Pediatric CancerCCPSNCT04511806
Childhood Cancer Predisposition Study (CCPS)
An observational study of Pediatric Cancer, run by Emory University at 7 sites in 2 countries.
Trial summary
The Childhood Cancer Predisposition Study (CCPS) is a multi-center, longitudinal, observational study that will collect clinical and biological data and specimens from children with a cancer predisposition syndromes (CPS) and their relatives. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Pediatric Cancer
- What is being tested?
- Registry
- How many people
- 1,050 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 22 Apr 2021to Oct 2030
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The Primary Subjects of this study are children (age 0-21) with a CPS. Members of their Primary Family Unit will also be recruited for this study, including CPS-Affected Parents, Unaffected Parents and Siblings. Other adult family members (with documented or obligate CPS) are also eligible to enroll as Affected Family Members.
Who may be able to join
- Primary Subjects must meet all of the below criteria to be eligible for enrollment:
- Be less than 21 years of age at the time of enrollment
- Have a diagnosis of a specific CPS, whether they have had cancer or not
- Based on clinical laboratory testing demonstrating a Pathogenic or Likely Pathogenic germline variant and/or
- Based on well-established clinical diagnostic criteria and/or
- Based on high clinical suspicion of a specific CPS with clinical laboratory testing demonstrating a variant of uncertain significance (VUS)
Show all 18Show fewer
- Affected Parents must meet all of the following criteria to be eligible for enrollment:
- Be the biologic parent of a Primary Subject and
- Carry a diagnosis (or obligate diagnosis) of the familial CPS
- Adult Affected Siblings must meet all of the following criteria to be eligible for enrollment:
- Be the biologic sibling of a Primary Subject and
- Carry a diagnosis (or obligate diagnosis) of the familial CPS
- Unaffected Parents and Siblings must meet all of the following criteria to be eligible for enrollment
- Be the biologic parent or sibling of a Primary Subject and
- Not carry a diagnosis (or obligate diagnosis) of the familial CPS
- Affected Family Members must meet all of the following criteria to be eligible for enrollment:
- 1. Carry a diagnosis of (or obligate diagnosis of) the familial CPS. Documentation is requested but not required.
- More than one child from a Primary Family Unit may be a Primary Subject. An Unaffected Sibling may be reclassified as a Primary Subject if diagnosed with a CPS during childhood.
Who cannot join
- Individuals with a strong personal or family history of cancer without a genetic or clinical diagnosis of a specific CPS are not eligible for enrollment.
Trial locations and contacts
7 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 2 countries
United States6 sites, 6 recruiting
- Recruiting
Children's Healthcare of Atlanta (CHOA)
Atlanta, Georgia, 30322
Christopher Porter, MD, 404-727-4881, chris.porter@emory.edu
- Recruiting
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Lisa Diller, MD, lisa_diller@dfci.harvard.edu
- Recruiting
Baylor College of Medicine
Houston, Texas, 77030
Surya Rednam, MD, sprednam@texaschildrens.org
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105-3678
Kim Nichols, MD, kim.nichols@stjude.org
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Garrett Brodeur, MD, brodeur@email.chop.edu
- Recruiting
Primary Children's Hospital
Salt Lake City, Utah, 84113
Joshua Schiffman, MD, joshua.schiffman@hci.utah.edu
Canada1 site, 1 recruiting
- Recruiting
Hospital for Sick Children
Toronto, Ontario
Anita Villani, MD, anita.villani@sickkids.ca
About this study
The Childhood Cancer Predisposition Study (CCPS) is a multi-center, longitudinal, observational study that will collect clinical and biological data and specimens from children with a cancer predisposition syndromes (CPS) and their relatives.
The central hypothesis is that studying individuals at high risk for childhood cancer creates a unique opportunity for improving the understanding of carcinogenesis, tumor surveillance, early detection, and cancer prevention, which will collectively contribute to improving care and outcomes for pediatric patients with cancer and those with cancer predisposition syndromes (CPS).
Read the detailed description
The CCPS is designed as a multi-center, longitudinal, observational study that will collect clinical and biological data and specimens from children with a CPS and their relatives. The investigators plan to:
- Establish and maintain a framework for recruitment, participation, and surveillance of children with cancer predisposition syndromes (CPS) in clinical and translational research studies;
- Define the natural history of disease in children with CPS; and
- Evaluate the clinical impact and effectiveness of standard and emerging tumor surveillance strategies.
