects who are skeletally-mature.

Following the safety lead in, the Phase 1 component of this trial will be initiated. Skeletally-immature children with refractory or recurrent medulloblastoma of the SHH subgroup, will be administered CX-4945 twice a day on a continuous basis at a starting dose of 600mg/m2 BID which corresponds approximately to the BSA adjusted recommended Phase 2 dose (RP2D) of 1000mg. The Phase 1 study will escalate doses to determine the maximum tolerated dose skeletally-immature children.

The surgical study will be initiated after the first 3 patients in the skeletally-mature cohort are treated for initial assessment of safety and did not experience excessive toxicity. Skeletally-mature subjects with recurrent or refractory SHH medulloblastoma will be eligible as soon the surgical study is initiated and will receive drug at 1000mg BID or its BSA adjusted equivalent depending upon age and BSA. Skeletally-immature subjects will only be eligible to enroll on the surgical trial once the MTD is defined in the Phase 1 component and will receive drug at the established MTD for this cohort.

After completion of study treatment, patients are followed up to 2 years.

Testing the Safety and Tolerability of CX-4945 in Patients With Recurrent Medulloblastoma Who May or May Not Have Surgery

PBTC-053: A Pediatric Brain Tumor Consortium Phase I/ II and Surgical Study of CX-4945 in Patients With Recurrent SHH Medulloblastoma

Defined as the highest dose level at which six patients have been treated with at most one patient experiencing a dose limiting toxicity (DLT) and the next higher dose level has been determined to be too toxic.

To report the plasma drug concentration of CX-4945 on this schedule in skeletally-immature children

Percentage of patients who achieve sustained objective response.

Interval of time between the date of initiation of protocol treatment and minimum date of documentation of PD, second malignancy, death due to any cause or date of last follow-up.

Percentage of patients who achieve objective response in the skeletally-immature cohort

To report the plasma drug concentration of CX-4945 in skeletally-mature subjects

Percentage of various genomic alterations will be reported

Change in CK2 activity in tumor tissue from patients on the surgical are at baseline and after treatment.

National Cancer Institute (NCI)

American Lebanese Syrian Associated Charities (ALSAC)

Pediatric Brain Tumor Consortium

Participants will receive an individualized treatment recommendation including a combination of up to four FDA-approved drugs within 21 business days of tissue acquisition using the results of real-time high-throughput drug screening, whole exome sequencing (WES), and RNA sequencing.

Specialized Treatment Plan of up to four FDA approved drugs based on participant's screening results will be assigned by PNOC specialized tumor board

Specialized Tumor Board Treatment Plan

Aubrie Drechsler

Ashley Margol, MD

Tom Davidson, MD

John Crawford, MD, MS

Jennifer Elster, MD

Krystal Pryor

Sabine Mueller, MD, PhD

Alyssa Reddy, MD

Lindsay Kilburn, MD

Roger Packer, MD

Mohamed AbdelBaki, MD

Margaret Shatara, MD

Cassie Kline, MD, MAS

Jane Minturn, MD, PhD

Sabine Mueller, MD, PhD, MAS

Joshua Rubin, MD, PhD

Robert Wechsler-Reya, PhD

Megan Paul, MD

The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility of performing real-time drug screening on tissue taken during surgery, and of having a specialized tumor board assign a treatment plan based on the results of this screening and genomic sequencing. The aim of this trial is to allow every child and young adult with medulloblastoma to receive the most effective and least toxic therapies currently available, and will pave the way for improved understanding and treatment of these tumors in the future.

This is a single arm multi-center pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). Relapsed participants will receive an individualized treatment recommendation including up to four FDA-approved drugs based on the results of real-time high-throughput drug screening, whole exome sequencing (WES), and RNA sequencing.

PRIMARY OBJECTIVE:

I. To determine the feasibility of using the results of real-time in vitro drug screening, whole exome sequencing, and RNA sequencing of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma.

SECONDARY OBJECTIVE:

I. To determine the safety and describe the toxicity of treating children and young adults with relapsed medulloblastoma according to a specialized tumor board that makes treatment recommendations based on real-time drug screening and genomic sequencing.

