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Inolitazone Dihydrochloride and Paclitaxel in Treating Patients With Advanced Anaplastic Thyroid 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: NCT02152137
Recruitment Status : Active, not recruiting
First Posted : June 2, 2014
Last Update Posted : May 29, 2019
National Cancer Institute (NCI)
Daiichi Sankyo, Inc.
Information provided by (Responsible Party):
Alliance for Clinical Trials in Oncology

Brief Summary:
This phase II trial studies how well inolitazone dihydrochloride (efatutazone dihydrochloride) and paclitaxel work in treating patients with anaplastic thyroid cancer that has spread to other places in the body and usually cannot be cured or controlled with treatment (advanced). Drugs used in chemotherapy, such as efatutazone dihydrochloride and paclitaxel, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Condition or disease Intervention/treatment Phase
Anaplastic Thyroid Cancer Recurrent Thyroid Cancer Drug: efatutazone Drug: paclitaxel Phase 2

Detailed Description:


I. To determine if the combination of paclitaxel and efatutazone (efatutazone dihydrochloride) improves the confirmed response rate in patients with advanced anaplastic thyroid cancer.


I. To estimate the overall survival (OS), duration of response, progression-free survival (PFS), and adverse event rates for the combination of paclitaxel and efatutazone.


I. The association of biomarkers with clinical outcome data will be assessed in an exploratory translational analysis.


Patients receive paclitaxel intravenously (IV) over 3 hours on day 1 and efatutazone dihydrochloride orally (PO) twice daily (BID) on days 1-21. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up within 28 days, every 8 weeks until disease progression, and then every 6 months for 5 years.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Study of Efatutazone, an Oral PPAR Agonist, In Combination With Paclitaxel in Patients With Advanced Anaplastic Thyroid Cancer
Study Start Date : September 2014
Actual Primary Completion Date : February 27, 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Paclitaxel

Arm Intervention/treatment
Experimental: efatutazone dihydrochloride, paclitaxel
Patients receive paclitaxel IV over 3 hours on day 1 and efatutazone dihydrochloride PO BID on days 1-21. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Drug: efatutazone
Given PO
Other Name: CS-7017

Drug: paclitaxel
Given IV
Other Name: Taxol

Primary Outcome Measures :
  1. Confirmed response rate (partial response [PR] or complete response [CR]) per Response Evaluation Criteria In Solid Tumors (RECIST) 1.1 criteria [ Time Frame: Up to 5 years ]

Secondary Outcome Measures :
  1. Overall Survival [ Time Frame: Time from study entry to death from any cause, assessed up to 12 months of follow-up ]
  2. Duration of confirmed response [ Time Frame: The time from the first documented date of confirmed response (CR or PR) to date at which progression is first documented, assessed up to 5 years ]
  3. PFS determined based on RECIST 1.1 criteria [ Time Frame: The time from study entry to the first of either disease progression or death from any cause, assessed up to 5 years ]
  4. Incidence of adverse events summarized using CTCAE version 4.0 [ Time Frame: Up to 5 years ]

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
  • Patients must have histologically or cytologically diagnosed advanced anaplastic thyroid cancer (ATC)
  • Patients must have measurable disease
  • Patients must have either metastatic (stage IVC) or locally advanced unresectable disease (stage IVB)
  • Patients should have resolution of any toxic effects of prior therapy (except alopecia) to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.0, grade 1
  • There is no limit to the number of prior lines of treatment a patient has received
  • No treatment with chemotherapy, radiation therapy, immunotherapy, biological therapy, hormonal therapy, or other thiazolidinediones (TZDs) =< 21 days before study registration
  • No prior taxane therapy =< 6 months, except as a radiosensitizer
  • No history of the following:

    • Class III or IV congestive heart failure (CHF)
    • Grade 3 or 4 thromboembolic event =< 6 months
    • Pericardial effusion =< 12 months (any grade)
    • Pericardial involvement with tumor
    • Grade 2 or higher pleural effusion =< 6 months
  • No current symptomatic, untreated, or uncontrolled brain metastases present
  • No major surgery =< 14 days prior to registration
  • No grade 2 or higher neuropathy
  • No known history of severe hypersensitivity reactions to any of the components of efatutazone or paclitaxel formulations
  • Not pregnant and not nursing; women of childbearing potential only, a negative pregnancy test done =< 7 days prior to registration is required
  • Patients with diabetes mellitus requiring concurrent treatment with insulin or thiazolidinedione (TZD) oral agents are not eligible
  • Patients with known hypersensitivity to any TZD oral agents are not eligible
  • Eastern Cooperative Oncology Group (ECOG) performance status =< 2
  • Absolute neutrophil count (ANC) >= 1,500/mm^3
  • Platelet count >= 100,000/mm^3
  • Creatinine =< 1.5 x upper limit of normal (ULN) mg/dL OR calculated (calc.) creatinine clearance >= 60 mL/min
  • Bilirubin =< 1.5 x ULN
  • Aspartate aminotransferase (AST) =< 2.5 x ULN
  • Although they will not be considered formal eligibility (exclusion) criteria, physicians should recognize that the following may seriously increase the risk to the patient entering this protocol:

    • Psychiatric illness which would prevent the patient from giving informed consent
    • Medical condition such as uncontrolled infection (including human immunodeficiency virus [HIV]), uncontrolled diabetes mellitus or cardiac disease which, in the opinion of the treating physician, would make this protocol unreasonably hazardous for the patient
    • Patients with a "currently active" second malignancy other than non-melanoma skin cancers; patients are not considered to have a "currently active" malignancy if they have completed therapy and are free of disease for >= 3 years; there is an exception for patients with a history of well differentiated thyroid cancer that has progressed to anaplastic thyroid cancer
    • Patients who cannot swallow oral formulations of the agent(s)
    • Women and men of reproductive potential should agree to use an appropriate method of birth control throughout their participation in this study ; appropriate methods of birth control include abstinence, oral contraceptives, implantable hormonal contraceptives or double barrier method (diaphragm plus condom)
    • Efatutazone is metabolized by cytochrome P450, family 3, subfamily A, polypeptide 4/5 (CYP3A4/5), and inhibits CYP2C8, 2C9, 2C19, and 3A4, and is a substrate of P-glycoprotein (PgP) and breast cancer resistance protein (BCRP)

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): NCT02152137

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Sponsors and Collaborators
Alliance for Clinical Trials in Oncology
National Cancer Institute (NCI)
Daiichi Sankyo, Inc.
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Study Chair: Robert Smallridge, M.D. Mayo Clinic

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Alliance for Clinical Trials in Oncology Identifier: NCT02152137     History of Changes
Other Study ID Numbers: A091305
U10CA031946 ( U.S. NIH Grant/Contract )
U10CA180821 ( U.S. NIH Grant/Contract )
NCI-2014-00686 ( Registry Identifier: NCI Clinical Trials Reporting Office )
First Posted: June 2, 2014    Key Record Dates
Last Update Posted: May 29, 2019
Last Verified: May 2019
Additional relevant MeSH terms:
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Thyroid Neoplasms
Thyroid Carcinoma, Anaplastic
Thyroid Diseases
Endocrine System Diseases
Endocrine Gland Neoplasms
Neoplasms by Site
Head and Neck Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Albumin-Bound Paclitaxel
Antineoplastic Agents, Phytogenic
Antineoplastic Agents
Tubulin Modulators
Antimitotic Agents
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action