The study will enroll approximately 350 Children and 700 Relatives per year. The investigators plan to collect demographic and diagnostic data at enrollment. Longitudinal follow-up will be performed at least annually.
The CCPS includes the establishment of a biorepository, with a hub and spoke structure, with a central repository at Emory University/Children's Healthcare of Atlanta for prospective collection of some tissues, linked to local biorepositories at participating institutions. Information about inventory of tumor specimens already banked locally will be available in the database, along with reference to existing genomic studies of the tumor, such that investigators may identify and request such tissue or data for specific studies, subject to approval of the CCPS Scientific Committee.
- Conditions
- Pediatric Cancer
- Being tested
- Registry
- Keywords
- Cancer predisposition
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RegistryOther
This prospective registry and biorepository will collect clinical data and specimens for research in childhood cancer predisposition.
| Study group | Role | What they receive |
|---|---|---|
| Primary Subjects | Children (age 0-21) with a cancer predisposition syndromes (CPS). Receives: Other: Registry | |
| Relatives of Children with CPS | Members of their Primary Family Unit will also be recruited for this study, including CPS-Affected Parents, Unaffected Parents and Siblings. Other adult family members (with documented or obligate CPS) are also eligible to enroll as Affected Family Members. Receives: Other: Registry |
What is being measured
The primary outcome is the main question the study is designed to answer.
Establish and maintain a framework for recruitment, participation, and surveillance of children with cancer predisposition syndromes (CPS) in clinical and translational research studies.
This multicenter registry and biorepository will be developed with the purpose of studying individuals at high risk for childhood cancer to improve care and outcomes for pediatric patients with cancer and those with cancer predisposition syndromes (CPS).
Measured: Up to 10 years
Define the natural history of disease in children with CPS.
To define the natural history of disease in children with CPS.
Measured: Up to 10 years
Evaluate the clinical impact and effectiveness of standard and emerging tumor surveillance strategies.
Evaluate the clinical impact and effectiveness of standard and emerging tumor surveillance strategies to improve care and outcomes for pediatric patients with cancer and those with cancer predisposition syndromes (CPS).
Measured: Up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2020
- Study start22 Apr 2021
- Primary completionOct 2030 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pediatric CancerStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,050 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Christopher Porter, Emory University
- Investigators
- Christopher Porter, MD, Principal investigator, Emory UniversityAnita Villani, MD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04511806
- Sponsor's protocol ID
- STUDY00000109
- Other IDs
- C3P-001
- Acronym
- CCPS
- Status last verified
- Oct 2025
- First submitted
- 11 Aug 2020
- Last updated
- 29 Oct 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified, individual participant data reported in publications
Publications
Perrino MR, MacFarland SP, Maese L, Vagher J, Kamihara J, Rednam S, Lupo PJ, Brodeur GM, Schiffman J, Diller L, Desrosiers-Battu L, Plon SE, Nichols KE, Volchenboum SL, Malkin D, Villani A, Porter CC. Childhood cancer predisposition study: a prospective registry and biorepository protocol. BMJ Open. 2026 Aug 5;16(8):e120576. doi: 10.1136/bmjopen-2026-120576.
PubMed 42556843
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Childhood Cancer Predisposition Study (CCPS)
- ClinicalTrials.gov ID
- NCT04511806
- Acronym / sponsor ID
- CCPS / STUDY00000109
- Conditions
- Pediatric Cancer
- Keywords
- Cancer predisposition
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,050 participants (estimated)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- 22 Apr 2021 (actual)
- Primary completion
- Oct 2030 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 13 Aug 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The Childhood Cancer Predisposition Study (CCPS) is a multi-center, longitudinal, observational study that will collect clinical and biological data and specimens from children with a cancer predisposition syndromes (CPS) and their relatives.
The central hypothesis is that studying individuals at high risk for childhood cancer creates a unique opportunity for improving the understanding of carcinogenesis, tumor surveillance, early detection, and cancer prevention, which will collectively contribute to improving care and outcomes for pediatric patients with cancer and those with cancer predisposition syndromes (CPS).
