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Phenylbutyrate and Valganciclovir in Treating Patients With Relapsed or Refractory Epstein-Barr Virus-Positive Cancer

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00387530
Recruitment Status : Withdrawn
First Posted : October 13, 2006
Last Update Posted : August 2, 2019
National Cancer Institute (NCI)
Information provided by (Responsible Party):
University of California, San Diego

Brief Summary:

RATIONALE: The Epstein-Barr virus can cause cancer and lymphoproliferative disorders. Valganciclovir is an antiviral drug that acts against the Epstein-Barr virus. Phenylbutyrate may make cells infected with Epstein-Barr virus more sensitive to valganciclovir. Giving phenylbutyrate together with valganciclovir may block the growth of Epstein-Barr virus-infected cells and kill more cancer cells.

PURPOSE: This phase II trial is studying how well giving phenylbutyrate together with valganciclovir works in treating patients with relapsed or refractory Epstein-Barr virus-positive cancer.

Condition or disease Intervention/treatment Phase
Gastric Cancer Head and Neck Cancer Lymphoma Lymphoproliferative Disorder Drug: oral sodium phenylbutyrate Drug: valganciclovir Genetic: polymerase chain reaction Genetic: protein expression analysis Procedure: biopsy Phase 2

Detailed Description:



  • Determine the rate of Epstein-Barr virus (EBV) lytic phase activation by BZLF1 expression in patients with relapsed or refractory, EBV-positive malignancies treated with phenylbutyrate.


  • Determine tumor responses in patients treated with phenylbutyrate followed by valganciclovir.
  • Track serum EBV load by quantitative polymerase chain reaction and correlate changes with EBV lytic phase activation/tumor response.

OUTLINE: This is an open-label study.

Patients receive oral phenylbutyrate three times daily on days 1-21 and oral valganciclovir once or twice daily on days 4-21. Treatment repeats every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.

Patients undergo biopsy on day 3 of course 1. Serum Epstein-Barr virus DNA is analyzed for expression of BZLF1 and LMP2 by quantitative polymerase chain reaction on days 3 and 14 of course 1 and on day 1 of each subsequent course.

After completion of study treatment, patients are followed at 1 and 3 months.

PROJECTED ACCRUAL: A total of 14 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Phenylbutyrate and Valganciclovir in Epstein-Barr Virus Positive Tumors
Actual Study Start Date : May 2006
Actual Primary Completion Date : January 2008
Actual Study Completion Date : January 2008

Arm Intervention/treatment
Single Arm Study of Biopsy Drug: oral sodium phenylbutyrate
Drug: valganciclovir
Genetic: polymerase chain reaction
Genetic: protein expression analysis
Procedure: biopsy

Primary Outcome Measures :
  1. Evidence of Epstein-Barr virus (EBV) lytic phase activation (expression of EBV antigens BZLF1 and LMP2) as assessed by biopsy on day 3 of course 1

Secondary Outcome Measures :
  1. Tumor response in patients with measurable disease as assessed by RECIST criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Biopsy-proven Epstein-Barr virus (EBV)-positive malignancy

    • Must have tissue analysis to confirm EBV positivity

      • Archival tissue ≤ 1 year old may be used
  • Any of the following malignancies:

    • WHO type II or III nasopharyngeal carcinoma
    • Post-transplant lymphoproliferative disorder
    • Nasal NK/T-cell lymphoma
    • Hodgkin's lymphoma
    • Lymphoepithelioma-variant gastric carcinoma
    • AIDS-related lymphomas

      • Patients with CNS non-Hodgkin's lymphoma must have tumor cells present in the cerebrospinal fluid (and assessable with lumbar puncture)
  • Relapsed or refractory disease

    • Must have received and failed all prior potentially curative treatment for disease
    • Eligible only for salvage therapy
  • Must have tumor tissue amenable for minimally invasive biopsy (e.g., fine-needle aspiration or bone marrow biopsy)

    • No brain tumors not amenable to biopsy
  • CNS metastases allowed provided ≥ 2 weeks since prior radiotherapy


  • ECOG performance status 0-2
  • Life expectancy ≥ 3 months
  • Absolute granulocyte count ≥ 500/mm³
  • Platelet count ≥ 50,000/mm³
  • Bilirubin ≤ 1.5 times upper limit of normal
  • Creatinine ≤ 2.0 mg/dL OR creatinine clearance ≥ 40 mL/min
  • Recovered from uncontrolled intercurrent illness, including, but not limited to, any of the following:

    • Ongoing or active infection
    • Symptomatic congestive heart failure
    • Unstable angina pectoris
    • Cardiac arrhythmia
  • Able to take medication orally or by gastrostomy tube
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception prior to, during, and for 90 days after completion of study treatment
  • No uncontrolled grade 1 symptomatic diarrhea (i.e., > 3 stools/day)
  • No concurrent serious medical or psychiatric illness that would preclude study participation


  • See Disease Characteristics
  • Concurrent cerebrospinal fluid drugs allowed
  • No concurrent zidovudine for HIV-positive patients

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00387530

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United States, California
Rebecca and John Moores UCSD Cancer Center
La Jolla, California, United States, 92093-0658
Sponsors and Collaborators
University of California, San Diego
National Cancer Institute (NCI)
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Study Chair: William L. Read, MD University of California, San Diego
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Responsible Party: University of California, San Diego Identifier: NCT00387530    
Other Study ID Numbers: UCSD-050126
CDR0000504022 ( Registry Identifier: PDQ (Physician Data Query) )
First Posted: October 13, 2006    Key Record Dates
Last Update Posted: August 2, 2019
Last Verified: July 2019
Keywords provided by University of California, San Diego:
recurrent lymphoepithelioma of the nasopharynx
post-transplant lymphoproliferative disorder
adult nasal type extranodal NK/T-cell lymphoma
recurrent adult Hodgkin lymphoma
AIDS-related peripheral/systemic lymphoma
AIDS-related primary CNS lymphoma
recurrent gastric cancer
Additional relevant MeSH terms:
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Stomach Neoplasms
Lymphoproliferative Disorders
Neoplasms by Histologic Type
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Gastrointestinal Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Gastrointestinal Diseases
Stomach Diseases
4-phenylbutyric acid
Antiviral Agents
Anti-Infective Agents
Antineoplastic Agents