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Sulindac and Tamoxifen in Treating Patients With Desmoid Tumor

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: NCT00068419
Recruitment Status : Completed
First Posted : September 11, 2003
Results First Posted : December 4, 2013
Last Update Posted : February 19, 2020
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group

Brief Summary:
This phase II trial is studying how well giving sulindac together with tamoxifen works in treating patients with desmoid tumor. Sulindac may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth. Hormone therapy using tamoxifen may fight cancer by blocking the use of estrogen. Combining sulindac with tamoxifen may kill more cancer cells.

Condition or disease Intervention/treatment Phase
Desmoid Tumor Drug: tamoxifen citrate Drug: sulindac Other: laboratory biomarker analysis Phase 2

Detailed Description:


I. To estimate the safety and efficacy of sulindac and tamoxifen in patients with recurrent desmoid tumor (DT) and primary DT that is not readily amenable to surgery or radiation therapy.


I. Determine the tumor response rate in patients treated with this regimen.

II. Correlate changes in Magnetic Resonance Imaging (MRI) signal features of the tumor with clinical outcome in patients treated with this regimen.

III. Correlate pathological studies of cyclooxygenase-2 (COX-2) and estrogen/progesterone receptor expression in the tumor with clinical outcome in patients treated with this regimen.

IV. Collect information about clinical factors that make a tumor unresectable at diagnosis and resectable during the four courses of study treatment.

V. Determine whether short-term endocrine toxicity is associated with treatment with this regimen in these patients.

OUTLINE: This is a multicenter study.

Patients receive oral sulindac and oral tamoxifen twice daily for up to 12 months (four 3-month courses) in the absence of disease progression or unacceptable toxicity. Patients who achieve a complete response (CR) receive 1 additional month of treatment beyond documentation of CR.

After completion of study treatment, patients are followed for 5 years.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 70 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Sulindac and Tamoxifen in Patients With Desmoid Tumors That Are Recurrent or Not Amenable to Standard Therapy
Study Start Date : February 2004
Actual Primary Completion Date : April 26, 2010
Actual Study Completion Date : April 26, 2010

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Treatment (enzyme inhibitor therapy, anti-estrogen therapy)
Patients receive oral sulindac and oral tamoxifen citrate twice daily for up to 12 months (four 3-month courses) in the absence of disease progression or unacceptable toxicity. Patients who achieve a complete response (CR) receive 1 additional month of treatment beyond documentation of CR.
Drug: tamoxifen citrate
Given orally
Other Names:
  • Nolvadex
  • TAM
  • tamoxifen
  • TMX

Drug: sulindac
Given orally
Other Names:
  • Aflodac
  • Algocetil
  • Clinoril

Other: laboratory biomarker analysis
Correlative studies

Primary Outcome Measures :
  1. Percentage of Patients Failure Free at 2 Years Following Study Entry [ Time Frame: Up to 2 years ]
    Kaplan Meier estimate of failure free survival at 2 years, where failure free survival is defined as the time to relapse, progression, second malignancy, and death whichever occurs first.

  2. Percentage of Patients Experiencing a Grade 3 or Higher Adverse Event During Therapy. [ Time Frame: Up to 12 months ]
    The percentage of patients experiencing a grade 3 or higher adverse event as assessed by the National Cancer Institute Common Toxicity Terminology for Adverse Events v3.0

Secondary Outcome Measures :
  1. Percentage of Patients With Tumor Response From Imaging [ Time Frame: Baseline up to 5 years ]
    Percentage of patients with a tumor response where tumor response is assessed according to Response Evaluation Criteria in Solid Tumors (RECIST)

  2. Mean Change in Response Measured by MRI [ Time Frame: From baseline to up to 5 years ]
    The mean change in response measured by MRI. Response is assessed by the lesion size which is derived from the sum of the longest of the three orthogonal diameters (from MRI) of each target lesion.

