Very Rare TumorsNCT07489378
NCI Childhood Cancer Data Initiative (CCDI) Led Pediatric, Adolescent, and Young Adult Rare Cancer Registry for Very Rare Solid Tumors
An observational study of Very Rare Tumors and Very Rare Cancers, run by National Cancer Institute (NCI) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 month to 120 years
- Conditions the study is for
- Very Rare Tumors, Very Rare Cancers, Other Solid Tumors
- What is being tested?
- Natural history study of individuals with very rare tumors
- How many people
- 4,000 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Oct 2026to 1 Apr 2037
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 120 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This is a longitudinal observational study and registry for children, adolescents and young adults (AYAs) with very rare cancers.
Who may be able to join
- 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.
Who cannot join
- Diagnosis of any of the following at any time:
- Ewing Sarcoma
- Osteosarcoma
- Rhabdomyosarcoma
- Diffuse midline glioma (H3K27 altered)
- Atypical teratoid rhabdoid tumor
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
National Cancer Institute Referral Office, 888-624-1937, ncimo_referrals@mail.nih.gov
About this study
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.
Read more
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.
Read the 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
- Being tested
- Natural history study of individuals with very rare tumors
- Keywords
- Longitudinal Study, Registry, Patient Reported Outcomes, Family History, Molecular Characterization
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Natural history study of individuals with very rare tumorsOther
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.
| Study group | Role | What they receive |
|---|---|---|
| 1/ Cohort 1 | Participants with very rare tumors Receives: Other: Natural history study of individuals with very rare tumors |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Through 5 years after enrollment
Secondary outcomes (2)
To evaluate the feasibility of PRO using validated reporting platforms suitable for pediatric and AYA populations
The percentage of participants who successfully complete longitudinal data collection will be described.
Measured: At time of enrollment/study entry, and 2 and 5 years after enrollment
To conduct comprehensive clinical molecular characterization, utilizing CCDI MCI
The percentage of participants who successfully provide required sample(s) and where correlative analyses are able to be performed will be described.
Measured: At time of enrollment/study entry
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2026
- Study start28 Oct 2026 (estimated)
- Primary completion1 Apr 2035 (estimated)
- Study completion1 Apr 2037 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Very Rare TumorsStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 4,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Mary F Wedekind Malone, D.O., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07489378
- Sponsor's protocol ID
- 10002496
- Other IDs
- 002496-C
- Status last verified
- 3 Jun 2026
- First submitted
- 21 Mar 2026
- Last updated
- 23 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: This study will comply with the NIH Data Management and Sharing (DMS) Policy as waived or approved by the Center for Cancer Research, which applies to all new and ongoing NIH-funded research in the IRP, as of January 25, 2023, that is associated with a ZIA, with a clinical protocol that undergoes scientific review and/or will involve genomic data sharing.