Detailed description
The CCPS is designed as a multi-center, longitudinal, observational study that will collect clinical and biological data and specimens from children with a CPS and their relatives. The investigators plan to:
- Establish and maintain a framework for recruitment, participation, and surveillance of children with cancer predisposition syndromes (CPS) in clinical and translational research studies;
- Define the natural history of disease in children with CPS; and
- Evaluate the clinical impact and effectiveness of standard and emerging tumor surveillance strategies.
The study will enroll approximately 350 Children and 700 Relatives per year. The investigators plan to collect demographic and diagnostic data at enrollment. Longitudinal follow-up will be performed at least annually.
The CCPS includes the establishment of a biorepository, with a hub and spoke structure, with a central repository at Emory University/Children's Healthcare of Atlanta for prospective collection of some tissues, linked to local biorepositories at participating institutions. Information about inventory of tumor specimens already banked locally will be available in the database, along with reference to existing genomic studies of the tumor, such that investigators may identify and request such tissue or data for specific studies, subject to approval of the CCPS Scientific Committee.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Primary Subjects | – | Other: RegistryChildren (age 0-21) with a cancer predisposition syndromes (CPS). |
| Relatives of Children with CPS | – | Other: RegistryMembers of their Primary Family Unit will also be recruited for this study, including CPS-Affected Parents, Unaffected Parents and Siblings. Other adult family members (with documented or obligate CPS) are also eligible to enroll as Affected Family Members. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The Primary Subjects of this study are children (age 0-21) with a CPS. Members of their Primary Family Unit will also be recruited for this study, including CPS-Affected Parents, Unaffected Parents and Siblings. Other adult family members (with documented or obligate CPS) are also eligible to enroll as Affected Family Members.
Key inclusion criteria (18 listed)
- Be less than 21 years of age at the time of enrollment
- Have a diagnosis of a specific CPS, whether they have had cancer or not
- Be the biologic parent of a Primary Subject and
- Carry a diagnosis (or obligate diagnosis) of the familial CPS
- Adult Affected Siblings must meet all of the following criteria to be eligible for enrollment:
Key exclusion criteria (1 listed)
- Individuals with a strong personal or family history of cancer without a genetic or clinical diagnosis of a specific CPS are not eligible for enrollment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RegistryThis prospective registry and biorepository will collect clinical data and specimens for research in childhood cancer predisposition. | Other | Primary Subjects, Relatives of Children with CPS |
Primary outcome
Establish and maintain a framework for recruitment, participation, and surveillance of children with cancer predisposition syndromes (CPS) in clinical and translational research studies.
Time frame: Up to 10 years
This multicenter registry and biorepository will be developed with the purpose of studying individuals at high risk for childhood cancer to improve care and outcomes for pediatric patients with cancer and those with cancer predisposition syndromes (CPS).
Define the natural history of disease in children with CPS.
Time frame: Up to 10 years
To define the natural history of disease in children with CPS.
Evaluate the clinical impact and effectiveness of standard and emerging tumor surveillance strategies.
Time frame: Up to 10 years
Evaluate the clinical impact and effectiveness of standard and emerging tumor surveillance strategies to improve care and outcomes for pediatric patients with cancer and those with cancer predisposition syndromes (CPS).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2025.
- Derived from this study: PubMed 42556843Perrino MR, MacFarland SP, Maese L, Vagher J, Kamihara J, Rednam S, Lupo PJ, Brodeur GM, Schiffman J, Diller L, Desrosiers-Battu L, Plon SE, Nichols KE, Volchenboum SL, Malkin D, Villani A, Porter CC. Childhood cancer predisposition study: a prospective registry and biorepository protocol. BMJ Open. 2026 Aug 5;16(8):e120576. doi: 10.1136/bmjopen-2026-120576.
- Individual participant dataSharing planned: Yes. De-identified, individual participant data reported in publications
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Christopher Porter, MD principal investigator
Emory University
Anita Villani, MD principal investigator
The Hospital for Sick Children
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pediatric Cancer, run by Emory University at 7 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Christopher Porter, MD (4047274881, chris.porter@emory.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Establish and maintain a framework for recruitment, participation, and surveillance of children with cancer predisposition syndromes (CPS) in clinical and translational research studies. (time frame: Up to 10 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04511806, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2025.