EXPLORATORY OBJECTIVES:

I. To estimate the objective response rate, progression free survival at 6 months (PFS-6) and overall survival (OS) of relapsed medulloblastoma in children and young adults treated with an individualized treatment regimen.

II. To assess Quality of Life (QOL) measures in participants with relapsed medulloblastoma treated with an individualized regimen.

III. To archive tumor and normal DNA from each participant along with serial blood draw following therapies as biospecimens for later studies to determine whether circulating tumor DNA (ctDNA) sequences in the participant's blood serve as biomarkers of tumor burden, response to therapy, or development of drug resistance.

Participants may continue treatment as tolerated for up to two years or until disease progression. Participants will be followed until progression, death, or up to 5 years from start of therapy.

Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma

A Pilot Trial of Real Time Drug Screening and Genomic Testing to Determine an Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma

Individual participant data after de-identification.

Time to tissue collection will be used to determine the feasibility of using the results of real-time in vitro drug screening, WES and RNAseq of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma. Participants are expected to receive treatment plan within 21 business days

Proportion of participants with grade 3 or higher toxicities classified according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Adverse events and clinically significant laboratory abnormalities (meeting Grade 3 or higher criteria will be summarized by maximum grade and relationship to study drug(s).

Washington University School of Medicine

St. Baldrick's Foundation

Ashion Analytics

University of Washington

Pacific Pediatric Neuro-Oncology Consortium

University of California, San Francisco

Blood and Lymph Conditions

Immune System Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Neuroblastoma

Sarcoma

Lymphoma

Hodgkin Disease

Wilms Tumor

Rhabdomyosarcoma

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Neoplasms, Connective and Soft Tissue

Myosarcoma

Osteosarcoma

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Kidney Neoplasms

Urologic Neoplasms

Urogenital Neoplasms

Syndrome

Neoplastic Syndromes, Hereditary

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Kidney Diseases

Urologic Diseases

Male Urogenital Diseases

Genetic Diseases, Inborn

Central Nervous System Neoplasms

Nervous System Neoplasms

Brain Diseases

Central Nervous System Diseases

Soft Tissue Sarcoma

Lymphosarcoma

Hodgkin Lymphoma

Germ Cells Tumors

Wilms' Tumor

Ewing Sarcoma

All Drugs and Chemicals

Antineoplastic Agents

Herbal and Botanical

Estrogens

Genistein

Protective Agents

Protein Kinase Inhibitors

Enzyme Inhibitors

Phytoestrogens

Hormones

Hormone Antagonists

Soy Bean

Genistein daily throughout chemotherapy cycles 1 and 2, and placebo daily during chemotherapy cycles 3 and 4

Placebo daily throughout chemotherapy cycles 1 and 2, and genistein daily during chemotherapy cycles 3 and 4

Estrogen-like compound (isoflavone) derived from soybeans

Placebo

William C. Petersen, Jr., M.D.

Toxicities related to pediatric cancer treatment can lead to significant illness, organ damage, treatment delays, increased health care cost, and decrease in quality of life. Such toxicities are largely due to tissue damage sustained by chemotherapy, and strategies designed to limit such cellular damage to normal tissues may reduce therapy-related morbidity and mortality. In addition to their in vitro and in vivo anti-cancer effects, naturally occurring soy isoflavones have anti-inflammatory and anti-oxidant properties, and have been shown to reduce side effects of therapy in adult oncology clinical trials. This study will examine the effect of genistein, the major isoflavone component in soybeans and the most extensively studied of the soy isoflavones, on short-term side effects of myelosuppressive chemotherapy in pediatric cancer patients. Subjects will be randomized to receive either: a) 30 mg genistein daily throughout chemotherapy Cycles 1 and 2 and placebo during chemotherapy Cycles 3 and 4; or b) placebo daily during chemotherapy Cycles 1 and 2 and 30 mg genistein daily during chemotherapy Cycles 3 and 4. Investigators hypothesize that subjects will have fewer short-term therapy-related side effects during cycles of chemotherapy given in conjunction with genistein supplementation than cycles given with placebo.

