Megestrol in Treating Patients With Liver Cancer That Cannot Be Removed By Surgery

This study has been completed.
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: July 8, 2002
Last updated: September 19, 2013
Last verified: September 2002

RATIONALE: Estrogen can stimulate the growth of cancer cells. Hormone therapy using megestrol may fight liver cancer by blocking the uptake of estrogen. It is not yet known if megestrol is an effective treatment for liver cancer.

PURPOSE: Randomized phase III trial to determine the effectiveness of megestrol in treating patients who have liver cancer that cannot be removed by surgery.

Condition Intervention Phase
Liver Cancer
Drug: megestrol acetate
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: Randomized Double Blind Trial Of Megestrol Acetate Versus Placebo For The Treatment Of Inoperable Hepatocellular Carcinoma

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Survival measured weekly [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Quality of life as measured by EORTC quality of life instrument monthly [ Designated as safety issue: No ]

Estimated Enrollment: 300
Study Start Date: May 2002
Study Completion Date: August 2011
Primary Completion Date: June 2007 (Final data collection date for primary outcome measure)
Detailed Description:


  • Compare the overall survival of patients with inoperable hepatocellular carcinoma treated with megestrol vs placebo.
  • Compare the quality of life of patients treated with these drugs.

OUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study. Patients are stratified according to participating center. Patients are randomized to 1 of 2 treatment arms.

  • Arm I: Patients receive oral megestrol twice daily for 1 year.
  • Arm II: Patients receive an oral placebo twice daily for 1 year. Quality of life is assessed at baseline and then monthly for 1 year.

PROJECTED ACCRUAL: A minimum of 300 patients (200 for arm I and 100 for arm II) will be accrued for this study.


Ages Eligible for Study:   20 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Diagnosis of hepatocellular carcinoma (HCC)

    • Histologically confirmed OR
    • Meets 2 of the following criteria:

      • Radiological evidence of HCC on CT scan, MRI, or ultrasound
      • Serum alpha-fetoprotein level at least 400 µg/L
      • Positive lipiodol retention
  • Not amenable to surgery



  • 20 to 100

Performance status:

  • ECOG 0-3

Life expectancy:

  • Not specified


  • Not specified


  • Bilirubin no greater than 5.8 mg/dL


  • Creatinine less than 1.7 mg/dL


  • Not pregnant
  • No clinical encephalopathy
  • No other malignancy within the past 5 years


Biologic therapy:

  • Not specified


  • No prior chemoembolization for HCC
  • No prior systemic chemotherapy for HCC

Endocrine therapy:

  • Not specified


  • Not specified


  • See Disease Characteristics
  • No prior surgery for HCC


  • No prior percutaneous injection for HCC
  Contacts and Locations
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Please refer to this study by its identifier: NCT00041275

Bangabandhu Sheikh Mujib Medical University
Dhaka, Bangladesh, 1000
Rumah Sakit Sanglah
Denpasar, Bali, Indonesia, 20114
Korea, Republic of
St. Vincent Hospital
Suwon, Korea, Republic of, 442-060
Yangon General Hospital
Yangon, Myanmar
New Zealand
Auckland City Hospital
Auckland, New Zealand, 5
Davao Doctors Hospital
Davao City, Philippines
Changi General Hospital
Singapore, Singapore, 529889
National Cancer Centre - Singapore
Singapore, Singapore, 169608
Chang-Gung Memorial Hospital - Taipei
Taipei, Taiwan, 333
Ramathibodi Hospital
Bangkok, Thailand, 10400
National Cancer Institute
Ha Noi, Vietnam
Cho Ray Hospital
Ho Chi Minh City, Vietnam
Sponsors and Collaborators
National Medical Research Council (NMRC), Singapore
Study Chair: Pierce Chow, MD, PhD, MBBS, FRCS, FAMS National Cancer Centre, Singapore
  More Information

Additional Information:
Publications: Identifier: NCT00041275     History of Changes
Other Study ID Numbers: CDR0000069460, NMRC-AHCC02, EU-20203
Study First Received: July 8, 2002
Last Updated: September 19, 2013
Health Authority: United States: Federal Government

Keywords provided by National Cancer Institute (NCI):
localized unresectable adult primary liver cancer
advanced adult primary liver cancer
adult primary hepatocellular carcinoma

Additional relevant MeSH terms:
Carcinoma, Hepatocellular
Liver Neoplasms
Digestive System Diseases
Digestive System Neoplasms
Liver Diseases
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Glandular and Epithelial
Megestrol Acetate
Antineoplastic Agents
Antineoplastic Agents, Hormonal
Appetite Stimulants
Central Nervous System Agents
Central Nervous System Stimulants
Contraceptive Agents
Contraceptive Agents, Female
Contraceptives, Oral
Contraceptives, Oral, Synthetic
Pharmacologic Actions
Physiological Effects of Drugs
Reproductive Control Agents
Therapeutic Uses processed this record on October 08, 2015