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Itacitinib in Treating Patients With Refractory Metastatic/Advanced Sarcomas

Phase 1
Terminated
Conditions
Metastatic Leiomyosarcoma
Metastatic Undifferentiated Pleomorphic Sarcoma
Refractory Undifferentiated Pleomorphic Sarcoma
Advanced Synovial Sarcoma
Advanced Undifferentiated Pleomorphic Sarcoma
Advanced Leiomyosarcoma
Metastatic Synovial Sarcoma
Refractory Leiomyosarcoma
Refractory Round Cell Liposarcoma
Refractory Soft Tissue Sarcoma
Interventions
Other: Laboratory Biomarker Analysis
Registration Number
NCT03670069
Lead Sponsor
Fred Hutchinson Cancer Center
Brief Summary

This pilot phase I trial studies how well itacitinib works in treating patients with sarcomas that do not respond to treatment (refractory) and have spread to other parts of the body (advanced/metastatic). Itacitinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed Description

OUTLINE:

Patients receive itacitinib orally (PO) once daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 7 and 30 days, every 12 weeks from baseline for up to 1 year, and then every 6 months thereafter.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
27
Inclusion Criteria
  • Subjects >= 18 years old

  • Must have a histologically confirmed diagnosis of sarcoma with one of the following subtypes:

    • Cohort 1: Leiomyosarcoma
    • Cohort 2: Undifferentiated pleiomorphic sarcoma
    • Cohort 3: Synovial sarcoma or myxoid/round cell liposarcoma
    • Cohort 4: Chondrosarcoma (all subtypes of chondrosarcoma are allowed)
  • Subjects enrolling to cohorts 1, 2, or 3 must have received at least two prior lines of systemic therapy. Subjects enrolling to cohort 4 only may have received any number of prior lines of systemic therapy or may be treatment naïve

  • All ongoing toxicities related to prior therapies must be resolved to grade 1 or better (except alopecia)

  • Subjects must have one or more measurable lesions, as determined by Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1 assessed by computed tomography (CT) or magnetic resonance imaging (MRI)

  • Subjects must have at least one superficial lesion accessible for multiple biopsies; the tumor being biopsied cannot have been previously targeted for radiation therapy or have previously received intra-lesional treatment

    * NOTE: Superficial lesions previously targeted with radiation therapy that have demonstrated significant new growth via radiological imaging may be targeted for biopsy, with sponsor-investigator approval.

  • Total bilirubin level =< 1.5 x the upper limit of normal (ULN) range mg/dL

  • Aspartate aminotransferase (AST) =< 2.5 x ULN and alanine aminotransferase (ALT) levels =< 2.5 x ULN

  • Alkaline phosphatase < 2.5 x ULN

  • Serum creatinine =< 1.5 x ULN

  • Calculated creatinine clearance >= 30 mL/min using the Cockcroft-Gault formula may be included

  • Absolute neutrophil count (ANC) >= 1.5 × 10^9/L

  • Platelet count >= 100 x 10^9/L; transfusion is permitted as clinically indicated

  • Hemoglobin >= 9 g/dL

    * Transfusion is permitted as clinically indicated

  • Subjects must have a life expectancy >= 6 months, as determined by the treating physician

  • Eastern Cooperative Oncology Group (ECOG) performance status =< 2 or Karnofksy performance status >= 60

  • Male or non-pregnant and non-breast feeding female:

    • Females of child-bearing potential must agree to use highly effective contraception without interruption from initiation of therapy and while on study medication and have a negative serum pregnancy test (beta - human chorionic gonadotropin [hCG]) result at screening and agree to ongoing pregnancy testing during the course of the study, and at the end of study treatment; a highly effective method of contraception is defined as one that results in a low failure rate (that is, < 1% per year), when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine contraceptive devices, sexual abstinence, or a vasectomized partner
    • Male subjects must practice abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study
  • Ability to understand and sign informed consent document

  • Willingness and ability to comply with scheduled visits, laboratory tests, and other study procedures

