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A Phase II Study of the Orally Administered Negative Enantiomer of Gossypol (AT-101) in Patients With Advanced Adrenocortical Carcinoma (ACC)

Phase 2
18 Years
Not Enrolling
Recurrent Adrenocortical Carcinoma, Stage III Adrenocortical Carcinoma, Stage IV Adrenocortical Carcinoma

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Trial Information

A Phase II Study of the Orally Administered Negative Enantiomer of Gossypol (AT-101) in Patients With Advanced Adrenocortical Carcinoma (ACC)


I. To determine the proportion of patients with recurrent, metastatic, or primary
unresectable adrenocortical carcinoma who achieve an objective response to R-(-)-gossypol
acetic acid.


I. To evaluate the safety of this drug in these patients. II. To determine the
progression-free and overall survival of these patients.

OUTLINE: This is a multicenter study.

Patients receive oral R-(-)-gossypol acetic acid once daily on days 1-21. Treatment repeats
every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months for up to 2

Inclusion Criteria:

- Histologically or cytologically confirmed adrenocortical carcinoma

- Recurrent, metastatic, or primary unresectable disease

- Measurable disease, defined as ≥ 1 lesion accurately measured in ≥ 1 dimension as ≥
2.0 cm by conventional techniques or ≥ 1.0 cm by spiral CT scan

- No adrenocortical tumors that, in the Principal Investigator's opinion, are
potentially resectable by surgical excision alone

- No symptomatic or progressive brain metastases

- Patients with treated brain metastases ≥ 6 months prior to study who are
clinically and radiographically stable or improved and are off steroids are

- Must undergo an MRI of the brain or CT scan of the head with contrast ≤ 4 weeks
prior to study

- ECOG performance status (PS) 0-2 or Karnofsky PS 60-100%

- Life expectancy ≥ 12 weeks

- WBC ≥ 3,000/mm3

- ANC ≥ 1,500/mm3

- Platelet count ≥ 100,000/mm3

- Total bilirubin < 1.5 mg/dL

- AST and ALT ≤ 2.5 times upper limit of normal

- Serum creatinine ≤ 1.7 mg/dL or creatinine clearance ≥ 40 mL/min

- Able to take oral medications on a regular basis

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception prior to, during, and for ≥ 1 month
after completion of study treatment

- No HIV positivity

- No uncontrolled intercurrent illness including, but not limited to, any of the

- Ongoing or active infection

- Symptomatic congestive heart failure

- Unstable angina pectoris

- Cardiac arrhythmia

- Psychiatric illness or social situation that would limit compliance with study

- No condition or disease that significantly affects gastrointestinal (GI) function or
impairs the ability to swallow and retain oral medications including, but not limited
to, any of the following:

- GI tract disease or a requirement for IV alimentation

- Prior resection of the stomach or small bowel or surgical procedures affecting

- Active peptic ulcer disease

- Malabsorption syndrome

- Ulcerative colitis

- Inflammatory bowel disease

- Partial or complete small bowel obstruction

- No other malignancy within the past 2 years except nonmelanoma skin cancer or in situ
cervical cancer

- No symptomatic hypercalcemia > grade 2

- No history of allergic reactions attributed to compounds of similar chemical or
biological composition to R-(-)-gossypol acetic acid

- Fully recovered from prior surgical procedures and recovered to ≤ grade 1 from
adverse events due to previous treatments

- No prior racemic gossypol or R-(-)-gossypol acetic acid

- More than 4 weeks since prior chemotherapy, biologic therapy, major surgery, or
radiotherapy (≥ 6 weeks for carmustine or mitomycin C)

- Prior and concurrent mitotane and ketoconazole allowed for patients with hormonal

- More than 4 weeks since prior and no concurrent treatment with another
investigational agent

- No concurrent prophylactic use of hematopoietic growth factors (e.g., filgrastim
[G-CSF], sargramostim [GM-CSF], interleukin-11) during the first course of study

- Not requiring routine use of platelet transfusions to maintain ANC or platelet count
above required thresholds

Exclusion Criteria:

- No concurrent combination antiretroviral therapy for HIV-positive patients

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The proportion of patients who achieve a confirmed objective response to treatment, either partial response (PR) or complete response (PR) as defined by Response Evaluation Criteria In Solid Tumors (RECIST) criteria

Outcome Time Frame:

Up to 2 years

Safety Issue:


Principal Investigator

Michael Menefee

Investigator Role:

Principal Investigator

Investigator Affiliation:

Mayo Clinic


United States: Food and Drug Administration

Study ID:




Start Date:

February 2009

Completion Date:

Related Keywords:

  • Recurrent Adrenocortical Carcinoma
  • Stage III Adrenocortical Carcinoma
  • Stage IV Adrenocortical Carcinoma
  • Carcinoma
  • Adrenocortical Carcinoma
  • Adrenal Cortex Neoplasms



Mayo ClinicRochester, Minnesota  55905
University of Southern CaliforniaLos Angeles, California  90033