Symptoms and General Pathology

All Conditions

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Rhabdoid Tumor

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Histiocytosis

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Lymphoma, Non-Hodgkin

Neoplasms by Histologic Type

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Disease Attributes

Neoplasms, Connective and Soft Tissue

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

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Neoplasms, Connective Tissue

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Urogenital Diseases

Female Urogenital Diseases

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Female Urogenital Diseases and Pregnancy Complications

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Lung Diseases, Interstitial

Respiratory Tract Diseases

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Ewing Sarcoma

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Neuroepithelioma

Malignant Germ Cell Tumor

Antineoplastic Agents

All Drugs and Chemicals

Vemurafenib

Protein Kinase Inhibitors

Enzyme Inhibitors

Patients receive vemurafenib PO BID on day 1-28. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.

Laboratory Biomarker Analysis

AeRang Kim

This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body (advanced) and have come back (recurrent) or do not respond to treatment (refractory). Vemurafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

PRIMARY OBJECTIVE:

I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with vemurafenib with advanced solid tumors (including central nervous system \[CNS\] tumors), lymphomas or histiocytic disorders that harbor activating BRAF V600 mutations.

SECONDARY OBJECTIVES:

I. To estimate the progression free survival in pediatric patients treated with vemurafenib with advanced solid tumors (including CNS tumors), lymphomas or histiocytic disorders that harbor activating BRAF V600 mutations.

II. To obtain information about the tolerability of vemurafenib in children with relapsed or refractory cancer.

EXPLORATORY OBJECTIVE:

I. To explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid (DNA).

OUTLINE:

Patients receive vemurafenib orally (PO) twice daily (BID) on day 1-28. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.

Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Vemurafenib in Patients With Tumors Harboring BRAF V600 Mutations

Will explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid. Will be summarized with simple summary statistics and will be descriptive in nature.

ORR will be defined as complete response + partial response and assessed by Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1). Will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed using the Wilson score interval method.

PFS will be defined as time from the initiation of protocol treatment to the I of any of the following events: disease progression or disease recurrence or death from any cause. Patients with local calls of disease progression (i.e. calls made by the treating institution), will be counted as having had an event, even if the central review does not declare progression. PFS along with the confidence intervals will be estimated using the Kaplan-Meier method.

Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. All patients who receive at least one dose of protocol therapy will be considered in the evaluation of toxicity.

National Cancer Institute (NCI)

Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.

Treatment (Vemurafenib)

Age, Categorical

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Results Reporting Coordinator

Objective Response Rate (ORR)

Progress Free Survival (PFS)

Percentage of Patients Experiencing Grade 3 or Higher Adverse Events

Changes in Tumor Genomics

Overall Study

Gland and Hormone Related Diseases

Nutritional and Metabolic Diseases

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Glioblastoma

Brain Neoplasms

Astrocytoma

Meningioma

Oligodendroglioma

Body Weight

Nausea

Vomiting

Liposarcoma

Weight Loss

Body Weight Changes

Medulloblastoma

Carcinosarcoma

Mixed Tumor, Mullerian

Testicular Neoplasms

Sarcoma, Endometrial Stromal

Chondrosarcoma

Fibrosarcoma

Wasting Syndrome

Cachexia

Hemangiosarcoma

Leiomyosarcoma

Sarcoma, Synovial

Neurofibrosarcoma

Lymphoma, Large-Cell, Anaplastic

Leukemia, Myelomonocytic, Acute

Gliosarcoma

Sarcoma, Alveolar Soft Part

Sarcoma, Clear Cell

Leukemia, Promyelocytic, Acute

Leukemia, Monocytic, Acute

Choroid Plexus Neoplasms

Leukemia, Erythroblastic, Acute

Leukemia, Megakaryoblastic, Acute

Mast-Cell Sarcoma

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Malignant Mixed Mullerian Tumor

Ovarian Carcinosarcoma

Endometrial Stromal Sarcoma

Brain Tumor, Childhood

Anaplastic Astrocytoma

Ovarian Germ Cell Tumor

Synovial Sarcoma

Malignant Peripheral Nerve Sheath Tumor

Epithelioid Sarcoma

Testicular Germ Cell Tumor

Anaplastic Large Cell Lymphoma

Uterine Sarcoma

Nonseminomatous Germ Cell Tumor

Anaplastic Oligodendroglioma

Optic Pathway Glioma

Alveolar Soft Part Sarcoma

Diffuse Astrocytoma

Childhood Brain Stem Glioma

Supratentorial Primitive Neuroectodermal Tumor

Pilocytic Astrocytoma

Uterine Carcinosarcoma

Anti-Allergic Agents

Dermatologic Agents

Gastrointestinal Agents

Cyproheptadine

Histamine H1 Antagonists

Histamine

Histamine phosphate

Histamine Antagonists

Neurotransmitter Agents

Serotonin

Patients receive oral cyproheptadine hydrochloride twice daily for 8 weeks.

Patients receive an oral placebo twice daily for 8 weeks.

cyproheptadine hydrochloride

placebo

Jeffrey P. Krischer, PhD

RATIONALE: Cyproheptadine hydrochloride may prevent weight loss caused by cancer or cancer treatment. It is not yet known whether cyproheptadine is more effective than a placebo in preventing weight loss in young patients receiving chemotherapy for cancer.

PURPOSE: This randomized phase III trial is studying cyproheptadine hydrochloride to see how well it works in preventing weight loss in young patients receiving chemotherapy for cancer.

