NCI Childhood Cancer Data Initiative (CCDI) Led Pediatric, Adolescent, and Young Adult Rare Cancer Registry for Very Rare Solid Tumors
Purpose
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
- Very Rare Tumors
- Very Rare Cancers
- Other Solid Tumors
- Solid Tumor
- Pediatric Rare Tumors
Eligibility
- Eligible Ages
- Between 1 Month and 120 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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.
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Case-Only
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| 1/ Cohort 1 | Participants with very rare tumors |
|
Recruiting Locations
Bethesda, Maryland 20892
More Details
- Status
- Recruiting
- Sponsor
- National Cancer Institute (NCI)
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.