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Testing of ADI-PEG in Hepatocellular Carcinoma

This study has been completed.
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: March 26, 2003
Last updated: March 24, 2015
Last verified: September 2002

Amino acid deprivation therapy is an effective means for the treatment of some forms of cancer. Recently it has been found that human hepatocellular carcinomas (HCC) cell lines appear to require arginine for growth. Arginine is not an essential amino acid for human adults or infants as it can be synthesized from citrulline (for review see Rogers 1994). Therefore, selective elimination of arginine from the circulation may be a means of treating patients with metastatic melanoma or non resectable HCC.

The enzyme arginine deiminase (ADI) metabolizes arginine into citrulline (Cunin 1986). However, ADI is only found in microbes and not in humans. ADI is therefore, highly immunogenic and has a short serum half-life following injection. These potential drawbacks (microbial source and thus viewed as foreign by the human immune system, and a short serum half-life) can be overcome by covalent attachment of polyethylene glycol (PEG) to argininedeiminase and termed this drug ADI-PEG 20.

ADI-PEG 20 appears to be an effective anti-cancer treatment for human HCC. Pharmacokinetic and pharmacodynamic data indicates a once a week injection of 160 IU/m2 of ADI-PEG 20 eliminates all detectable arginine from the circulation for at least 7 days. This treatment appears to be well tolerated. The purpose of this study is to determine the efficacy of this treatment in patients with HCC. Efficacy is a primary end point of this study. No patients will recieve placebo.

Condition Intervention Phase
Carcinoma, Hepatocellular Drug: ADI-PEG 20 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Testing of ADI-PEG in Hepatocellular Carcinoma

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 34
Study Start Date: September 2002
Estimated Study Completion Date: October 2003

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • Histologically confirmed diagnosis of hepatocellular carcinoma.
  • Non-resectable disease.
  • Progressive disease after chemotherapy, radiotherapy, surgery or immuno-therapy, and be no longer responding to such therapy, or have refused such therapy.
  • Been off previous treatment for at least 4 weeks.
  • Been fully recovered from all prior surgery.
  • Age of > 18 years.
  • Karnofsky performance status of > 70.
  • Expected survival of > 12 weeks.
  • Total bilirubin < 3.0 mg/dl.
  • Serum albumin > 3.0 g/dl.
  • Serum SGOT < 5 x upper limit of normal.
  • Serum alkaline phosphatase < 5 x upper limit of normal.
  • Serum ammonia < 55 mg/dl.
  • Serum glucose > 60 mg/dl.
  • Serum amylase < 1.5 x upper limit of normal.
  • ANC > 1,500 / ml.
  • Platelets > 100,000 / ml.
  • Female subjects of childbearing age and male subjects must be asked to use appropriate contraception for both the male and female for the duration of the study. Subjects must agree to use two forms of contraception or agree to refrain from intercourse for the duration of the study. Females must not be pregnant at the start of the study, and a serum HCG pregnancy test must be negative before entry into the study.
  • Informed consent.
  • Not be enrolled in other IND studies.
  • Disease must be measurable or evaluable.
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Please refer to this study by its identifier: NCT00056992

United States, Texas
MD Anderson Cancer center
Houston, Texas, United States
Sponsors and Collaborators
FDA Office of Orphan Products Development
  More Information Identifier: NCT00056992     History of Changes
Other Study ID Numbers: 2206
Study First Received: March 26, 2003
Last Updated: March 24, 2015

Additional relevant MeSH terms:
Carcinoma, Hepatocellular
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Liver Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Liver Diseases processed this record on August 22, 2017