NCI CCDI registry for very rare pediatric and young adult solid tumors

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

This registry collects medical records and tumor genetic data from children, teens, and young adults (1 month–39 years) newly diagnosed with very rare solid tumors to help researchers learn more and guide future treatments.

Quick facts

• Study type: Observational
• Enrollment: 4000 (estimated)
• Ages: 1 Month to 120 Years
• Sex: All
• Sponsor: National Institutes of Health Clinical Center (CC) (NIH)
• Locations: 1 site (Bethesda, Maryland)
• Trial ID: NCT07489378 on ClinicalTrials.gov

What this trial studies

This is a longitudinal observational registry run by the NCI Childhood Cancer Data Initiative that enrolls children, adolescents, and young adults (≤39 years at diagnosis) with very rare solid tumors diagnosed within the past year. Participation is remote by phone or email with no clinic visits required; researchers review medical records, request previously removed tumor tissue for molecular testing, and may mail saliva or cheek‑swab kits for germline samples. Molecular testing results are shared with the participant's treating physicians and standardized data are compiled into a central registry. The project aims to describe natural history, molecular features, and treatment patterns across fragmented rare‑tumor populations to enable future research and therapy development.

Who should consider this trial

Good fit: Patients aged 1 month to 39 years with a newly diagnosed (within one year) very rare solid tumor who have an established treating physician and can provide consent are ideal candidates.

Not a fit: Patients with excluded tumor types (for example, Ewing sarcoma, osteosarcoma, rhabdomyosarcoma, diffuse midline glioma H3K27‑altered, atypical teratoid rhabdoid tumor, pleuropulmonary blastoma, or common adult cancers presenting in pediatrics) or those diagnosed more than one year earlier are unlikely to qualify or receive direct benefit from this registry.

Why it matters

Potential benefit: If successful, the registry could improve diagnostic accuracy, reveal actionable molecular targets, and accelerate development of new treatments for very rare pediatric and AYA solid tumors.

How similar studies have performed: Other rare‑cancer registries and genomic initiatives have successfully improved understanding and led to targeted therapies for some rare tumors, although many pediatric very‑rare tumor types remain understudied.

Eligibility criteria

* INCLUSION CRITERIA:
* 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.

EXCLUSION CRITERIA:

* 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.

Where this trial is running

Bethesda, Maryland

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)

Study contacts

• Principal investigator: Mary F Wedekind Malone, D.O. — National Cancer Institute (NCI)
• Study coordinator: Mary F Wedekind Malone, D.O.

Conditions Very Rare Tumors; Very Rare Cancers; Other Solid Tumors; Solid Tumor; Pediatric Rare Tumors; Longitudinal Study; Registry; Patient Reported Outcomes