  3. Percentage of Patients Failure Free at 2 Years by Pathological Response [ Time Frame: From enrollment to up to 2 years ]
    The failure free survival is compared by the log-rank test between patient subgroups defined by pathological response of cyclooxygenase-2 (COX-2) and estrogen/progesterone receptor expression

  4. Percentage of Patients Experiencing Short-term Endocrine Toxicity [ Time Frame: At study entry ]
    The percentage of patients experiencing short-term endocrine toxicity between treatment groups is compared using the chi-square test

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:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Histologically confirmed desmoid tumor, meeting 1 of the following criteria:

    • Newly diagnosed disease

      • Not previously treated
      • Not amenable to complete surgical resection and/or radiotherapy

        • If surgical resection was attempted, there must be gross residual disease measurable by MRI
    • Radiographically documented recurrent or progressive disease

      • No prior chemotherapy or radiotherapy for the present recurrence

        • Tumors that progressed on prior chemotherapy are allowed provided patients have not received chemotherapy for this recurrence
  • Measurable disease by gadolinium-enhanced MRI
  • No other fibroblastic lesions or fibromatoses

    • Lipofibromatosis or desmoplastic fibroma of the bone allowed
  • Performance status - Karnofsky Score 50-100% (patients over age 16)
  • Performance status - Lansky Score 50-100% (patients age 16 and under)
  • At least 8 weeks
  • Absolute neutrophil count at least 1,000/mm^3
  • Platelet count at least 100,000/mm^3 (transfusion independent)
  • Hemoglobin at least 10.0 g/dL (transfusion allowed)
  • No hemophilia
  • No von Willebrand disease
  • No other clinically significant bleeding diathesis
  • Bilirubin no greater than 1.5 times upper limit of normal (ULN)
  • Alanine aminotransferase (ALT) less than 2.5 times ULN
  • Creatinine adjusted according to age as follows:

    • No greater than 0.4 mg/dL (≤ 5 months)
    • No greater than 0.5 mg/dL (6 months -11 months)
    • No greater than 0.6 mg/dL (1 year-23 months)
    • No greater than 0.8 mg/dL (2 years-5 years)
    • No greater than 1.0 mg/dL (6 years-9 years)
    • No greater than 1.2 mg/dL (10 years-12 years)
    • No greater than 1.4 mg/dL (13 years and over [female])
    • No greater than 1.5 mg/dL (13 years to 15 years [male])
    • No greater than 1.7 mg/dL (16 years and over [male])
  • Creatinine clearance or radioisotope glomerular filtration rate at least 70 mL/min
  • No prior deep venous thrombosis
  • Electrocardiogram (EKG) normal
  • Chest x-ray normal
  • No prior significant gastrointestinal hemorrhage
  • No prior peptic ulcer disease
  • Not pregnant or nursing
  • Fertile patients must use effective nonhormonal contraception
  • No evidence of active graft-versus-host disease
  • No allergy to aspirin
  • Recovered from prior immunotherapy
  • At least 7 days since prior anticancer biologic agents
  • At least 6 months since prior allogeneic stem cell transplantation
  • More than 1 week since prior growth factors
  • No concurrent immunomodulating agents
  • No prior nonsteroidal anti-inflammatory drugs (NSAIDs) for desmoid tumor
  • More than 2 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas) and recovered
  • No concurrent anticancer chemotherapy
  • No prior estrogen antagonists for desmoid tumor
  • No concurrent hormonal contraceptives
  • No concurrent steroids except for non tumor indications (e.g., asthma or severe allergic reactions)
  • No concurrent NSAIDs for desmoid tumor

    • Occasional NSAIDs for musculoskeletal or other pain are allowed
  • Recovered from prior radiotherapy
  • No concurrent adjuvant radiotherapy
  • No concurrent participation in another COG therapeutic study

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): NCT00068419

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United States, California
Children's Oncology Group
Monrovia, California, United States, 91016
Sponsors and Collaborators
Children's Oncology Group
National Cancer Institute (NCI)
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Principal Investigator: Stephen Skapek, MD Children's Oncology Group
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Responsible Party: Children's Oncology Group Identifier: NCT00068419    
Other Study ID Numbers: ARST0321
NCI-2009-00424 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
CDR0000322260 ( Other Identifier: PDQ (Physician Data Query) )
COG-ARST0321 ( Other Identifier: Children's Oncology Group )
U10CA098543 ( U.S. NIH Grant/Contract )
First Posted: September 11, 2003    Key Record Dates
Results First Posted: December 4, 2013
Last Update Posted: February 19, 2020
Last Verified: February 2019
Additional relevant MeSH terms:
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Fibromatosis, Aggressive
Neoplasms, Fibrous Tissue
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Molecular Mechanisms of Pharmacological Action
Estrogen Antagonists
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Selective Estrogen Receptor Modulators
Estrogen Receptor Modulators
Bone Density Conservation Agents
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Anti-Inflammatory Agents
Antirheumatic Agents
Cyclooxygenase Inhibitors
Enzyme Inhibitors