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17-N-Allylamino-17-Demethoxygeldanamycin in Treating Patients With Systemic Mastocytosis

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: NCT00132015
Recruitment Status : Completed
First Posted : August 19, 2005
Last Update Posted : March 15, 2012
National Cancer Institute (NCI)
Information provided by:
National Institutes of Health Clinical Center (CC)

Brief Summary:

RATIONALE: Drugs used in chemotherapy, such as 17-N-allylamino-17-demethoxygeldanamycin (17-AAG), work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying how well 17-AAG works in treating patients with systemic mastocytosis.

Condition or disease Intervention/treatment Phase
Chronic Myeloproliferative Disorders Leukemia Lymphoma Nonneoplastic Condition Precancerous Condition Drug: tanespimycin Phase 2

Detailed Description:



  • Determine the efficacy of 17-N-allylamino-17-demethoxygeldanamycin (17-AAG), in terms of decreases in the number of mast cells in the bone marrow and in serum tryptase levels, in patients with systemic mastocytosis.


  • Determine the quality of life of patients treated with this drug.
  • Determine hematological and non-hematological toxicity of this drug in these patients.

OUTLINE: This is a multicenter study.

Patients receive 17-N-allylamino-17-demethoxygeldanamycin (17-AAG) IV over 2-6 hours on days 1, 4, 8, and 11. Treatment repeats every 21 days for at least 6 courses in the absence of disease progression or unacceptable toxicity. Patients achieving a complete response (CR) receive at least 2 additional courses beyond CR. Patients achieving a partial response receive at least 4 additional courses beyond their maximum response. Selected patients may receive additional courses of therapy beyond the protocol guidelines at the discretion of the principal investigator.

Quality of life is assessed at baseline and before each treatment course.

PROJECTED ACCRUAL: A total of 12-37 patients will be accrued for this study within approximately 10-18 months.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 37 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Clinical Trial of 17-(Allylamino)-17- Demethoxygeldanamycin (17-AAG, NSC 330507 and EPL Diluent, NSC 704057) in Adults With Systemic Mastocytosis
Study Start Date : May 2006
Actual Primary Completion Date : December 2007
Actual Study Completion Date : June 2008

Primary Outcome Measures :
  1. Objective response (complete and partial response)

Secondary Outcome Measures :
  1. Quality of life as assessed by the European Organization for Research of the Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) at baseline and prior to each treatment course

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


  • Histologically confirmed systemic mastocytosis

    • Objective evidence of disease, as defined by the following:

      • Hemoglobin < 10 g/dL
      • Recurrent mast cell mediator-release symptoms that impair the patient's quality of life
      • Symptomatic hepatosplenomegaly
      • Ascites
      • Symptomatic bone disease
      • Profound constitutional symptoms (e.g., fatigue, asthenia, flushing, hyperpyrexia, weight loss, myalgia, and arthralgia)
      • Elevated serum tryptase level
  • Mast cell leukemia allowed
  • Mastocytosis associated with myeloproliferative disease (e.g., hypereosinophilic syndrome or chronic myelomonocytic leukemia) allowed
  • Patients with eosinophilia (i.e., absolute eosinophil count ≥ 1,000/mm^3) must be evaluated for the presence or absence of FIP1L1-PDGFRA mutation; if the mutation is absent, the patient is eligible; if the mutation is present, the patient is eligible provided disease is refractory to imatinib mesylate
  • Patients with indolent disease must have a serum tryptase level ≥ 50 ng/mL OR episodes of anaphylaxis that occur with a frequency of > 1 per month



  • 18 and over

Performance status

  • ECOG 0-2

Life expectancy

  • At least 3 months


  • See Disease Characteristics
  • Platelet count ≥ 100,000/mm^3 (> 25,000/mm^3 for patients with organomegaly)
  • Absolute granulocyte count ≥ 1,500/mm^3(> 750/mm^3 for patients with organomegaly)


  • AST and ALT ≤ 2 times upper limit of normal (ULN) (< 4 times ULN for patients with hepatomegaly)
  • Bilirubin normal
  • Alkaline phosphatase ≤ 3 times ULN


