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Fenretinide in Treating Patients With Refractory or Relapsed Hematologic 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: NCT00104923
Recruitment Status : Completed
First Posted : March 4, 2005
Last Update Posted : July 19, 2017
National Cancer Institute (NCI)
Information provided by (Responsible Party):
California Cancer Consortium

Brief Summary:

RATIONALE: Drugs used in chemotherapy, such as fenretinide, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving fenretinide in a different way may kill more cancer cells.

PURPOSE: This phase I trial is studying the side effects and best dose of intravenous fenretinide in treating patients with refractory or relapsed hematologic cancer.

Condition or disease Intervention/treatment Phase
Chronic Myeloproliferative Disorders Leukemia Lymphoma Multiple Myeloma and Plasma Cell Neoplasm Drug: fenretinide Phase 1

Detailed Description:


  • Determine the maximum tolerated dose of intravenous emulsified fenretinide in patients with refractory or relapsed hematologic malignancies.
  • Determine the toxic effects of this drug in these patients.
  • Determine the pharmacokinetics and in vivo activity of this drug in these patients.
  • Determine, preliminarily, disease or tumor response in patients treated with this drug.

OUTLINE: This is a pilot, dose-escalation, multicenter study.

Patients receive emulsified fenretinide IV continuously over 5 days. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who achieve a complete or partial response may continue to receive fenretinide at the discretion of the study chair.

Cohorts of 1 patient receive accelerated escalating doses of fenretinide until 2 patients experience moderate toxicity (cumulative across all dose levels) OR 1 patient experiences dose-limiting toxicity (DLT). After completion of the accelerated dose-escalation portion, the standard dose-escalation portion begins. Cohorts of 3-6 patients receive escalating doses of fenretinide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience DLT. At least 6 patients are treated at the MTD. An additional 12 patients are treated at the MTD.

After completion of study treatment, patients are followed every 3 months for 2 years, every 6 months for 3 years, and then annually thereafter.

PROJECTED ACCRUAL: Approximately 40 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 29 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Trial of Intravenous Fenretinide (4-HPR) for Patients With Hematologic Malignancies
Study Start Date : February 2005
Actual Primary Completion Date : April 2017
Actual Study Completion Date : April 2017

Intervention Details:
  • Drug: fenretinide
    Current dose level as an IV continuous infusion via central line over 5 days. Cycle is repeated every 3 weeks for up to 6 cycles

Primary Outcome Measures :
  1. To determine the maximum tolerated dose of fenretinide [ Time Frame: participants will be followed for the duration of cycle 1, which is expected to be 3 weeks. ]
  2. To describe the toxicities of fenretinide [ Time Frame: participants will be followed for the duration of treatment, which is expected to be 18 weeks or less ]

Information from the National Library of Medicine

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.

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Ages Eligible for Study:   18 Years to 120 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically or cytologically confirmed diagnosis of 1 of the following hematologic malignancies:

    • Non-Hodgkin's lymphoma (NHL)
    • Hodgkin's lymphoma
    • Multiple myeloma
    • Acute lymphoblastic leukemia
    • Acute myeloid leukemia
    • Chronic hematologic malignancy with a poor prognosis (e.g., failed 3 prior standard therapies), including any of the following:

      • Chronic lymphocytic leukemia
      • Chronic myelogenous leukemia
      • Indolent NHL
      • Myeloproliferative disorders
  • Refractory or relapsed disease, as defined by 1 of the following:

    • Resistant to standard therapy for refractory or relapsed disease
    • Progressed after standard therapy for advanced disease
  • No effective treatment exists
  • Measurable or evaluable disease
  • No active CNS disease

    • Previously treated leptomeningeal disease or brain metastases allowed provided there is no evidence of remaining cancer by positron-emission tomography, MRI, or spinal fluid cytology



  • 18 and over

Performance status

  • ECOG 0-2

Life expectancy

  • At least 3 months


  • Absolute neutrophil count ≥ 1,500/mm^3 (unless due to bone marrow involvement of disease)
  • Platelet count ≥ 75,000/mm^3 (unless due to bone marrow involvement of disease)
  • Hemoglobin ≥ 8.0 g/dL (transfusion allowed)
  • No coagulation disorders


  • AST and ALT < 2.5 times upper limit of normal (ULN) (≤ 5 times ULN for patients with liver metastasis)
  • Bilirubin ≤ 1.5 times ULN


