Children and Adults With Chordoma

Natural History Study of Children and Adults With Chordoma

Background:

Chordoma is a rare type of bone cancer. It occurs in the skull base or spine. Researchers want to study people with chordoma in different ways. They hope this will help them design better future treatments and supportive care studies for this disease.

Objective:

To learn more about chordoma by looking at its clinical course, how it appears on imagine scans, and how it responds to therapies and treatments.

Eligibility:

People ages 2 and older with chordoma who are enrolled in NCI protocol 19-C-0016

Design:

Participants will be screened with their medical history.

Participants will have a visit to examine their disease. This will include:

• Physical exam
• Neurologic exam
• CT scan and MRI: Participants will lie on a table. The table will slide into a machine. The machine will take pictures of the body.

Participants will have other tests every 6-12 months:

• Smell test
• Surveys to assess their emotional, physical, and behavioral well-being and needs
• Cognitive function tests

Participants or their home doctors will be contacted every 6 12 months. They will be asked to provide information about their disease. This could include test results and imaging evaluations.

Some participants may be asked to come to the clinic for more visits.

Study Overview

• Status: Recruiting
• Conditions: Chordoma

Detailed Description

Background:

• Chordomas are a rare tumor with an incidence of 325 new cases per year in the United States. Pediatric chordomas are very rare and comprise only 5% of all chordoma cases.
• The standard therapy for localized primary or recurrent chordomas is surgical resection. However, complete surgical resection is often not possible due to the location of the

chordoma.

-For chordomas that cannot be surgically resected, treatment options are limited. Currently,

no standard therapy approach exists for recurrent chordomas. In addition, there are no FDA approved medical therapies for chordoma.

• The natural history of pediatric and adult chordoma is incompletely understood. Patients with chordoma seek expert advice in the management of their care.
• The NCI has basic and clinical expertise and research interest in chordoma. The planned natural history study as part of the NCI POB Rare Tumor Patient Engagement Network (RTPEN) will allow for comprehensive evaluation and recommendations to these patients while longitudinally collecting clinical, epidemiologic and biological data.

Objective:

-To characterize the natural history of chordoma, including clinical presentation and patterns of disease progression.

Eligibility:

• Subjects with histologically documented chordoma.
• Age greater than or equal to 2 years old

Design:

-This protocol is a subprotocol to protocol 19-C-0016: Natural History and Biospecimen

Acquisition Study for Children and Adults with Rare Solid Tumors . After enrollment on the master protocol and undergoing evaluations detailed, patients will be enrolled on this subprotocol specific for chordoma.

-Medical histories will be documented, and patients followed throughout the course of their disease, with particular attention to patterns of disease recurrence and progression, response to therapies, duration of responses and patient reported outcomes. Tumor growth rates will also be calculated throughout the course of the disease.

• Study Type: Observational
• Enrollment (Estimated): 300

Contacts and Locations

• Study Contact: Name: Barbara (BJ) Thomas, R.N.; Phone Number: (240) 858-3633; Email: barbara.thomas2@nih.gov
• Study Contact Backup: Name: Brigitte C Widemann, M.D.; Phone Number: (240) 760-6203; Email: widemanb@mail.nih.gov

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • Recruiting
      • National Institutes of Health Clinical Center
      • Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office; Phone Number: 888-624-1937

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

primary clinical

Description

• INCLUSION CRITERIA:
• Ability of subject or Legally Authorized Representative (LAR) to understand and the willingness to sign a written consent document.
• Subjects with histologically documented chordoma
• Age greater than or equal to 2 years old
• Subjects must be enrolled into NCI protocol 19-C-0016: Natural History and Biospecimen Acquisition Study for Children and Adults with Rare Solid Tumors .

EXCLUSION CRITERIA:

-None

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
1/Cohort 1; Subjects with confirmed chordoma.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To characterize the natural history of chordoma, including clinical presentation and patterns of disease progression	Natural history of chordoma, including clinical presentation and patterns of disease progression	10 years

Collaborators and Investigators

• Sponsor: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Brigitte C Widemann, M.D., National Cancer Institute (NCI)

Publications and helpful links

General Publications

• Wedekind MF, Widemann BC, Cote G. Chordoma: Current status, problems, and future directions. Curr Probl Cancer. 2021 Aug;45(4):100771. doi: 10.1016/j.currproblcancer.2021.100771. Epub 2021 Jul 1.

Helpful Links

• NIH Clinical Center Detailed Web Page

Study record dates

Study Major Dates

• Study Start (Actual): April 16, 2019
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: April 9, 2019
• First Submitted That Met QC Criteria: April 9, 2019
• First Posted (Actual): April 10, 2019

Study Record Updates

• Last Update Posted (Actual): December 22, 2023
• Last Update Submitted That Met QC Criteria: December 21, 2023
• Last Verified: December 14, 2023

More Information

Terms related to this study

• Keywords: Natural History; Rare Tumor; Comprehensive Evaluation and Recommendations; Collecting Clinical, Epidemiologic and Biological Data
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Germ Cell and Embryonal; Chordoma
• Other Study ID Numbers: 190082; 19-C-0082

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: .All IPD recorded in the medical record will be shared with intramural investigators upon request.
• IPD Sharing Time Frame: Clinical data available during the study and indefinitely.
• IPD Sharing Access Criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No