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Active, not recruitingNCT03296371Updated Feb 6, 2026

Genetic Mutational Analysis of Saliva or Buccal Mucosa Samples From Patients With Embryonal or Alveolar Rhabdomyosarcoma

An observational study in Alveolar Rhabdomyosarcoma and Embryonal Rhabdomyosarcoma, sponsored by Children's Oncology Group. Active, not recruiting at 1 site in United States. Open to participants aged Up to 50 Years. Per ClinicalTrials.gov, last updated 2026-02-06.

Sponsored by Children's Oncology Group · Observational

Study type
Observational
Model
Family-based
Time perspective
Prospective
Enrollment
900
Ages
Up to 50 Years
Sex
All
01

Study summary

This research trial studies genetic mutations in saliva or buccal mucosa samples from patients with embryonal or alveolar rhabdomyosarcoma. Identifying gene mutations may help doctors learn about the prognosis of patients with embryonal or alveolar rhabdomyosarcoma.

Read the detailed description

PRIMARY OBJECTIVES:

I. To identify novel recurrent de novo germline mutations among rhabdomyosarcoma (RMS) case-parent trios.

II. To identify the frequency of de novo germline mutations in cancer predisposition genes among RMS case-parent trios.

SECONDARY OBJECTIVES:

I. To conduct ?deep phenotyping? of children diagnosed with RMS utilizing questionnaire data and information from medical records.

OUTLINE:

Patients and their parents undergo collection of saliva or buccal mucosa samples for genetic mutational analysis. Germline deoxyribonucleic acid (DNA) from saliva or buccal mucosa is evaluated via whole exome sequencing.

02

Conditions studied

  • Alveolar Rhabdomyosarcoma
  • Embryonal Rhabdomyosarcoma
03

Who can participate

Ages eligible
Up to 50 Years
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Patients with embryonal or alveolar rhabdomyosarcoma enrolled on ACCRN07 and/or APEC14B1 and registered with Children's Oncology Group (COG)

Eligibility criteria

Inclusion Criteria:

  • The patient must be enrolled on ACCRN07 and/or APEC14B1 and registered with COG by a North American member institution
  • The patient must have a diagnosis of embryonal rhabdomyosarcoma or alveolar rhabdomyosarcoma
  • The patient must be diagnosed with rhabdomyosarcoma between January 1, 2012 and November 30, 2019
  • Concomitant treatment on a therapeutic trial is not required
  • The patient must have at least one biological parent alive and willing to participate
  • All questionnaire respondents must understand English or Spanish
  • All patients and/or their parents or legal guardians must sign a written informed consent
  • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
04

Study design

Observational model
Family-based
Time perspective
Prospective
Enrollment
900 participants (estimated)
Biospecimen retention
Samples with dna

Groups and cohorts

  • Ancillary-Correlative (biospecimen collection)

    Patients and their parents undergo collection of saliva or buccal mucosa samples for genetic mutational analysis. Germline DNA from saliva or buccal mucosa is evaluated via whole exome sequencing.

    Procedure: Biospecimen Collection · Other: Laboratory Biomarker Analysis · Other: Questionnaire Administration

Interventions

  • ProcedureBiospecimen Collection

    Undergo saliva or buccal mucosa collection

  • OtherLaboratory Biomarker Analysis

    Correlative studies

  • OtherQuestionnaire Administration

    Ancillary studies

05

What researchers measure

Primary outcomes

  1. Novel recurrent de novo germline mutation identification

    Will analyze de novo single-nucleotide variants (SNVs), copy-number variants (CNVs), and insertions/deletions (INDELs) obtained through next-generation exome sequencing of rhabdomyosarcoma (RMS) case-parent trios.

    Time frame: Up to 3 years

  2. Frequency of de novo germline mutations in cancer predisposition genes

    Will conduct targeted sequencing using samples collected from the case and his/her parents in order to determine the prevalence of novel de novo mutations in cancer-syndrome genes associated with RMS.

    Time frame: Up to 3 years

Secondary outcomes

  1. Deep phenotyping of children diagnosed with rhabdomyosarcoma utilizing questionnaires and medical record information

    Analyses will be descriptive in nature.

    Time frame: Up to 3 years

06

Study locations

1 site
  • Childrens Oncology Group
    Philadelphia, Pennsylvania 19104, United States
07

Registry details

Key details

Study ID
NCT03296371
Lead sponsor
Children's Oncology Group
Collaborators
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Sep 28, 2017
Start date
Oct 23, 2017
Primary completion
Dec 31, 2022
Completion
Dec 31, 2026 (estimated)
Last update
Feb 6, 2026

Study contacts

Philip Lupo
principal investigator · Children's Oncology Group

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.

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