Natural History and Tissue Acquisition Study of Adrenocortical Carcinoma

ID: NCT02015026
Status: Completed
Phase: N/A
Start Date: December 13, 2013
First Submitted: December 13, 2013
Last Updated: February 22, 2018
Results: N/A
Sponsors & Collaborators: National Cancer Institute (NCI)
Location: United States
Conditions: Adrenocortical Carcinoma
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Study Description

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.

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.
Condition or disease Intervention/treatment Phase

Adrenocortical Carcinoma

N/A

Tracking Information

First Submitted DateDecember 13, 2013
Last Update Posted DateFebruary 22, 2018
Start DateDecember 13, 2013
Actual Completion DateJuly 28, 2016
Primary Completion DateOctober 16, 2015
Results First Submitted DateN/A
Received Results Disposit DateN/A

Current Primary Outcome Measures

  • 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 Measures

Not Available

Current Secondary Outcome Measures

  • 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 Measures

Not Available

Study Design

Brief TitleNatural History and Tissue Acquisition Study of Adrenocortical Carcinoma
Official TitleNatural 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.

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.

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 TypeObservational
Study PhaseN/A
Estimated Enrollment
67
Allocation
Not Available
Interventional Model
Not Available
Masking
Not Available
Primary Purpose
Not Available
Conditions
Adrenocortical Carcinoma
Target Follow-Up Duration N/A
Biospecimen:
N/A
Sampling MethodN/A
Study PopulationN/A
Intervention
Not Available
Study Groups/Cohorts
Not Available
Study Arms
Not Available
Arm Intervention/Treatment

Recruitment Information

Recruitment Status:Completed
Enrollment67
Completion DateJuly 28, 2016
Eligibility Criteria: - 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.
GenderAll
Age12 Years to 100 Years
Accepts Healthy VolunteersAccepts Healthy Volunteers
Contacts
Not Available
Listed Location Countries
United States

Administrative Information

NCT Number:NCT02015026
Other Study ID Numbers
140029
14-C-0029
Has Data Monitoring CommitteeNo
U.S. FDA-regulated Product Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Device Product Not Approved or Cleared by U.S. FDA: No
IPD Sharing Statement
Not Available
Responsible Party,
Study Sponsor
National Cancer Institute (NCI)
Collaborators
Not Available
Investigators
Principal Investigator
Jeremy L Davis, M.D.
National Cancer Institute (NCI)