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Phase 1/2, Open-label, Dose-escalation Study of IONIS-STAT3Rx, Administered to Patients With Advanced Cancers

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.
ClinicalTrials.gov Identifier: NCT01563302
Recruitment Status : Completed
First Posted : March 26, 2012
Last Update Posted : June 25, 2018
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Brief Summary:
This is a Phase 1/2, open-label, dose-escalation, dose-expansion study for the treatment of patients with advanced cancers. Eligible patients with DLBCL or other advanced lymphomas will be enrolled into the dose-expansion cohort.

Condition or disease Intervention/treatment Phase
Advanced Cancers DLBCL Lymphoma Drug: IONIS-STAT3Rx Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 64 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Study of ISIS 481464, an Antisense Oligonucleotide Inhibitor of STAT3, Administered to Patients With Advanced Cancers
Actual Study Start Date : February 27, 2012
Actual Primary Completion Date : March 23, 2016
Actual Study Completion Date : March 23, 2016

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Group 1
Three-hour IV infusions on Cycle 0 Days 1, 3, 5, and weekly three-hour IV infusions in Cycles 1 and beyond, on Days 1, 8, and 15 of each cycle.
Other Name: ISIS 481464

Primary Outcome Measures :
  1. Safety of IONIS-STAT3Rx in patients with Advanced Cancers [ Time Frame: Approximately 28 days after last dose of IONIS -STAT3Rx ]
  2. Maximum-tolerated dose (MTD) of IONIS-STAT3Rx in patients with Advanced Cancers. [ Time Frame: Approximately 28 Days ]
    Highest dose level at which no more than 1 out of 6 patients develops a DLT

Secondary Outcome Measures :
  1. Clinical activity of IONIS-STAT3Rx [ Time Frame: Approximately 28 Days after last dose of IONIS-STAT3Rx ]
    Measured by RECIST in the study population who have measurable disease or relevant disease specific response criteria

  2. Pharmacokinetics-Cmax [ Time Frame: Approximately 28 days after last dose of IONIS-STAT3Rx ]
    Maximum observed drug concentration (Cmax)

  3. STAT3 and other biomarkers [ Time Frame: Approximately 28 days after last dose of IONIS-STAT3Rx ]
    The effect of treatment with IONIS-STAT3Rx on phospho-STAT3 and other biomarkers

  4. Pharmacokinetics - Tmax [ Time Frame: Approximately 28 days after last dose of IONIS-STAT3Rx ]
    Time taken to reach Cmax

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

Inclusion Criteria:

  • Aged 18 years or older
  • Tumors that are relapsed or refractory to at least 1 prior anti-cancer systemic therapy and for which no standard therapy exists
  • Expansion cohort only: Advanced lymphoma confirmed by histopathology
  • Measurable or evaluable disease according to RECIST for solid tumors or according to IWRC for NHL tumors
  • ECOG Performance Status less than or equal to 2
  • Life expectancy greater than 12 weeks in the opinion of the Investigator

Exclusion Criteria:

  • Any active or uncontrolled infection
  • NYHA Grade II or greater congestive heart failure
  • History of myocardial infarction within 6 months prior to screening
  • Prior radiation therapy, chemotherapy, hormonal therapy, or immunotherapy within 4 weeks prior to screening or 5 half-lives of the therapy, whichever is shorter

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 ClinicalTrials.gov identifier (NCT number): NCT01563302

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United States, Arizona
Mayo Clinic Arizona
Scottsdale, Arizona, United States, 85259
United States, California
Moores UC San Diego Cancer Center
La Jolla, California, United States, 92093
United States, Connecticut
Yale Cancer Center
New Haven, Connecticut, United States, 06510
United States, Georgia
Blood and Marrow Transplant Group of Georgia at Northside Hospital
Atlanta, Georgia, United States, 30342
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, Texas
Mary Crowley Cancer Research Centers
Dallas, Texas, United States, 75201
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
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Study Director: Steve Hughes, MD Ionis Pharmaceuticals, Inc.
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01563302    
Other Study ID Numbers: ISIS 481464-CS1
First Posted: March 26, 2012    Key Record Dates
Last Update Posted: June 25, 2018
Last Verified: June 2018
Keywords provided by Ionis Pharmaceuticals, Inc.:
Advanced Cancers
Advanced Lymphoma
Additional relevant MeSH terms:
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