RecruitingNot Applicable

Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors

United States10,000 participantsStarted 2019-01-28

Plain-language summary

Background: Approximately 150 cases of cancer per one million per year are considered rare cancers. While all tumors originate from genetic changes, a small percentage of these tumors are familial. Researchers want to study these changes in biological samples from people with rare tumors in order to learn more about how these tumors develop. The information obtained from this study may lead to improved screening, preventive guidelines, and treatments. Objective: To better understand rare cancers and hereditary cancer syndromes. Eligibility: People who have a rare tumor, a family history of a rare tumor, a hereditary cancer syndrome, or a mutation that leads to rare tumors. Design: Participants will be screened with questions about their medical history and/or that of their family members. They will give a saliva sample. Participants who have a tumor will have their medical records and tests reviewed. They will answer questions about their wellbeing and needs. They may provide a tumor tissue sample. Participants may also have: * Physical exam * Clinical photography * Blood, urine, saliva, and stool samples taken * Consultation with specialists * A scan that produces a picture of the body. Either one that uses a small amount of radiation, or one that uses a magnetic field. * Genetic testing/genetic counseling. Participants will be contacted once a year. They will answer updated questions about their medical and family history. Participants will be asked to contact the study team if there are changes in their tumors. Participants may be invited to join focus groups for people with the same diagnosis of rare tumors. Participants may be invited to participate in other NIH protocols. \*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\* \*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\* RARE TUMOR LIST: 1. Acinar cell carcinoma of the pancreas 2. Adamantinoma 3. Adenosqaumous carcinoma of the pancreas 4. Adrenocortical carcinoma 5. Alveolar soft part sarcoma 6. Anaplastic Thyroid Cancer 7. Angiosarcoma 8. Atypical Teratoid Rhabdoid Tumor/MRT 9. Carcinoid 10. Carcinoma of Unknown Primary 11. Chondrosarcoma 12. Chondromyxoid fibroma 13. Chordoma 14. Clear cell renal carcinoma 15. Clear Cell Sarcoma 16. Clear cell sarcoma of kidney 17. Conventional chordoma 18. Dedifferentiated chordoma 19. Desmoid 20. Desmoplastic small round cell tumor 21. Epithelioid hemangioendothelioma 22. Esthenioneuroblastoma 23. Ewing Sarcoma 24. Fibrolamellar carcinoma 25. Fusion negative rhabdomyosarcoma 26. Fusion positive renal cell carcinoma 27. Fusion positive rhabdomyosarcoma 28. Gastro-enteropancreatic neuroendocrine tumor 29. Hepatoblastoma 30. Hereditary Diffuse Gastric Cancer 31. Inflammatory myofibroblastic tumor 32. Kaposiform hemangioendothelioma 33. Malignant ectomesenchymal tumor 34. Malignant peripheral nerve sheath tumor 35. Malignant triton tumor 36. Medullary thyroid cancer 37. Mixed acinar adenocarcinoma 38. Mixed acinar neuroendocrine carcinoma 39. Myxoid Liposarcoma 40. Neuroblastoma 41. Neuroendocrine tumors 42. NUT midline carcinoma 43. Osteosarcoma 44. Pancreas ductal adenocarcinoma with squamous features 45. Pancreatic acinar cell carcinoma 46. Papillary renal cell carcinoma 47. Paraganglioma 48. Parosteal Osteosarcoma 49. Periosteal Osteosarcoma 50. Peripheral nerve sheath tumor 51. Peripheral primitive neuroectodermal tumor 52. Pheochromocytoma 53. Pituitary cancer 54. Poorly differentiated chordoma 55. Renal medullary carcinoma 56. Rhabdomyosarcoma 57. Round cell Liposarcoma 58. Schwannoma 59. Sclerosing Epithelioid Fibrosarcoma 60. SDH deficient GIST 61. SMARCB1 deficient tumors 62. SMARCA4 deficient tumors 63. Synovial sarcoma 64. Undifferentiated Sarcoma \*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\* \*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*\*

Who can participate

Age range

4 Weeks

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

Cohort 2: Participants without a rare tumor who have a germline genetic variant that predisposes to a rare solid tumor
Cohort 3: Relatives of participants with diagnosis of rare solid tumors who do NOT have a known germline variant that predisposes to a rare solid tumor

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Since this is a natural history study and not a treatment trial, would joining it mean my care team would still be free to pursue standard treatments or other clinical trials at the same time?
2This study collects biospecimens and tracks disease over time — what kinds of samples would be taken from me or my child, how often, and would any of these collections require extra procedures beyond my normal care visits?
3The study focuses on rare solid tumors and tumor predisposition syndromes — does my specific diagnosis qualify as 'rare' enough that this kind of longitudinal tracking could meaningfully help doctors understand my disease course better than existing data already does?
4Because this is a natural history study rather than a phase 1, 2, or 3 treatment trial, it isn't testing a new drug or therapy — so what would I or my child actually gain from participating, compared to receiving standard care without enrolling?
5If my tumor changes, progresses, or goes into remission during the study, would I still be expected to continue participating, and are there any circumstances under which I might need to leave the study or would be asked to leave?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

To comprehensively and longitudinally evaluate the natural history of patients with rare solid tumors or tumor predisposition syndromes, estimating and defining their clinical spectrum (e.g. disease course and survival)

Timeframe: 10 years

Trial details

NCT IDNCT03739827

SponsorNational Cancer Institute (NCI)

Sponsor typeNIH

Study typeOBSERVATIONAL

Primary completion2028-05-31

Contact for this trial

Barbara J Thomas, R.N.

(240) 858-3633 barbara.thomas2@nih.gov

View on ClinicalTrials.gov More Malignant Solid Tumors trials