Safety, Pharmacokinetics and Preliminary Anti-Tumor Activity of Intravenous TKM-080301 in Subjects With Advanced Hepatocellular Carcinoma

NCT ID: NCT02191878

Last Updated: 2019-01-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

43 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-06-30

Study Completion Date

2016-07-31

Brief Summary

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This study is an open-label, multi-center, phase 1, dose escalation study with a phase 2 expansion cohort to determine the safety, pharmacokinetics and preliminary anti-tumor activity of intravenous TKM-080301 in subjects with advanced hepatocellular carcinoma (HCC).

This study is being done to:

* Test the safety and tolerability of TKM-080301 in subjects with advanced hepatocellular carcinoma
* Find the highest dose of TKM-080301 that can be given without causing side effects, called the maximum tolerated dose (MTD).
* Provide a preliminary assessment of anti-tumor activity of TKM-080301

Detailed Description

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Study design: Open label, multi-center, 3 + 3 dose-escalation study with an expansion cohort at the maximum tolerated dose (MTD) to investigate safety, tolerability, PK, and preliminary anti-tumor activity of TKM 080301 in subjects with HCC.

Sequential cohorts of 3 to 6 subjects will receive escalating doses of TKM 080301 according to a pre-specified dose escalation scheme. Assessment of dose-limiting toxicities (DLTs) will be made during Cycle 1 to determine the maximum tolerated dose (MTD). Once the MTD level is established, approximately 20 subjects will be enrolled in an expansion cohort to further confirm the safety and tolerability of TKM-080301 at the MTD.

Study Population: A minimum of 9 and up to approximately 18 adult male or female subjects with histologically or cytologically confirmed metastatic or locally advanced inoperable HCC and a life expectancy of 3 months or more are planned in the dose escalation phase. Approximately 20 subjects are planned in the expansion cohort.

Study Treatment: TKM-080301 will be administered by intravenous (IV) infusion, once weekly for 3 consecutive weeks followed by a 1 week rest period. This 28-day treatment period constitutes 1 cycle.

Subjects who demonstrate clinical benefit without progression per RECIST 1.1 guidelines may receive treatment beyond 6 cycles if the Investigator considers it is in the best interest of the subject, and only with the approval of the Medical Monitor. Subjects would then continue TKM 080301 therapy until withdrawal of consent, disease progression or unacceptable toxicity occurs.

Pharmacokinetics (PK) Subjects will undergo blood sample collection for PK analysis during cycles 1 and 2.

Study Duration: Each treatment cycle will have duration of 28 days and each subject will typically receive up to 6 cycles of treatment. The total duration of the study is expected to be approximately 28 months.

Conditions

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Hepatocellular Carcinoma Hepatoma Liver Cancer, Adult Liver Cell Carcinoma, Adult

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Phase 1 Escalation / Phase 2 Expansion

Phase 1 - dose escalation with intravenous infusion of TKM-080301 to determine MTD.

Phase 2 - dose expansion at the MTD.

Group Type EXPERIMENTAL

TKM-080301

Intervention Type DRUG

TKM-080301 intravenous infusion

Interventions

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TKM-080301

TKM-080301 intravenous infusion

Intervention Type DRUG

Other Intervention Names

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PLK1-HCC

Eligibility Criteria

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Inclusion Criteria

* Child-Pugh class of A
* Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
* Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤5.0 × ULN
* Total bilirubin ≤3.0 mg/dL
* Platelets ≥75,000 /mL
* International Normalized Ratio (INR) ≤1.7
* Subjects must meet the protocol-defined criteria for both hepatitis B virus (HBV) and hepatitis C virus (HCV) status

Exclusion Criteria

* History of significant cardiovascular disease will be excluded
* History of liver transplant.
* Diagnosis of fibrolamellar HCC or tumors of mixed histology.
* Subjects known to be positive for Human immunodeficiency virus (HIV) infection.
* Known central nervous system (CNS) or brain metastases.
* Poorly controlled ascites and/or requirement for therapeutic paracentesis more frequently than once every 3 months.
* Symptomatic encephalopathy within 3 months prior to the first dose of TKM-080301 and/or requirement for medication for encephalopathy.
* Esophageal variceal bleeding within 2 weeks prior to the first dose of TKM-080301.
* Asthma or chronic obstructive pulmonary disease (COPD) requiring daily medication.
* Prior therapy with nitrosoureas or mitomycin within 6 weeks prior to the first dose of TKM-080301.
* Prior therapy with any biologic chemotherapeutic or investigational drug within 5 half-lives or 3 weeks, whichever is longer prior to the first dose of TKM 080301.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Arbutus Biopharma Corporation

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Mark Kowalski, M.D., Ph.D.

Role: STUDY_DIRECTOR

Tekmira Pharmaceuticals

Locations

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Arizona Clinical Research Center

Tucson, Arizona, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

Kansas City Research Institute

Kansas City, Missouri, United States

Site Status

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status

Mary Crowley Cancer Research Centers

Dallas, Texas, United States

Site Status

Princess Margaret Hospital

Toronto, Ontario, Canada

Site Status

Queen Mary Hospital

Hong Kong, , China

Site Status

National University Hospital

Singapore, , Singapore

Site Status

Seoul National University Hospital

Seoul, Gyeonggi-do, South Korea

Site Status

Samsung Medical Center

Seoul, Gyeonggi-do, South Korea

Site Status

ASAN Medical Center

Seoul, Gyeonggi-do, South Korea

Site Status

Severence Hospital, Yonsei, University Health System

Seoul, , South Korea

Site Status

National Taiwan University Hospital

Taipei, , Taiwan

Site Status

Taipei Medical University Hospital, Shuang-Ho Hospital

Taipei, , Taiwan

Site Status

Taipei Veterans General Hospital

Taipei, , Taiwan

Site Status

Countries

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United States Canada China Singapore South Korea Taiwan

References

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El Dika I, Lim HY, Yong WP, Lin CC, Yoon JH, Modiano M, Freilich B, Choi HJ, Chao TY, Kelley RK, Brown J, Knox J, Ryoo BY, Yau T, Abou-Alfa GK. An Open-Label, Multicenter, Phase I, Dose Escalation Study with Phase II Expansion Cohort to Determine the Safety, Pharmacokinetics, and Preliminary Antitumor Activity of Intravenous TKM-080301 in Subjects with Advanced Hepatocellular Carcinoma. Oncologist. 2019 Jun;24(6):747-e218. doi: 10.1634/theoncologist.2018-0838. Epub 2018 Dec 31.

Reference Type DERIVED
PMID: 30598500 (View on PubMed)

Other Identifiers

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TKM-HCC-001

Identifier Type: -

Identifier Source: org_study_id

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