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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2016 by CASI Pharmaceuticals, Inc.
Sponsor:
Information provided by (Responsible Party):
CASI Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT02234986
First received: September 5, 2014
Last updated: March 19, 2016
Last verified: March 2016
  Purpose
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 Intervention Phase
Advanced Adult Hepatocellular Carcinoma
Advanced Fibrolamellar Carcinoma
Drug: ENMD-2076
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: 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)

Resource links provided by NLM:


Further study details as provided by CASI Pharmaceuticals, Inc.:

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


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


Other Outcome Measures:
  • Safety [ Time Frame: 6 months ]
    Safety of ENMD-2076 as defined by the frequency and severity of adverse events


Estimated Enrollment: 29
Study Start Date: October 2015
Estimated Study Completion Date: June 2019
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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
  Eligibility

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

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02234986

Contacts
Contact: David Markowitz, MS davidm@casipharmaceuticals.com

Locations
United States, California
CASI Site 03 Recruiting
San Francisco, California, United States, 94158
United States, Colorado
CASI Site 04 Recruiting
Aurora, Colorado, United States, 80045
United States, Massachusetts
CASI Site 02 Recruiting
Boston, Massachusetts, United States, 01003
United States, New York
Casi Site 01 Recruiting
NYC, New York, United States, 10065
United States, Texas
CASI Site 05 Recruiting
Dallas, Texas, United States, 75231
Sponsors and Collaborators
CASI Pharmaceuticals, Inc.
Investigators
Study Director: Ken Ren, PhD CASI Pharmaceuticals, Inc.
Study Chair: Ghassan Abou-Alfa, MD MSKCC
  More Information

Responsible Party: CASI Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02234986     History of Changes
Other Study ID Numbers: 2076-CL-006
Study First Received: September 5, 2014
Last Updated: March 19, 2016
Individual Participant Data  
Plan to Share IPD: Undecided

Keywords provided by CASI Pharmaceuticals, Inc.:
Advanced Fibrolamellar Carcinoma

Additional relevant MeSH terms:
Carcinoma
Carcinoma, Hepatocellular
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Adenocarcinoma
Liver Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Liver Diseases

ClinicalTrials.gov processed this record on May 25, 2017