NCI Childhood Cancer Data Initiative (CCDI) Led Pediatric, Adolescent, and Young Adult Rare Cancer Registry for Very Rare Solid Tumors

NCT07489378 | Recruiting

• 2 other identifiers: 10002496002496-C
• Study Type: observational
• Target: 4,000
• Locations: 1 country
• Sites: 1

Brief Summary

Background: All childhood cancers are rare, but some are called very rare. Very rare cancers are diagnosed in 2 or fewer out of 1 million people each year. Researchers want to gather data so they can learn more about these very rare cancers. They hope to use the data to develop future treatments. Objective: To gather data for a registry of very rare cancers found in children, teens, and young adults. Eligibility: People aged 1 month to 39 years newly diagnosed (within the past year) with a very rare cancer. Design: Participation will be by phone or email. No clinic visits are required. Researchers will look at the participant s medical records. They will ask for samples of tumor tissue that were already removed. They will use the samples for genetic testing. The results of these tests will be sent to the participant s own doctors. Some participants will be asked for saliva or cheek swab samples. They will receive a kit in the mail. They will spit into a tube or swab the inside of their cheek. They will mail the sample back to the lab. Participants will fill out questionnaires once a year for 5 years. They will answer questions about: Family history, such as other cancers in the family and their income, work, and education. Demographics, such as their gender, nationality, ethnicity, education, and work history. Symptoms and treatment for their cancer. This may include level of pain, and emotional and physical well-being. Participants data will be added to a secure database for other researchers. Their data will be anonymous.

Conditions

Solid Tumor; Pediatric Rare Tumors; Other Solid Tumors; Very Rare Cancers; Very Rare Tumors

Keywords

Longitudinal Study; Registry; Patient Reported Outcomes; Family History; Molecular Characterization

Outcome Measures

Primary Outcomes (1)

• To establish a longitudinal observational study and registry for very rare pediatric and AYA solid tumors

  Percentage of participants from identified recruitment sources will be tabulated and described. An analysis of the ability to adequately obtain medical records at initial evaluation and follow-up to perform medical data extraction, which is critical to establishing a registry and longitudinal observational study will be assessed.

  Through 5 years after enrollment

Secondary Outcomes (2)

• To evaluate the feasibility of PRO using validated reporting platforms suitable for pediatric and AYA populations

  At time of enrollment/study entry, and 2 and 5 years after enrollment

• To conduct comprehensive clinical molecular characterization, utilizing CCDI MCI

  At time of enrollment/study entry

Study Arms (1)

1/ Cohort 1

Participants with very rare tumors

Other: Natural history study of individuals with very rare tumors

Interventions

Natural history study of individuals with very rare tumors OTHER

We will collect information about the initial presentation and diagnosis of the disease, management, and tumor treatment history. Participants or parent/guardian will be asked to complete questionnaires and patient-reported outcome (PRO) instruments. Pathology materials (e.g., tissue samples, slides, or blocks) and saliva and/or buccal sample will be requested.

1/ Cohort 1

Eligibility Criteria

• Age: 1 Month - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This is a longitudinal observational study and registry for children, adolescents and young adults (AYAs) with very rare cancers.

You may qualify if:

• History of newly diagnosed (within 1 year of diagnosis) very rare solid tumor (defined as an estimated 2 incident cases per million per year).
• Age \>= 1 month and \<= 39 years at the time of diagnosis.
• Participants must have established care with a local treating physician.
• Ability of the participant, parent/guardian, or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.

You may not qualify if:

• Diagnosis of any of the following at any time:
• Ewing Sarcoma
• Osteosarcoma
• Rhabdomyosarcoma
• Diffuse midline glioma (H3K27 altered)
• Atypical teratoid rhabdoid tumor
• Pleuropulmonary blastoma
• Common adult cancers that occur in pediatric/AYA populations (i.e., colorectal cancer, breast cancer)
• The participant is unlikely to comply with the terms of the protocol.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Links

• NIH Clinical Center Detailed Web Page

Study Officials

• Mary F Wedekind Malone, D.O.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mary F Wedekind Malone, D.O.

CONTACT

(240) 858-3765; maryfrances.wedekindmalone@nih.gov

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 21, 2026
• First Posted: March 24, 2026
• Study Start (Estimated): August 5, 2026
• Primary Completion (Estimated): April 1, 2035
• Study Completion (Estimated): April 1, 2037
• Last Updated: July 31, 2026

Record last verified: 2026-06-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be made available as soon as possible or at the time of associated publication. Data not published in a manuscript will be shared via public source once the data set completes QC.
• Access Criteria: Clinical data will be made available upon request and with the permission of the study PI. Genomic data are made available via dbGAP through requests to the data custodians.

Locations

US (1)