Exclusion Criteria
  • Known active, uncontrolled, or symptomatic central nervous system (CNS) metastases; a subject with controlled and asymptomatic CNS metastases may participate in this study; as such, the subject must have completed any prior treatment for CNS metastases >= 28 days (including radiotherapy and/or surgery) prior to the start of treatment in this study and should not be receiving chronic corticosteroid therapy for CNS metastases; subjects with known CNS metastases must be confirmed radiographically stable by at least one imaging study, at least 28 days from last treatment
  • Receipt of any type of cytotoxic, biologic, or other systemic anticancer therapy (including investigational) within 2 weeks of enrollment
  • Prior treatment with a drug targeting JAK1, JAK1/2 or STAT3 inhibitor; Food and Drug Administration (FDA) approved small molecule tyrosine kinase inhibitors (TKIs) not specifically designed to target this pathway are okay (e.g. pazopanib, sunitinib, sorafenib)
  • Known, active drug or alcohol abuse
  • Pregnant or lactating females
  • Active or recent infection requiring systemic anti-infective treatment that was completed =< 14 days prior to enrollment (with the exception of uncomplicated urinary tract infection or upper respiratory infection)
  • Uncontrolled or concurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  • Oral steroid usage within =< 14 days prior to enrollment
  • Known inflammatory or autoimmune disease which requires patient to occasionally require high dose oral steroids
  • Subjects with known, active human immunodeficiency virus (HIV) infection (subjects with undetectable viral load and normal CD4+ T-cell count are permitted)
  • Inability to swallow food or tablets, or significant gastrointestinal disorder that, in the opinion of the investigator, could interfere with absorption of the study drug
  • Previous reaction to any component of itacitinib or known hypersensitivity to the active substance or any of the excipients
  • Subjects with a sarcoma which has other, defined treatments or biology distinctly different from those of soft tissue sarcomas in general; including, but not limited to, Ewing's sarcoma, rhabdomyosarcoma, gastrointestinal stromal tumors, Kaposi's sarcoma, Wilm's tumor

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Treatment (itacitinib)Laboratory Biomarker AnalysisPatients receive itacitinib PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Treatment (itacitinib)ItacitinibPatients receive itacitinib PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Primary Outcome Measures
NameTimeMethod
Difference in the percentage of cells which are immune inhibitory (CD11B+, CD163+) macrophages from pre-treatment to first post-treatment biopsyFrom baseline to 2 years

Evaluation of the change in percentages of cells against the null hypothesis of no difference will be performed using a 1-sided t-test at the 0.05 level.

Secondary Outcome Measures
NameTimeMethod
Incidence of adverse eventsUp to 2 years

Will be graded according to Common Terminology Criteria for Adverse Events version 5.0. The frequency and severity of toxicities will be evaluated by proportions and associated 95% confidence intervals.

Progression-free survival rateAt 6 months

Assessed by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1. Distributions and percentages at landmark times for time to event outcomes will be estimated using the method of Kaplan-Meier. Confidence intervals about medians will be estimated using the method of Brookmeyer-Crowley. With 12 LMS patients, binary proportions can be estimated to within 29% with 95% confidence. With 8 (SS or MRCL) and PUS, respectively, binary proportions can be estimated to within 36% with 95% confidence.

Median overall survivalAt 12 months

Distributions and percentages at landmark times for time to event outcomes will be estimated using the method of Kaplan-Meier. Confidence intervals about medians will be estimated using the method of Brookmeyer-Crowley. With 12 LMS patients, binary proportions can be estimated to within 29% with 95% confidence. With 8 (SS or MRCL) and PUS, respectively, binary proportions can be estimated to within 36% with 95% confidence.

Clinical benefit rate (complete response [CR]+ partial response [PR]+stable disease [SD])At 12 weeks

CR and PR will be defined as per RECIST 1.1 Distributions and percentages at landmark times for time to event outcomes will be estimated using the method of Kaplan-Meier. Confidence intervals about medians will be estimated using the method of Brookmeyer-Crowley. With 12 LMS patients, binary proportions can be estimated to within 29% with 95% confidence. With 8 (SS or MRCL) and PUS, respectively, binary proportions can be estimated to within 36% with 95% confidence.

Trial Locations

Locations (1)

Fred Hutch/University of Washington Cancer Consortium

🇺🇸

Seattle, Washington, United States

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