OBJECTIVES:

Primary

* To determine the effect of cyproheptadine hydrochloride in the prevention of cancer- or treatment-related weight loss (defined as ≥ 5% reduction in weight from baseline measurement) in children who are initiating a course of moderately or highly emetic chemotherapy.

Secondary

* To investigate the effect of cyproheptadine HCl on the change in weight for age scores after 8 weeks of study drug administration in comparison to placebo.
* Investigate the relationship between the secondary outcome variables (prealbumin, triceps skin fold, mid-upper arm circumference, and weight loss)from baseline to end of treatment in each group (treatment and placebo) separately.

OUTLINE: This is a multicenter study. Patients are stratified according to enrolling center and steroid use with cancer treatment (yes vs no). Study agent can start anytime up to and including day 28 after the first dose of chemotherapy.

* Arm I: Patients receive oral cyproheptadine hydrochloride twice daily for 8 weeks.
* Arm II: Patients receive an oral placebo twice daily for 8 weeks.

Patients undergo weight and height measurements at baseline and at each follow-up visit in weeks 4 and 8 to evaluate the effect of cyproheptadine hydrochloride and duration of response. Patients or parents complete medicine logs at each follow-up visit in weeks 4 and 8 to evaluate drug compliance and tolerance. Patients also undergo measures of nutrition; and measures of body composition, lean body mass, and fat percentage using standardized equipment and procedures for measuring triceps skin fold and mid-arm muscle circumference at baseline and at the end of the study.

Patients undergo blood sample collection at baseline and at the end of the study for biomarker studies. Samples are analyzed for pre-albumin levels.

Cyproheptadine in Preventing Weight Loss in Children Receiving Chemotherapy for Cancer

Prevention of Cancer/Treatment-Related Weight Loss in Children at High Nutritional Risk

University of South Florida

Patients receive oral cyproheptadine hydrochloride twice daily for 8 weeks.

cyproheptadine hydrochloride: Given orally

Arm I Cyproheptadine Hydrochloride

Patients receive an oral placebo twice daily for 8 weeks.

placebo: Given orally

Arm II Placebo

Total

Angelina Fink, Research Base Administrator

Participant With Weight Loss ≥ 5% at the 8- Week Assessment When Compared to Baseline

Severity of Weight Loss

Pattern of Weight in the Study Population

Rhabdomyosarcoma, Embryonal

Trabectedin

Antineoplastic Agents, Alkylating

Alkylating Agents

Patients receive trabectedin over 3 hours on day 1. Treatment repeats every 21 days for up to 26 courses in the absence of disease progression or unacceptable toxicity. A cohort of 6 patients will be enrolled at the 1.3 mg/m2 dose level.

Patients receive trabectedin over 3 hours on day 1. Treatment repeats every 21 days for up to 26 courses in the absence of disease progression or unacceptable toxicity. Six toxicity-evaluable patients are assigned this treatment.

Patients receive trabectedin over 3 hours on day 1. Treatment repeats every 21 days for up to 26 courses in the absence of disease progression or unacceptable toxicity.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)	NCT03220035	Advanced Malign... Ann Arbor Stage... Ann Arbor Stage... Ependymoma Ewing Sarcoma Hepatoblastoma Langerhans Cell... Malignant Germ ... Malignant Gliom... Osteosarcoma Peripheral Prim... Recurrent Child... Recurrent Child... Recurrent Malig... Recurrent Neuro... Refractory Mali... Refractory Neur... Refractory Non-... Refractory Prim... Rhabdoid Tumor Rhabdomyosarcom... Soft Tissue Sar... Wilms Tumor	Laboratory Biom... Vemurafenib	12 Months - 21 Years	National Cancer Institute (NCI)
Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)	NCT03220035	Advanced Malign... Ann Arbor Stage... Ann Arbor Stage... Ependymoma Ewing Sarcoma Hepatoblastoma Langerhans Cell... Malignant Germ ... Malignant Gliom... Osteosarcoma Peripheral Prim... Recurrent Child... Recurrent Child... Recurrent Malig... Recurrent Neuro... Refractory Mali... Refractory Neur... Refractory Non-... Refractory Prim... Rhabdoid Tumor Rhabdomyosarcom... Soft Tissue Sar... Wilms Tumor	Laboratory Biom... Vemurafenib	12 Months - 21 Years	National Cancer Institute (NCI)
Cyproheptadine in Preventing Weight Loss in Children Receiving Chemotherapy for Cancer	NCT01132547	Cancer	cyproheptadine ... placebo	2 Years - 21 Years	University of South Florida
Trabectedin in Treating Young Patients With Recurrent or Refractory Soft Tissue Sarcoma or Ewing's Family of Tumors	NCT00070109	Previously Trea... Recurrent Child... Recurrent Child... Recurrent Ewing... Peripheral Prim...	trabectedin pharmacological...	12 Months - 50 Years	Children's Oncology Group
Liquid Biopsy in Ewing Sarcoma and Osteosarcoma as a Prognostic And Response Diagnostic: LEOPARD	NCT06068075	Ewing Sarcoma Ewing Sarcoma o... Ewing Sarcoma o... Peripheral Prim... Peripheral Prim... Peripheral Prim... High-grade Oste...	FoundationOne L...	12 Months - 50 Years	Dana-Farber Cancer Institute

Spots Global Cancer Trial Database for peripheral primitive neuroectodermal tumor