  • Creatinine ≤ 1.5 times ULN


  • No major cardiovascular disease


  • No major respiratory disease


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective double-method contraception prior to study entry, during study, and for at least 6 months after study participation
  • No uncontrolled systemic infection
  • No uncontrolled hypertriglyceridemia (i.e., triglyceride level > 500 mg/dL)
  • No known HIV positivity
  • No known allergy to egg products
  • No known familial hyperlipidemia disorders
  • No previously undiscovered hypertriglyceridemia
  • No poorly controlled diabetes


Biologic therapy

  • Not specified


  • More than 2 weeks since prior chemotherapy except hydroxyurea

    • No concurrent hydroxyurea during study drug administration
  • No other concurrent anticancer chemotherapy

Endocrine therapy

  • No concurrent hormone-ablative agents
  • No concurrent steroids
  • No concurrent tamoxifen or any of its analogues


  • No prior cranial radiotherapy
  • More than 2 weeks since prior radiotherapy


  • More than 20 days since prior surgery except for biopsy


  • Recovered from all prior therapy
  • More than 2 weeks since prior investigational agents
  • No other concurrent investigational agents
  • No other concurrent antineoplastic therapy
  • No other concurrent antioxidants
  • No concurrent herbal or other alternative therapies
  • No concurrent vitamin supplements (e.g., vitamin A, ascorbic acid, or vitamin E)

    • Standard dose multivitamin allowed
  • No other concurrent medications that may act as modulators of intracellular ceramide levels or ceramide cytotoxicity, sphingolipid transport, or p-glycoprotein or multidrug resistance protein 1 (MRP1) drug/lipid transporters, including any of the following:

    • Cyclosporine or any of its analogues
    • Verapamil
    • Ketoconazole
    • Chlorpromazine
    • Mifepristone
    • Indomethacin
    • Sulfinpyrazone
  • No concurrent medications that may cause pseudotumor cerebri, including any of the following:

    • Tetracycline
    • Nalidixic acid
    • Nitrofurantoin
    • Phenytoin
    • Sulfonamides
    • Lithium
    • Amiodarone
  • No concurrent medication to control hypertriglyceridemia

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): NCT00104923

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United States, California
USC/Norris Comprehensive Cancer Center and Hospital
Los Angeles, California, United States, 90089-9181
United States, Maryland
Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office
Bethesda, Maryland, United States, 20892-1182
United States, Texas
M. D. Anderson Cancer Center at University of Texas
Houston, Texas, United States, 77030-4009
Joe Arrington Cancer Research and Treatment Center
Lubbock, Texas, United States, 79410-1894
Sponsors and Collaborators
California Cancer Consortium
National Cancer Institute (NCI)
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Study Chair: Ann Mohrbacher, MD University of Southern California
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Responsible Party: California Cancer Consortium Identifier: NCT00104923    
Other Study ID Numbers: CDR0000413887
P30CA033572 ( U.S. NIH Grant/Contract )
First Posted: March 4, 2005    Key Record Dates
Last Update Posted: July 19, 2017
Last Verified: July 2017
Keywords provided by California Cancer Consortium:
adult acute myeloid leukemia with 11q23 (MLL) abnormalities
adult acute myeloid leukemia with inv(16)(p13;q22)
adult acute myeloid leukemia with t(15;17)(q22;q12)
adult acute myeloid leukemia with t(16;16)(p13;q22)
adult acute myeloid leukemia with t(8;21)(q22;q22)
recurrent grade 1 follicular lymphoma
recurrent grade 2 follicular lymphoma
recurrent grade 3 follicular lymphoma
recurrent small lymphocytic lymphoma
recurrent mantle cell lymphoma
Waldenstrom macroglobulinemia
adult grade III lymphomatoid granulomatosis
recurrent adult grade III lymphomatoid granulomatosis
secondary acute myeloid leukemia
recurrent adult Burkitt lymphoma
recurrent adult immunoblastic large cell lymphoma
refractory chronic lymphocytic leukemia
recurrent adult lymphoblastic lymphoma
recurrent adult Hodgkin lymphoma
refractory multiple myeloma
recurrent adult acute lymphoblastic leukemia
relapsing chronic myelogenous leukemia
chronic eosinophilic leukemia
primary myelofibrosis
chronic neutrophilic leukemia
essential thrombocythemia
polycythemia vera
stage II multiple myeloma
stage III multiple myeloma
recurrent adult diffuse large cell lymphoma
Additional relevant MeSH terms:
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Multiple Myeloma
Neoplasms, Plasma Cell
Myeloproliferative Disorders
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Bone Marrow Diseases
Antineoplastic Agents
Anticarcinogenic Agents
Protective Agents
Physiological Effects of Drugs