International Pediatric Adrenocortical Tumor Registry

Study Description

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Brief Summary:

This study aims to collect demographic and medical information including detailed family history of cancer of children and adolescents with adrenocortical tumors in order to learn more about the clinical and epidemiological aspects, treatment modalities, and outcome of patients with this rare disease, worldwide.

In addition, investigators at St. Jude Children's Research Hospital (SJCRH) plan to perform molecular studies of tumor cells aimed to clarify the role of the TP53 gene and other genetic pathways in these tumors. They aim to obtain relevant biological material from participants with adrenocortical tumor (ACT), their biological parents, and relatives for determination of the TP53 germline status, molecular studies of the TP53 gene, and other molecular pathways.

Condition or disease
Adrenocortical Tumor

Detailed Description:

Adrenocortical tumors (ACT) are rare cancer types that form in the outer layer of the adrenal gland and are very uncommon in children and teenagers. There is variation in pediatric ACT incidence worldwide. In the United States, only about 25 new cases of ACT per million per year, making this a very rare tumor. However, in southern Brazil, the annual incidence of ACT is 15 times that seen in the United States accounting for 3.4-4.2 per million per year.

Molecular studies have revealed that the majority of children with ACT, particularly those younger than 4 years of age, have constitutional TP53 mutations and/or imprinting defects at chromosome 11p as observed in Beckwith Wiedemann syndrome (BWS) patients. Some mutations, as exemplified by the R337H TP53 germline mutation, in which the function of the mutant protein is relatively preserved, the history of cancer in the carriers and their families is relatively unremarkable. In other cases, the TP53 mutated gene encodes a functionally-impaired protein that predicts for a pervasive history of familial cancer (Li-Fraumeni syndrome). Therefore, these observations have implications for genetic counseling of families with childhood ACT and underscore the importance of genotype-phenotype correlations in familial cancer syndromes.

The creation of a rare tumor registry provides a mechanism to collect information that cannot be gathered in a single institution. The analysis of the registry data would permit an overview of the clinical, epidemiological, current treatment standards, and survival data of these patients and thus create opportunities for research. It also may facilitate the development of treatment consensus among investigators who register their patients and help to design future studies. Moreover, the combined Children's Oncology Group (COG) and IPACTR studies are expected to provide meaningful insight into the biology of ACT, including clinical phenotype/genotype relationships, treatment outcome and long-term follow-up data in subjects with this rare tumor. Finally, it would provide data on the long-term consequences of exposure to tumor-secreted androgens (found in more than 80% of the pediatric cases) on children's growth and development.

Study Design

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Study Type :	Observational
Estimated Enrollment :	9999 participants
Observational Model:	Other
Time Perspective:	Prospective
Official Title:	International Pediatric Adrenocortical Tumor Registry
Actual Study Start Date :	October 1, 2001
Estimated Primary Completion Date :	December 2040
Estimated Study Completion Date :	December 2040

Groups and Cohorts

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Group/Cohort
Participants; Any participant who meets eligibility criteria and consents to participate in the trial.

Outcome Measures

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Primary Outcome Measures :

1. Collect demographic/medical information, detailed family history of cancer of children/adolescents with adrenocortical tumors, learn more about the clinical and epidemiological aspects, treatment modalities, and outcome of patients [ Time Frame: Annually from diagnosis until no longer being followed, defined as up to 5 years from the date of last follow-up ]

Biospecimen Retention:   Samples With DNA

Peripheral blood sample with DNA and RNA; Tumor Tissue (of any histology); Adrenocortical tumors;

Eligibility Criteria

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Ages Eligible for Study:	up to 21 Years (Child, Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Age ≤ 21 years old at diagnosis of adrenocortical tumor.

Relatives of the ACT patients of any age with a diagnosis of malignant tumor.

Criteria

STRATIFICATION ASSIGNMENT:

• Stratum A: participant suspected or confirmed diagnosis of adrenocortical tumor (ACT)
• Stratum R: relative of participant with ACT and TP53 mutation who has diagnosis of malignancy
• Stratum P: biological parent of participant with ACT

Inclusion Criteria - Stratum A (participant with ACT):

• Age ≤ 21 years old at diagnosis
• Suspected or confirmed diagnosis of adrenocortical tumor (adenoma, carcinoma or undefined histology).
• Signed informed consent

Inclusion Criteria - Stratum R (relative):

• Any age
• Diagnosis of malignant tumor
• Signed informed consent

Inclusion Criteria - Stratum P (parent):

• Biological parent of Stratum A participant

Contacts and Locations

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Contacts

Contact: Raul C Ribeiro, MD	866-278-5833	referralinfo@stjude.org

Locations

United States, California
Stanford University	Completed
Stanford, California, United States, 94305
United States, Florida
All Children's Hospital/St. Petersburg Hospital	Completed
Saint Petersburg, Florida, United States, 33701
United States, Ohio
The Children's Medical Center	Recruiting
Dayton, Ohio, United States, 45404
Contact: Mukund Dole, MD
Contact: Jenny Dillon, RN, CCRP
Principal Investigator: Mukund Dole, MD
United States, Tennessee
St. Jude Children's Research Hospital	Recruiting
Memphis, Tennessee, United States, 38105
Contact: Raul C Ribeiro, MD 866-278-5833 referralinfo@stjude.org
Principal Investigator: Raul C Ribeiro, MD
United States, Texas
Cook Children's Medical Center	Completed
Fort Worth, Texas, United States, 76104

Sponsors and Collaborators

St. Jude Children's Research Hospital

Investigators

Principal Investigator:	Raul C Ribeiro, MD	St. Jude Children's Research Hospital

More Information

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Additional Information:

St. Jude Children's Research Hospital 

Clinical Trials Open at St. Jude 

Responsible Party:	St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:	NCT00700414 History of Changes
Other Study ID Numbers:	IPACTR
First Posted:	June 18, 2008
Last Update Posted:	November 14, 2017
Last Verified:	November 2017

Studies a U.S. FDA-regulated Drug Product:		No
Studies a U.S. FDA-regulated Device Product:		No

Additional relevant MeSH terms:

Adrenal Cortex Neoplasms; Adrenal Gland Neoplasms; Endocrine Gland Neoplasms; Neoplasms by Site	Neoplasms; Adrenal Cortex Diseases; Adrenal Gland Diseases; Endocrine System Diseases