This is a multi-center, randomized, double blind, placebo-controlled crossover study to evaluate the effect of soy isoflavones on the short term untoward effects of myelosuppressive chemotherapy used to treat pediatric cancers. Newly diagnosed cancer patients aged 1-21 years will be registered to the study and informed consent will be obtained prior to any study-related procedures. Stratification will be based on length of chemotherapy cycles, between 14 day and 21 day cycles. Within strata registered subjects will be randomized 1:1 to one of two schedules:

Arm A: Subjects will receive genistein daily throughout chemotherapy cycles 1 and 2, and placebo during chemotherapy cycles 3 and 4

Arm B: Subjects will receive placebo daily throughout chemotherapy cycles 1 and 2, and genistein during chemotherapy cycles 3 and 4

Subjects will be assessed for safety and efficacy during each cycle with clinical labs, cytokine panels, and physical exams. Drug compliance will be monitored by use of a patient diary as well as monitoring of serum genistein levels. Adverse events will be monitored starting on Cycle 1 Day 1 through 30 days following the last day of protocol therapy (genistein/placebo).

Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)

A Randomized, Placebo-Controlled Pilot Study of Genistein Supplementation in Pediatric Cancer Patients Receiving Myelosuppressive Chemotherapy

This includes hearing loss/tinnitus, motor neuropathy, oral mucositis

University of Virginia

Director, Pediatric Novel Therapeutics Program (Hematology/Oncology)

Genistein daily throughout chemotherapy cycles 1 and 2, and placebo daily during chemotherapy cycles 3 and 4

Genistein: Estrogen-like compound (isoflavone) derived from soybeans

Placebo: Pill that contains no medicine

Arm A: Genistein Followed by Placebo - GENISTEIN Cycles

Placebo daily throughout chemotherapy cycles 1 and 2, and genistein daily during chemotherapy cycles 3 and 4

Genistein: Estrogen-like compound (isoflavone) derived from soybeans

Placebo: Pill that contains no medicine

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Multicenter Register for Children and Young Adults With Intracranial Localized Medulloblastoma, CNS-PNET or Ependymoma	NCT02238899	Medulloblastoma... Cerebral Primit... Ependymoma		- 21 Years	Universitätsklinikum Hamburg-Eppendorf
Dual Task Training On Children With Ataxia After Medulloblastoma Resection	NCT04860934	Medulloblastoma... Childhood Cance...	Dual Task Train... Control Group	5 Years - 10 Years	Children's Cancer Hospital Egypt 57357
Testing the Safety and Tolerability of CX-4945 in Patients With Recurrent Medulloblastoma Who May or May Not Have Surgery	NCT03904862	Medulloblastoma...	CX 4945	3 Years -	Pediatric Brain Tumor Consortium
Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma	NCT05057702	Medulloblastoma Medulloblastoma... Medulloblastoma...	Specialized Tum...	12 Months - 39 Years	University of California, San Francisco
Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)	NCT02624388	Lymphoma Childhood Lymph... Solid Tumor Childhood Solid... Neuroblastoma Ewing Sarcoma Hodgkin Lymphom... Non-Hodgkin Lym... Rhabdomyosarcom... Soft Tissue Sar... Medulloblastoma Germ Cell Tumor Wilms Tumor Brain Neoplasms Medulloblastoma... Neuroectodermal...	Genistein Placebo	1 Year - 21 Years	University of Virginia
Children's and Adolescents' Medulloblastoma Molecular Subgroups in China	NCT03288168	Medulloblastoma...		- 18 Years	Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Child's Study of the Impact of PF Lesion on Motor Skills, Language, Cognitive Functioning and Social Cognition	NCT04174820	Medulloblastoma... Healthy Volunte... Fossa Posterior... Low-grade Gliom...	Functional Magn...	6 Years - 17 Years	University Hospital, Angers
Multicenter Register for Children and Young Adults With Intracranial Localized Medulloblastoma, CNS-PNET or Ependymoma	NCT02238899	Medulloblastoma... Cerebral Primit... Ependymoma		- 21 Years	Universitätsklinikum Hamburg-Eppendorf
Establishment and Clinical Application of Risk Classification Model Based on Molecular Typing of Medulloblastoma in Children	NCT05406947	Medulloblastoma...	Prospective obs...	6 Months - 18 Years	Beijing Tiantan Hospital

Spots Global Cancer Trial Database for medulloblastoma, childhood