Clinical Trial: Natural History and Tissue Acquisition Study of Adrenocortical Carcinoma

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Natural History and Tissue Acquisition Study of Adrenocortical Carcinoma

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.

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
Sponsor: National Cancer Institute (NCI)

Current Primary Outcome: 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 ]

Original Primary Outcome: Characterize the natural history of adrenocortical cancer (ACC) and in the process, collect blood and tissue samples to study the biology involved in the development and progression of ACC. [ Time Frame: 10 years ]

Current Secondary Outcome:

  • 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 ]
  • 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 ]
  • Correlate clinical evolution with genetic alterations, DNA methylation patterns and gene expression profiles identified within tumors. [ Time Frame: 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 ]


Original Secondary Outcome:

Information By: National Institutes of Health Clinical Center (CC)

Dates:
Date Received: December 13, 2013
Date Started: December 3, 2013
Date Completion:
Last Updated: April 21, 2017
Last Verified: September 27, 2016