NCT07489378

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 4,000 (estimated)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 1 Month – 120 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Background: * Rare cancers are defined by the NCI as fewer than 15 incident cases per 100,000 people per year. Overall, pediatric tumors account for less than 1% of all cancers diagnosed in the United States (US) per year. * The European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT) group has determined that the definition of very rare pediatric cancers is fewer than 2 cases per million per year and/or those not eligible for clinical trials. * Frequently, for these very rare solid tumors and hematologic malignancies, little is known about their natural history, including clinical behavior, molecular/genetic characteristics, optimal management, drug response, and evidence-based standard therapy does not exist. Many patients endure the challenges of achieving an accurate and timely diagnosis, along with the difficulty of identifying a center with treatment expertise. * Although several disease-specific pediatric and adult rare cancer efforts exist, they are frequently siloed without standardization across efforts; and, many rare cancers have not been systematically studied. Objective: -To establish a longitudinal observational study and registry for very rare pediatric and Adolescents and Young Adults (AYA) solid tumors Eligibility: -Pediatric, adolescent, or young adult participants (\<= 39 years old at diagnosis) with very rare solid tumors Design: * This will be a longitudinal observational study and registry for children and AYAs with very rare cancers. * Recruitment will be through 2 mechanisms, the Children s Oncology Group s (COG) Project EveryChild Protocol (APEC14B1, NCT02402244) and physician/self-referral. * This study will be led by the NCI s CCDI and NCI s POB. * Enrollment, including consent, distribution of family history/individual medical history/patient report outcome questionnaires, collection of medical records and tissue/saliva/buccal samples, and extraction of data, as well as longitudinal follow-up will be managed centrally. * Tumor tissue and saliva/buccal samples will be run through the CCDI s Molecular Characterization Initiative (MCI) for comprehensive clinical molecular profiling. The remaining samples will be banked for research analyses. Research analysis will be per the CCDI research characterization pipeline. * Since long-term follow-up of individuals with very rare cancers is a major feature of the study, we intend to maintain active contact with study participants for up to 5 years. * Data will be deposited into the CCDI Data Ecosystem.

Conditions

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

Interventions

Type	Name	Description
OTHER	Natural history study of individuals with very rare tumors	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.

Timeline

• Start date: 2026-04-22
• Primary completion: 2035-04-01
• Completion: 2037-04-01
• First posted: 2026-03-24
• Last updated: 2026-04-17

Locations

1 site across 1 country: United States