Phenylbutyrate in Treating Patients With Refractory Solid Tumors or Lymphoma
![]() |
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. |
ClinicalTrials.gov Identifier: NCT00002909 |
Recruitment Status :
Completed
First Posted : August 11, 2004
Last Update Posted : February 5, 2013
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Lymphoma Unspecified Adult Solid Tumor, Protocol Specific | Drug: chemotherapy Drug: oral sodium phenylbutyrate | Phase 1 |
OBJECTIVES:
I. Determine the maximum tolerated dose of oral phenylbutyrate that can be given three times daily in patients with refractory solid tumors or lymphoma until disease progression or a trough plasma concentration of 2-6 micromoles per liter is achieved.
II. Characterize the pharmacokinetics of oral phenylbutyrate absorption, and determine whether there is time dependence in the systemic clearance rate during multiple dosing.
III. Seek preliminary evidence of therapeutic activity of phenylbutyrate when administered on this schedule in these patients.
IV. Correlate any observed responses and toxic effects with results of bioassays and tissue sampling for phenylbutyrate activity.
OUTLINE: This is a dose-seeking study.
All patients receive oral phenylbutyrate three times daily. Groups of 4 or more patients receive escalating doses of phenylbutyrate until the maximum tolerated dose (MTD) is determined. Treatment continues until disease progression or unacceptable toxicity intervenes.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 24 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A PHASE I CLINICAL AND PHARMACOLOGIC EVALUATION OF PHENYLBUTYRATE IN PATIENTS WITH REFRACTORY SOLID TUMORS: STUDY OF CONTINUOUS EXPOSURE ORAL PHENYLBUTYRATE ON A THREE TIMES DAILY SCHEDULE |
Study Start Date : | February 1997 |
Actual Primary Completion Date : | October 2000 |

Arm | Intervention/treatment |
---|---|
Experimental: Arm I
All patients receive oral phenylbutyrate three times daily. Groups of 4 or more patients receive escalating doses of phenylbutyrate until the maximum tolerated dose (MTD) is determined. Treatment continues until disease progression or unacceptable toxicity intervenes.
|
Drug: chemotherapy Drug: oral sodium phenylbutyrate |

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
- Histologically or cytologically confirmed solid tumor or lymphoma that is progressive or refractory to conventional therapy or for which no effective therapy is known
- Priority given to hormone-refractory prostate cancer and melanoma
- No untreated CNS metastases
- Head CT not required in the absence of clinical signs or symptoms but recommended for metastatic melanoma
PATIENT CHARACTERISTICS:
- Age: 18 and over
- Performance status: ECOG 0-2
- Life expectancy: At least 3 months
- WBC greater than 2,000/mm3 OR absolute neutrophil count greater than 1,500/mm3
- Platelet count greater than 100,000/mm3
- Hemoglobin greater than 9 g/dL
- Bilirubin less than 1.5 mg/dL
- AST/ALT less than 1.5 times normal
- Creatinine less than 2.0 mg/dL
- Obstructive uropathy allowed if relieved by nephrostomy or other appropriate device
- Left ventricular ejection fraction greater than 40% by MUGA or normal by echocardiogram
- No history of congestive heart failure
- No uncontrolled hypertension (diastolic greater than 110 mm Hg)
- Forced expiratory volume (1 second) greater than 1.5 L/min
- No active infection, including HIV or viral hepatitis
- No active seizure disorder
- No clinical evidence of increased intracranial pressure
- No baseline dementia (mini-mental exam less than 23)
- No autoimmune disease, including systemic lupus erythematosus, scleroderma, or other connective tissue illness
- No nonmalignant medical or psychiatric problem of sufficient severity to limit full compliance with study or to pose undue risk
- Not pregnant or nursing
- Adequate contraception required of fertile patients for 2 weeks prior to, during, and for 3 months after study
- Normal gastrointestinal function with ability to tolerate large intake of oral volume required for phenylbutyrate administration
- Patients with persistent nausea or moderate to severe anorexia may not be good candidates for study
PRIOR CONCURRENT THERAPY:
- Fully recovered from all prior therapy
- Prior suramin allowed if serum suramin level less than 50 micrograms/mL at entry
- At least 28 days since chemotherapy
- No prior phenylacetate, phenylbutyrate, or antineoplaston therapy
- The following may be continued during study:
- Luteinizing hormone-releasing hormone agonist with evidence of tumor progression (e.g., rising prostate-specific antigen, new lesions on bone scan) despite therapy
- Adrenal steroid replacement (if needed)
- Dexamethasone or other steroids
- At least 4-6 weeks since flutamide with evidence of PSA progression required (if possible)
- At least 28 days since radiotherapy
- At least 28 days since major surgery

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 ClinicalTrials.gov identifier (NCT number): NCT00002909
United States, Maryland | |
Johns Hopkins Oncology Center | |
Baltimore, Maryland, United States, 21287 |
Study Chair: | Michael A. Carducci, MD | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
Responsible Party: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00002909 |
Other Study ID Numbers: |
NCI-2012-02250 J-9616 JHOC-9616 NCI-T95-0017H CDR0000065274 ( Registry Identifier: PDQ (Physician Data Query) ) |
First Posted: | August 11, 2004 Key Record Dates |
Last Update Posted: | February 5, 2013 |
Last Verified: | February 2001 |
stage III adult Hodgkin lymphoma stage IV adult Hodgkin lymphoma recurrent adult Hodgkin lymphoma unspecified adult solid tumor, protocol specific stage III grade 1 follicular lymphoma stage III grade 2 follicular lymphoma stage III grade 3 follicular lymphoma stage III adult diffuse small cleaved cell lymphoma stage III adult diffuse mixed cell lymphoma stage III adult diffuse large cell lymphoma stage III adult immunoblastic large cell lymphoma stage III adult lymphoblastic lymphoma stage III adult Burkitt lymphoma stage IV grade 1 follicular lymphoma stage IV grade 2 follicular lymphoma |
stage IV grade 3 follicular lymphoma stage IV adult diffuse small cleaved cell lymphoma stage IV adult diffuse mixed cell lymphoma stage IV adult diffuse large cell lymphoma stage IV adult immunoblastic large cell lymphoma stage IV adult lymphoblastic lymphoma stage IV adult Burkitt lymphoma recurrent grade 1 follicular lymphoma recurrent grade 2 follicular lymphoma recurrent grade 3 follicular lymphoma recurrent adult diffuse small cleaved cell lymphoma recurrent adult diffuse mixed cell lymphoma recurrent adult diffuse large cell lymphoma recurrent adult immunoblastic large cell lymphoma recurrent adult lymphoblastic lymphoma |
Lymphoma Neoplasms by Histologic Type Neoplasms Lymphoproliferative Disorders Lymphatic Diseases |
Immunoproliferative Disorders Immune System Diseases 4-phenylbutyric acid Antineoplastic Agents |