Natural History and Tissue Acquisition Study of Adrenocortical Carcinoma

December 11, 2019 updated by: National Cancer Institute (NCI)

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.

Study Overview

Status

Completed

Conditions

Detailed Description

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. 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.

Study Type

Observational

Enrollment (Actual)

67

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • National Institutes of Health Clinical Center, 9000 Rockville Pike

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 100 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

  • INCLUSION CRITERIA:
  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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.
Time Frame: 10 years
10 years

Secondary Outcome Measures

Outcome Measure
Time Frame
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.
Time Frame: 10 years
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
Time Frame: 10 years
10 years
Correlate clinical evolution with genetic alterations, DNA methylation patterns and gene expression profiles identified within tumors.
Time Frame: 10 years
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
Time Frame: 10 years
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Cancer Institute (NCI)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 13, 2013

Primary Completion (Actual)

October 16, 2015

Study Completion (Actual)

March 2, 2018

Study Registration Dates

First Submitted

December 13, 2013

First Submitted That Met QC Criteria

December 13, 2013

First Posted (Estimate)

December 19, 2013

Study Record Updates

Last Update Posted (Actual)

December 12, 2019

Last Update Submitted That Met QC Criteria

December 11, 2019

Last Verified

March 2, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 140029
  • 14-C-0029

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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