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

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: NCT02234986
Recruitment Status : Completed
First Posted : September 9, 2014
Last Update Posted : September 6, 2018
Information provided by (Responsible Party):
CASI Pharmaceuticals, Inc.

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Advanced Adult Hepatocellular Carcinoma Advanced Fibrolamellar Carcinoma Drug: ENMD-2076 Phase 2

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 37 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Multi-center, Open-label Study of Oral ENMD-2076 for the Treatment of Patients With Advanced Fibrolamellar Carcinoma (FLC)
Study Start Date : October 2015
Actual Primary Completion Date : June 30, 2018
Actual Study Completion Date : June 30, 2018

Resource links provided by the National Library of Medicine

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

Primary Outcome Measures :
  1. Overall Response Rate [ Time Frame: 6 months ]
    6-month overall response rate (ORR rate) using RECIST v1.1 criteria

Secondary Outcome Measures :
  1. Progression Free Survival (PFS6) rate [ Time Frame: 6 months ]
    Overall response rate using RECIST v 1.1 criteria

Other Outcome Measures:
  1. Frequency and severity of adverse events [ Time Frame: 6 months ]
    Frequency and severity of adverse events

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 99 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

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): NCT02234986

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United States, California
CASI Site 03
San Francisco, California, United States, 94158
United States, Colorado
CASI Site 04
Aurora, Colorado, United States, 80045
United States, Massachusetts
CASI Site 02
Boston, Massachusetts, United States, 01003
United States, New York
Casi Site 01
New York, New York, United States, 10065
United States, Texas
CASI Site 05
Dallas, Texas, United States, 75231
Sponsors and Collaborators
CASI Pharmaceuticals, Inc.
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Study Director: Ken Ren, PhD CASI Pharmaceuticals, Inc.
Study Chair: Ghassan Abou-Alfa, MD MSKCC
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: CASI Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02234986    
Other Study ID Numbers: 2076-CL-006
First Posted: September 9, 2014    Key Record Dates
Last Update Posted: September 6, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by CASI Pharmaceuticals, Inc.:
Advanced Fibrolamellar Carcinoma
Additional relevant MeSH terms:
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Carcinoma, Hepatocellular
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Liver Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Liver Diseases