NCI:n Lasten syöpätietoaloite (CCDI) -ohjaama Lasten, nuorten ja nuorten aikuisten harvinaisten syöpien rekisteri hyvin harvinaisille kiinteille kasvaimille
NCI:n Lasten Syöpätietoaloite (CCDI) -johtama Lapsille, Nuorille ja Nuorille Aikuisille Tarkoitettu Erittäin Harvinaisten Kiinteiden Kasvainten Syöpärekisteri
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.
Tutkimuksen yleiskatsaus
Tila
Tila
Ehdot
Ehdot
Interventio / Hoito
Interventio / Hoito
Yksityiskohtainen kuvaus
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.
Opintotyyppi
Opintotyyppi
Ilmoittautuminen (Arvioitu)
Ilmoittautuminen
Yhteystiedot ja paikat
Opiskeluyhteys
Opiskeluyhteys
- Nimi: Mary F Wedekind Malone, D.O.
- Puhelinnumero: (240) 858-3765
- Sähköposti: maryfrances.wedekindmalone@nih.gov
Opiskelupaikat
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Maryland
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Bethesda, Maryland, Yhdysvallat, 20892
- Rekrytointi
- National Institutes of Health Clinical Center
-
Ottaa yhteyttä:
- National Cancer Institute Referral Office
- Puhelinnumero: 888-624-1937
- Sähköposti: ncimo_referrals@mail.nih.gov
-
-
Osallistumiskriteerit
Kelpoisuusvaatimukset
Kelpoisuusvaatimukset
Opintokelpoiset iät
Hyväksyy terveitä vapaaehtoisia
Sukupuolet, jotka voivat opiskella
Näytteenottomenetelmä
Tutkimusväestö
This is a longitudinal observational study and registry for children, adolescents and young adults (AYAs) with very rare cancers.
Kuvaus
- 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.
Opintosuunnitelma
Miten tutkimus on suunniteltu?
Suunnittelun yksityiskohdat
- Havaintomallit: CASE_ONLY
- Aikanäkymät: PROSPECTIVE
Ryhmien/kohorttien lukumäärä
Kohortit ja interventiot
Ryhmä/KohorttiRyhmä/Kohortti |
Interventio / HoitoInterventio / Hoito |
|---|---|
|
1/ Cohort 1
Participants 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. |
Mitä tutkimuksessa mitataan?
Ensisijaiset tulostoimenpiteet
Ensisijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
|---|---|---|
|
To establish a longitudinal observational study and registry for very rare pediatric and AYA solid tumors
Aikaikkuna: Through 5 years after enrollment
|
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. |
Through 5 years after enrollment
|
Toissijaiset tulostoimenpiteet
Toissijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
|---|---|---|
|
To evaluate the feasibility of PRO using validated reporting platforms suitable for pediatric and AYA populations
Aikaikkuna: At time of enrollment/study entry, and 2 and 5 years after enrollment
|
The percentage of participants who successfully complete longitudinal data collection will be described. |
At time of enrollment/study entry, and 2 and 5 years after enrollment
|
|
To conduct comprehensive clinical molecular characterization, utilizing CCDI MCI
Aikaikkuna: At time of enrollment/study entry
|
The percentage of participants who successfully provide required sample(s) and where correlative analyses are able to be performed will be described. |
At time of enrollment/study entry
|
Yhteistyökumppanit ja tutkijat
Sponsori
Sponsori
Tutkijat
Tutkijat
- Päätutkija: Mary F Wedekind Malone, D.O., National Cancer Institute (NCI)
Julkaisuja ja hyödyllisiä linkkejä
Hyödyllisiä linkkejä
Opintojen ennätyspäivät
Opi tärkeimmät päivämäärät
Opiskelun aloitus (Arvioitu)
Opiskelun aloitus
Ensisijainen valmistuminen (Arvioitu)
Ensisijainen valmistuminen
Opintojen valmistuminen (Arvioitu)
Opintojen valmistuminen
Opintoihin ilmoittautumispäivät
Ensimmäinen lähetetty
Ensimmäinen lähetetty
Ensimmäinen toimitettu, joka täytti QC-kriteerit
Ensimmäinen toimitettu, joka täytti QC-kriteerit
Ensimmäinen Lähetetty (Todellinen)
Ensimmäinen Lähetetty
Tutkimustietojen päivitykset
Viimeisin päivitys julkaistu (Todellinen)
Viimeisin päivitys julkaistu
Viimeisin lähetetty päivitys, joka täytti QC-kriteerit
Viimeisin lähetetty päivitys, joka täytti QC-kriteerit
Viimeksi vahvistettu
Viimeksi vahvistettu
Lisää tietoa
Tähän tutkimukseen liittyvät termit
Avainsanat
Muut tutkimustunnusnumerot
Muut tutkimustunnusnumerot
- 10002496
- 002496-C
Yksittäisten osallistujien tietojen suunnitelma (IPD)
Aiotko jakaa yksittäisten osallistujien tietoja (IPD)?
IPD-suunnitelman kuvaus
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.
IPD-jaon aikakehys
Data will be made available as soon as possible or at the time of associated publication. Data not published in a manuscript will be shared via public source once the data set completes QC.
IPD-jaon käyttöoikeuskriteerit
Clinical data will be made available upon request and with the permission of the study PI. Genomic data are made available via dbGAP through requests to the data custodians.
IPD-jakamista tukeva tietotyyppi
- STUDY_PROTOCOL
- MAHLA
- ICF
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