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Trial record 1 of 8 for:    "Hepatocellular carcinoma (fibrolamellar variant)"
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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
Sponsor:
Information provided by (Responsible Party):
CASI Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE September 5, 2014
First Posted Date  ICMJE September 9, 2014
Last Update Posted Date September 6, 2018
Study Start Date  ICMJE October 2015
Actual Primary Completion Date June 30, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 7, 2015)
Overall Response Rate [ Time Frame: 6 months ]
6-month overall response rate (ORR rate) using RECIST v1.1 criteria
Original Primary Outcome Measures  ICMJE
 (submitted: September 5, 2014)
Progression Free Survival [ Time Frame: 6 months ]
6-month progression free survival (PFS6) rate
Change History Complete list of historical versions of study NCT02234986 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 7, 2015)
Progression Free Survival (PFS6) rate [ Time Frame: 6 months ]
Overall response rate using RECIST v 1.1 criteria
Original Secondary Outcome Measures  ICMJE
 (submitted: September 5, 2014)
Overall response rate [ Time Frame: 6 months ]
Overall response rate using RECIST v 1.1 criteria
Current Other Pre-specified Outcome Measures
 (submitted: September 4, 2018)
Frequency and severity of adverse events [ Time Frame: 6 months ]
Frequency and severity of adverse events
Original Other Pre-specified Outcome Measures
 (submitted: September 5, 2014)
Safety [ Time Frame: 6 months ]
Safety of ENMD-2076 as defined by the frequency and severity of adverse events
 
Descriptive Information
Brief Title  ICMJE A Multi-center, Open-label Study of Oral ENMD-2076 for the Treatment of Patients With Advanced Fibrolamellar Carcinoma
Official Title  ICMJE A Phase 2, Multi-center, Open-label Study of Oral ENMD-2076 for the Treatment of Patients With Advanced Fibrolamellar Carcinoma (FLC)
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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Advanced Adult Hepatocellular Carcinoma
  • Advanced Fibrolamellar Carcinoma
Intervention  ICMJE Drug: ENMD-2076
250 mg oral dose, QD for 28 day cycles
Study Arms  ICMJE Experimental: ENMD-2076
ENMD-2076, oral capsule Once daily dose 250 mg/day
Intervention: Drug: ENMD-2076
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 4, 2018)
37
Original Estimated Enrollment  ICMJE
 (submitted: September 5, 2014)
53
Actual Study Completion Date  ICMJE June 30, 2018
Actual Primary Completion Date June 30, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 99 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02234986
Other Study ID Numbers  ICMJE 2076-CL-006
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party CASI Pharmaceuticals, Inc.
Study Sponsor  ICMJE CASI Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Ken Ren, PhD CASI Pharmaceuticals, Inc.
Study Chair: Ghassan Abou-Alfa, MD MSKCC
PRS Account CASI Pharmaceuticals, Inc.
Verification Date September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP