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Morphine

DNA methylation analysis

fluorescence in situ hybridization

gene expression analysis

reverse transcriptase-polymerase chain reaction

laboratory biomarker analysis

Caroline Y. Hu, MD

RATIONALE: Studying samples of tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.

PURPOSE: This research study is studying biomarkers in patients with rhabdomyosarcoma.

OBJECTIVES:

* Determine genome-wide alterations in DNA methylation in ARMS and ERMS.
* Determine genome-wide DNA copy number alterations in ARMS and ERMS.
* Determine pathogenic genes and pathways by integrative genomic analysis.

OUTLINE: Genome-wide DNA-methylation analysis on ARMS, ERMS, and normal human skeletal myoblasts will be conducted using the HELP (HpaII tiny fragment Enrichment by Ligation-mediated PCR) assay. The methylation status of 1.3 million CpGs at promoters, gene bodies, and intergenic areas will be analyzed. Parallel gene expression analysis will be done and correlated with changes in methylation to uncover genes regulated by epigenetic alterations and altered by genomic losses or gains.

Genes that are altered by both genetic and epigenetic alterations in different sets of patients will be selected by the MIGHT (Multi-dimensional Integration of Genomic data from Human Tissues) algorithm to uncover new genes that are potentially involved in the pathogenesis of ARMS and ERMS. Gene ontology, pathway, and DNA motif analysis algorithms, and other computational approaches will be used to determine the biological consequences of the changes. Prioritized set of epigenetic and genetic alterations will be validated by bisulfite MassArray, FISH, and qRT-PCR in larger numbers of ARMS and ERMS samples.

Biomarkers in Patients With Rhabdomyosarcoma

Integrative Epigenomic Approach to Gene Discovery in Rhabdomyosarcoma (RMS)

National Cancer Institute (NCI)

Children's Oncology Group

Rhabdomyosarcoma, Embryonal

Archived tumor tissue samples are analyzed for CRKL expression.

Lee Helman, MD

This research trial studies biomarkers in tumor tissue samples from younger patients with rhabdomyosarcoma. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer

PRIMARY OBJECTIVES:

I. To demonstrate CRKL expression at the protein level in rhabdomyosarcoma tumors by tissue microarray (TMA).

OUTLINE:

Archived tumor tissue samples are analyzed for CRKL expression.

Biomarkers in Tumor Tissue Samples From Younger Patients With Rhabdomyosarcoma

Observational - CRKL Expression at the Protein Level in Rhabdomyosarcoma Tumors by Tissue Microarray (TMA)

RNA analysis

Hajime Hosoi, MD

RATIONALE: Studying samples of blood from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.

PURPOSE: This research trial studies biomarkers in samples from patients with rhabdomyosarcoma.

OBJECTIVES:

* Determine whether circulating muscle-specific microRNAs, especially serum miR-206, are potential biomarkers for rhabdomyosarcoma (RMS).

OUTLINE: Archived serum samples are analyzed for miR-206 expression.

PROJECTED ACCRUAL: A total of 15 samples from patients with alveolar rhabdomyosarcoma (RMS), 15 from patients with embryonal RMS, and 10 from patients without RMS will be accrued for this study.

Studying Biomarkers in Samples From Patients With Rhabdomyosarcoma

A Retrospective Study on the Diagnostic Value of Serum miR-206 in Rhabdomyosarcoma

Antineoplastic Agents

Apatinib

Protein Kinase Inhibitors

Enzyme Inhibitors

All eligible patients signed informed consent. Three-dimensional conformal intensity modulation (IMRT) technique was used to treat the patients with radiation doses 45 Gy - 54 Gy. All patients took apatinib mesylate tablets 250 mg QD orally from 1 week before radiotherapy to the whole radiotherapy period. They were required to take apatinib mesylate tablets with warm boiling water half an hour after meal. The daily medication time should be as consistent as possible.

Apatinib mesylate tablets were orally administered 250 mg QD from 1 week before radiotherapy to the whole radiotherapy period. (Appatinib is no longer used after radiotherapy)

Apatinib mesylate tablets

Three-dimensional conformal intensity modulation (IMRT) technique was used for post-operative radiotherapy. The radiation dose was 45-54 Gy.

Three-dimensional conformal intensity modulation

Mawei Jiang, MD

Chuanying Zhu, MD

The purpose of this study is to evaluate the efficacy and safety of radiotherapy combined with apatinib mesylate in the treatment of rhabdomyosarcoma in children.

This study is a Interventional study. The target group was 3-18 years old, and the pathological stage was group II-IV recurrence or distant metastasis. A total of 48 patients were enrolled in this study. The treatment was radiotherapy combined with apatinib. The main outcome measures were ORR, DCR , OS , quality of life and drug safety. The main safety indicators were vital signs, laboratory indicators, adverse events (AE), and severe adverse events (SAE). This study is expected to be enrolled for 6 months.

Simons two-stage design method requires the enrollment of 13 patients in the first stage. If more than 7 patients fail to reach the expected end point, the first stage fails and the patients do not need to enter the second stage. A total of 48 patients were enrolled. If more than 30 patients did not reach the expected end point, the overall trial failed.

Efficacy and Safety of Radiotherapy Combined With Apatinib Mesylate in the Treatment of Rhabdomyosarcoma in Children

Refers to the proportion of patients whose tumors have shrunk to a certain extent and maintained for a certain period of time, including CR and PR cases. Objective remission of solid tumors was assessed by RECIST 1.1. Subjects must be accompanied by measurable tumor lesions at baseline. According to RECIST 1.1, the criteria for evaluating efficacy are complete remission (CR), partial remission (PR), stability (SD), and progression (PD).

The percentage of patients whose tumors shrink or remain stable for a certain period of time

The time from enrollment to death for any reason. For those who had lost interviews before death, the last follow-up time was usually calculated as the time of death.

Quality of life score refer to EORTC qlq-c30 (version 3, Chinese version). Quality of Life Questionnare-Core 30 prepared by The European O-rganization for Reasearch and Treatment of Cancer.

EORTC qlq-c30 (V3.0) is the core scale for all cancer patients, with a total of 30 items. Items 29 and 30 are rated on a scale of 1 to 7 according to their answer choices. The other items are divided into four levels: totally not, a little bit, a lot and very much, and are scored on a scale of 1 to 4.

The higher the score of functional domain and general health domain, the better the functional status and quality of life. The higher the score of symptom domain, the more symptoms or problems (the worse the quality of life).

The safety of the drug was evaluated by NCI-CTCAE 4.0. Common Terminology Criteria Adverse Events Version 4.0 prepared by National Cancer Institute.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Biomarkers in Patients With Rhabdomyosarcoma	NCT01466283	Sarcoma	DNA methylation... fluorescence in... gene expression... reverse transcr... laboratory biom...	-	Children's Oncology Group
Biomarkers in Tumor Tissue Samples From Younger Patients With Rhabdomyosarcoma	NCT01496573	Alveolar Childh... Childhood Rhabd... Embryonal Child...	laboratory biom...	-	Children's Oncology Group
Studying Biomarkers in Samples From Patients With Rhabdomyosarcoma	NCT01433237	Sarcoma	RNA analysis gene expression... laboratory biom...	-	Children's Oncology Group
Efficacy and Safety of Radiotherapy Combined With Apatinib Mesylate in the Treatment of Rhabdomyosarcoma in Children	NCT03868852	Childhood Rhabd...	Apatinib mesyla... Three-dimension...	3 Years - 18 Years	Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Biomarkers in Patients With Rhabdomyosarcoma	NCT01466283	Sarcoma	DNA methylation... fluorescence in... gene expression... reverse transcr... laboratory biom...	-	Children's Oncology Group

Spots Global Cancer Trial Database for childhood rhabdomyosarcoma