A Multi-center, Open-label Study of Oral ENMD-2076 for the Treatment of Patients With Advanced Fibrolamellar Carcinoma

September 4, 2018 updated by: CASI Pharmaceuticals, Inc.

A Phase 2, Multi-center, Open-label Study of Oral ENMD-2076 for the Treatment of Patients With Advanced Fibrolamellar Carcinoma (FLC)

The purpose of the study is to determine whether once-daily dosing with ENMD-2076 will be a safe and effective treatment in patients with FLC. Safety will be measured by looking at the adverse events that may happen and the efficacy will look at the progression of the disease over time.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Primary Objective:

• To determine the 6-month progression free survival (PFS6) rate when patients with advanced fibrolamellar carcinoma (FLC) are treated with daily oral ENMD 2076

Secondary Objectives:

  • To evaluate the overall response rate using RECIST v 1.1 criteria when patients with FLC are treated with daily oral ENMD 2076.
  • To evaluate the median Progression Free Survival (PFS), Time to Progression (TTP), and Overall Survival (OS).
  • To determine the safety of ENMD-2076 as defined by the frequency and severity of adverse events when patients with FLC are treated with daily oral ENMD-2076

Study Type

Interventional

Enrollment (Actual)

37

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • San Francisco, California, United States, 94158
        • CASI Site 03
    • Colorado
      • Aurora, Colorado, United States, 80045
        • CASI Site 04
    • Massachusetts
      • Boston, Massachusetts, United States, 01003
        • CASI Site 02
    • New York
      • New York, New York, United States, 10065
        • Casi Site 01
    • Texas
      • Dallas, Texas, United States, 75231
        • CASI Site 05

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 99 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Histologically or cytologically confirmed advanced fibrolamellar carcinoma (FLC).
  • All forms of prior local therapy are allowed as long as patients have either a target lesion, which has not been treated with local therapy and/or the target lesion(s) within the field of the local-regional therapy has shown an increase of ≥ 20% in size. Local-regional therapy must be completed at least 4 weeks prior to the baseline CT scan. Local therapies including chemoembolization do not count as prior systemic therapy.
  • Are at least 4 weeks from major surgery and recovered.
  • At least one measureable lesion by RECIST 1.1.
  • Male or non-pregnant, non-breastfeeding female at least 18 years of age. Patients aged at 12~18 years may be recruited but only at the site principle investigator's request and subject to IRB approval.
  • Has a pre-study echocardiogram or multi-gated acquisition (MUGA) scan with an actual left ventricular ejection fraction of greater than or equal to the institutional lower limit of normal within one month of initiating therapy.

  • Have clinically acceptable laboratory screening results within certain limits specified below:

    • AST and ALT ≤ 5 times upper limit of normal (ULN)
    • Total bilirubin ≤ 3.0 x ULN
    • Creatinine ≤ 1.5 x ULN or Cr Cl > 60 cc/min
    • Absolute neutrophil count ≥ 1500 cells/mm3
    • Platelets ≥ 50,000/mm3
  • Have an ECOG performance status of 0-2 for ≥ 16 years of age and a Lansky performance status of 70-100 for < 16 years of age
  • Women and men of child producing potential must agree to use effective contraceptive methods prior to study entry, during study participation, and for at least 30 days after the last administration of study medication. A serum pregnancy test within 72 hours prior to the initiation of therapy will be required for women of childbearing potential.
  • Have the ability to understand the requirements of the study, provide written informed consent, which includes authorization for release of protected health information, abide by the study restrictions, and agree to return for the required assessments.

Exclusion Criteria:

  • Have active, acute, or chronic clinically significant infections or bleeding within the last 6 months or previous thromboembolic or hemorrhagic events during anti angiogenic therapy.
  • Have uncontrolled hypertension (systolic blood pressure greater than 150 or diastolic blood pressure greater than 100) or history of congestive heart failure (AHA Grade 2 or higher).
  • Have active angina pectoris, stroke or recent myocardial infarction (within 6 months).
  • Have uncontrolled chronic atrial fibrillation.
  • Have chronic atrial fibrillation or QTc interval corrected for heart rate of greater than 470 msec in adults and 450 msec in pediatrics (< 18 years).
  • Have additional uncontrolled serious medical or psychiatric illness that in the point of view of the investigator can render the patient unable to receive therapy or make it unsafe to receive therapy.
  • Require treatment with any of the exclusionary medications listed in Appendix D.
  • Known untreated or unstable CNS metastatic disease.
  • Have persistent 2+ protein by urinalysis (patients with 2+ proteinuria that have a spot protein:creatinine ratio of less than 0.3 may be enrolled) or a history of nephrotic syndrome.
  • Subjects with history of another primary cancer, with the exception of: a) curatively resected non-melanoma skin cancer; b) curatively treated cervical carcinoma in situ; or c) other primary solid tumor with no known active disease present in the opinion of the investigator will not affect patient outcome in the setting of current FLC diagnosis.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: ENMD-2076
ENMD-2076, oral capsule Once daily dose 250 mg/day
Drug: ENMD-2076
250 mg oral dose, QD for 28 day cycles

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall Response Rate
Time Frame: 6 months
6-month overall response rate (ORR rate) using RECIST v1.1 criteria
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Progression Free Survival (PFS6) rate
Time Frame: 6 months
Overall response rate using RECIST v 1.1 criteria
6 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency and severity of adverse events
Time Frame: 6 months
Frequency and severity of adverse events
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CASI Pharmaceuticals, Inc.

Investigators

  • Study Director: Ken Ren, PhD, CASI Pharmaceuticals, Inc.
  • Study Chair: Ghassan Abou-Alfa, MD, MSKCC

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2015

Primary Completion (ACTUAL)

June 30, 2018

Study Completion (ACTUAL)

June 30, 2018

Study Registration Dates

First Submitted

September 5, 2014

First Submitted That Met QC Criteria

September 5, 2014

First Posted (ESTIMATE)

September 9, 2014

Study Record Updates

Last Update Posted (ACTUAL)

September 6, 2018

Last Update Submitted That Met QC Criteria

September 4, 2018

Last Verified

September 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2076-CL-006

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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