Clinical trial NCT00041275: Megestrol in Treating Patients With Liver Cancer That Cannot Be Removed By Surgery
Official title: Randomized Double Blind Trial Of Megestrol Acetate Versus Placebo For The Treatment Of Inoperable Hepatocellular Carcinoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Liver Cancer
Keywords: localized unresectable adult primary liver cancer
advanced adult primary liver cancer
adult primary hepatocellular carcinoma
Lead sponsor: National Cancer Institute (NCI)
Sponsor class: NIH
First posted: 2003-01-28
Enrollment: 300
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: OBJECTIVES:

* 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.

Eligibility criteria: DISEASE CHARACTERISTICS:

* 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

PATIENT CHARACTERISTICS:

Age:

* 20 to 100

Performance status:

* ECOG 0-3

Life expectancy:

* Not specified

Hematopoietic:

* Not specified

Hepatic:

* Bilirubin no greater than 5.8 mg/dL

Renal:

* Creatinine less than 1.7 mg/dL

Other:

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

PRIOR CONCURRENT THERAPY:

Biologic therapy:

* Not specified

Chemotherapy:

* No prior chemoembolization for HCC
* No prior systemic chemotherapy for HCC

Endocrine therapy:

* Not specified

Radiotherapy:

* Not specified

Surgery:

* See Disease Characteristics
* No prior surgery for HCC

Other:

* No prior percutaneous injection for HCC

Primary outcomes:
measure: Survival measured weekly
Secondary outcomes:
measure: Quality of life as measured by EORTC quality of life instrument monthly
Other outcomes:
Locations:
city: Dhaka
country: Bangladesh
facility: Bangabandhu Sheikh Mujib Medical University
city: Denpasar
country: Indonesia
facility: Rumah Sakit Sanglah
state: Bali
city: Suwon
country: Korea, Republic of
facility: St. Vincent Hospital
city: Yangon
country: Myanmar
facility: Yangon General Hospital
city: Auckland
country: New Zealand
facility: Auckland City Hospital
city: Davao City
country: Philippines
facility: Davao Doctors Hospital
city: Singapore
country: Singapore
facility: National Cancer Centre - Singapore
city: Singapore
country: Singapore
facility: Changi General Hospital
city: Taipei
country: Taiwan
facility: Chang-Gung Memorial Hospital - Taipei
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital
city: Ha Noi
country: Vietnam
facility: National Cancer Institute
city: Ho Chi Minh City
country: Vietnam
facility: Cho Ray Hospital
Study officials:
affiliation: National Cancer Centre, Singapore
name: Pierce Chow, MD, PhD, MBBS, FRCS, FAMS
role: STUDY_CHAIR
References:
citation: Chow PK, Machin D, Chen Y, Zhang X, Win KM, Hoang HH, Nguyen BD, Jin MY, Lobo R, Findlay M, Lim CH, Tan SB, Gandhi M, Soo KC; Asia-Pacific Hepatocellular Carcinoma Trials Group. Randomised double-blind trial of megestrol acetate vs placebo in treatment-naive advanced hepatocellular carcinoma. Br J Cancer. 2011 Sep 27;105(7):945-52. doi: 10.1038/bjc.2011.333. Epub 2011 Aug 23.