  • Creatinine ≤ 1.4 mg/dL OR
  • Creatinine clearance ≥ 60 mL/min


  • No New York Heart Association class III-IV congestive heart failure
  • No history of myocardial infarction within the past year
  • No history of uncontrolled dysrhythmia
  • No uncontrolled angina
  • No ischemic heart disease within the past 12 months
  • No congenital long QT syndrome
  • No left bundle branch block
  • No serious ventricular arrhythmia (ventricular tachycardia or ventricular fibrillation ≥ 3 beats in a row)
  • QTc interval < 450 msec for males or 470 msec for females
  • LVEF > 40% by MUGA
  • MUGA or echocardiogram normal
  • No prior history of cardiac toxicity after receiving anthracyclines (e.g., doxorubicin hydrochloride, daunorubicin hydrochloride, mitoxantrone hydrochloride, bleomycin, or carmustine)
  • No cardiac symptoms ≥ grade 2
  • No other significant cardiac disease


  • No symptomatic pulmonary disease requiring medication including any of the following:

    • Dyspnea on or off exertion
    • Paroxysmal nocturnal dyspnea
    • Requirement for oxygen
    • Significant pulmonary disease (e.g., chronic obstructive/restrictive pulmonary disease)
  • No home oxygen meeting the Medicare requirement
  • No compromised pulmonary status (i.e., DLCO ≤ 80%)
  • No prior history of pulmonary toxicity after receiving anthracyclines (e.g., doxorubicin hydrochloride, daunorubicin hydrochloride, mitoxantrone hydrochloride, bleomycin, or carmustine)
  • No pulmonary symptoms ≥ grade 2


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception during and for ≥ 6 months after completion of study treatment
  • HIV negative
  • No active uncontrolled infection
  • No serious medical illness
  • No other non-malignant systemic disease
  • No history of serious allergic reaction to eggs
  • No other malignancy within the past 2 years except dermatological cancer


Biologic therapy

  • Not specified


  • At least 4 weeks since prior chemotherapy

Endocrine therapy

  • Steroids allowed provided tapering to the lowest level possible to treat thrombocytopenia, diarrhea, or malabsorption symptoms of systemic mastocytosis


  • At least 4 weeks since prior radiotherapy
  • No prior radiation that included the heart in the field (e.g., mantle) or chest


  • Not specified


  • At least 4 weeks since prior tyrosine kinase inhibitors
  • No concurrent complimentary or alternative medications* including, but not limited to, the following:

    • Hypericum perforatum (St. John's wort)
    • Milk thistle
    • Kava kava
    • Mistletoe extract
  • No concurrent agents that cause QTc prolongation
  • No concurrent antiarrhythmic therapy
  • No other concurrent investigational therapy NOTE: *Unless approved by the investigator

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): NCT00132015

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United States, Maryland
Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office
Bethesda, Maryland, United States, 20892-1182
NCI - Center for Cancer Research
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Institutes of Health Clinical Center (CC)
National Cancer Institute (NCI)
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Principal Investigator: Antonio T. Fojo, MD, PhD National Cancer Institute (NCI)
Layout table for additonal information Identifier: NCT00132015    
Obsolete Identifiers: NCT00285311
Other Study ID Numbers: 060076
First Posted: August 19, 2005    Key Record Dates
Last Update Posted: March 15, 2012
Last Verified: March 2012
Keywords provided by National Institutes of Health Clinical Center (CC):
Waldenström macroglobulinemia
multicentric Castleman disease
unicentric Castleman disease
adult grade III lymphomatoid granulomatosis
polycythemia vera
essential thrombocythemia
hairy cell leukemia
monoclonal gammopathy of undetermined significance
adult Burkitt lymphoma
adult diffuse large cell lymphoma
adult diffuse mixed cell lymphoma
adult diffuse small cleaved cell lymphoma
adult immunoblastic large cell lymphoma
adult lymphoblastic lymphoma
grade 1 follicular lymphoma
grade 2 follicular lymphoma
grade 3 follicular lymphoma
mantle cell lymphoma
marginal zone lymphoma
small lymphocytic lymphoma
precancerous condition
Additional relevant MeSH terms:
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Precancerous Conditions
Mastocytosis, Systemic
Myeloproliferative Disorders
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Skin Diseases
Immune Complex Diseases