Natural History and Tissue Acquisition Study of Adrenocortical Carcinoma

NCT02015026 | Completed

• 2 other identifiers: 14002914-C-0029
• Study Type: observational
• Target: 67
• Locations: 1 country
• Sites: 1

Brief Summary

Background:

• Adrenocortical carcinoma (ACC) is a rare tumor with an incidence of 1.5 to 2 per million people per year. It has a very poor prognosis with an overall 5-year mortality rate of 75 - 90% and an average survival from the time of diagnosis of 14.5 months.
• The treatment of choice for a localized primary or recurrent tumor is surgical resection. Patients with recurrent or metastatic disease are infrequently curable by surgery alone.
• As with most solid tumors, chemotherapy options have limited benefit, although platinumbased therapies have response rates of 25 to 30%. To date no targeted therapy has been shown to be of any value in this disease.
• The natural history of ACC can vary greatly with some patients surviving only months while others can live with disease for years. The basis for these differing clinical presentations is not known. While one cannot exclude an immune or other host component as responsible for the diverse clinical courses, it is also possible that there may be a genetic basis for this phenomenon. A bio-specimen repository will be a major step towards more comprehensive studies of this very rare and unusual tumor, and allow us to begin to characterize subgroups within the disease.
• Patients with rare tumors seek expert advice in the management of their care. Dr. Fojo has such expertise and is frequently asked to consult in the care of ACC patients throughout the world. A natural history study would establish a more formal mechanism for such referrals, while allowing the systematic collection of epidemiologic data as well as much needed tumor samples. Objective:
• To characterize the natural history of adrenocortical cancer, and in the process, collect blood, and tissue samples to study genetic/biochemical pathways involved in the development and progression of adrenocortical cancer (ACC). Eligibility:
• Patients greater than or equal to 12 years of age with biopsy-proven ACC
• Patients greater than or equal to 12 years of age suspected of having ACC Design/Schema:
• Patients will be offered clinical consultation with treatment recommendations, including standard of care and clinical trial options. Computed tomography scans of the thorax, abdomen and pelvis will be performed for staging purposes as indicated; occasionally, magnetic resonance imaging will be performed for the visualization of lesions in the liver, spine, or other anatomic sites.
• Medical histories will be documented and patients followed throughout the course of their illnesses, with particular attention to patterns of disease recurrence and progression, response to therapies, duration of responses and hormone production in patients with hormone production as a manifestation of their disease. Tumor growth rates will also be calculated throughout the course of the disease.
• Blood and tumor samples will be obtained at baseline and at follow-up intervals when surgery is indicated. Tumor samples may include samples harvested at other facilities during or prior to enrollment on this trial.
• Genetic and epigenetic analysis of tumors and in selected cases expression array analysis will be performed.

Conditions

Adrenocortical Carcinoma

Keywords

Biospecimen Repository; Disease Progression Pattern; Specimen Analysis; Genetic Analysis

Outcome Measures

Primary Outcomes (1)

• Characterize the natural history of adrenocortical cancer (ACC). Data will include clinical presentation, patterns of disease progression, response or lack of response to therapeutic interventions, disease recurrence and overall survival.

  10 years

Secondary Outcomes (4)

• Establish a bio-specimen repository for genetic and epigenetic analysis of tumors and in selected cases for expression array analysis by collecting blood, and tissue samples to study the biology of ACC development and progression.

  10 years

• Estimate the growth and regression rate constants of tumor treated with standard of care or experimental therapies and correlate with genetic, epigenetic and gene expression profiles

  10 years

• Correlate clinical evolution with genetic alterations, DNA methylation patterns and gene expression profiles identified within tumors.

  10 years

• Correlate patterns of disease recurrence and impact on progression-free survival and overall survival following a laparoscopic resection or open laparotomy for ACC

  10 years

Eligibility Criteria

• Age: 12 Years - 100 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients greater than or equal to 12 years of age with biopsy-proven ACC that has been confirmed by the Laboratory of Pathology, NCI.
• Patients greater than or equal to 12 years of age suspected of having ACC. Patients with suspected ACC will undergo baseline workup at the clinical center and be referred for definitive surgical diagnosis and treatment.
• Ability of subject or Legally Authorized Representative to understand and the willingness to sign a written informed consent document.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Terzolo M, Berruti A. Adjunctive treatment of adrenocortical carcinoma. Curr Opin Endocrinol Diabetes Obes. 2008 Jun;15(3):221-6. doi: 10.1097/MED.0b013e3282fdf4c0.

  PMID: 18438168 BACKGROUND

• Fassnacht M, Terzolo M, Allolio B, Baudin E, Haak H, Berruti A, Welin S, Schade-Brittinger C, Lacroix A, Jarzab B, Sorbye H, Torpy DJ, Stepan V, Schteingart DE, Arlt W, Kroiss M, Leboulleux S, Sperone P, Sundin A, Hermsen I, Hahner S, Willenberg HS, Tabarin A, Quinkler M, de la Fouchardiere C, Schlumberger M, Mantero F, Weismann D, Beuschlein F, Gelderblom H, Wilmink H, Sender M, Edgerly M, Kenn W, Fojo T, Muller HH, Skogseid B; FIRM-ACT Study Group. Combination chemotherapy in advanced adrenocortical carcinoma. N Engl J Med. 2012 Jun 7;366(23):2189-97. doi: 10.1056/NEJMoa1200966. Epub 2012 May 2.

  PMID: 22551107 BACKGROUND

• Stein WD, Yang J, Bates SE, Fojo T. Bevacizumab reduces the growth rate constants of renal carcinomas: a novel algorithm suggests early discontinuation of bevacizumab resulted in a lack of survival advantage. Oncologist. 2008 Oct;13(10):1055-62. doi: 10.1634/theoncologist.2008-0016. Epub 2008 Sep 30.

  PMID: 18827177 BACKGROUND

MeSH Terms

Conditions

Adrenocortical Carcinoma

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Adrenal Cortex Neoplasms; Adrenal Gland Neoplasms; Endocrine Gland Neoplasms; Neoplasms by Site; Adrenal Cortex Diseases; Adrenal Gland Diseases; Endocrine System Diseases

Study Officials

• Jeremy L Davis, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 13, 2013
• First Posted: December 19, 2013
• Study Start: December 13, 2013
• Primary Completion: October 16, 2015
• Study Completion: March 2, 2018
• Last Updated: December 12, 2019

Record last verified: 2018-03-02

Locations

US (1)