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Trial record 16 of 611 for:    hepatic steatosis | Non-Alcoholic Steatohepatitis

The Impact of Deferasirox on Non-Alcoholic-Steatohepatitis (DEFINE)

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. Identifier: NCT01278056
Recruitment Status : Completed
First Posted : January 17, 2011
Last Update Posted : July 20, 2012
Estimate, GmbH
University of Magdeburg
Information provided by (Responsible Party):
Crolll Gmbh

Brief Summary:

This is a Phase I/II open-label uncontrolled, prospective study to assess the clinical and biological effects of Deferasirox (ICL 670, Exjade®) in patients with NASH and increased iron storage / distribution of iron on liver function and liver histology.

NASH is defined clinically and histologically by elevated liver enzymes, signs of hepatic steatosis on ultrasound and magnetic resonance imaging, impaired liver function as expressed by functional breath tests, and significantly altered liver histology.

Patients will be treated in a phase I and phase II part for either 12 or 48 weeks.

Both study parts have different endpoints: in phase I the side effect profile will be evaluated while in phase II the therapeutic response will be tested. Accordingly, measures will be different.

Approximately 10 patients in phase I and 50 patients in phase II will be enrolled according to sample size calculations.

The design is an "adaptive" Two-stage design, allowing to minimize the number of patients included into the trial as well as to introduce corrections for the second stage.

Condition or disease Intervention/treatment Phase
Non-alcoholic Steatohepatitis Increased Iron Storage / Disturbed Distribution Drug: Exjade Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: The Impact of Deferasirox on Non-Alcoholic-Steatohepatitis (NASH) - a Prospective Open Label Phase I/II Trial
Study Start Date : March 2010
Actual Primary Completion Date : March 2012
Actual Study Completion Date : July 2012

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron
Drug Information available for: Deferasirox

Arm Intervention/treatment
Experimental: Exjade Drug: Exjade
Two dose escalating cohorts of oral administration in Phase I. Phase II: oral administration of the maximum tolerated dose.
Other Name: Deferasirox, ICL670

Primary Outcome Measures :
  1. Safety and tolerability of deferasirox in all patients (Phase I) [ Time Frame: Phase I: 12 weeks of treatment ]
    Safety and tolerability assessments will consist of evaluating (serious) adverse events, laboratory parameters including hematology, chemistry; vital signs and physical examinations according to CTC.

  2. Changes in liver histology in all patients (Phase II) [ Time Frame: Phase II: 48 weeks of treatment ]
    A decrease in the NASH activity score (NAS) of ≥1 compared to baseline will be classified as response, an unchanged score or an increase in NAS will be judged as non-response.

Secondary Outcome Measures :
  1. Phase I: e.g. changes in liver enzymes, serum ferritin, and hemoglobin levels [ Time Frame: Phase I: 12 weeks of treatment ]
  2. Phase II: e.g. changes in MRI and histology based assessment of hepatic steatosis, fibrosis and iron content [ Time Frame: Phase II: 48 weeks of treatment ]

Information from the National Library of Medicine

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

Inclusion Criteria (shortened):

  • Patients with elevated liver enzymes
  • Elevated serum ferritin (females > 300 ng/ml, males > 450 ng/ml)
  • Liver Histology consistent with a diagnosis of NASH

Exclusion Criteria (shortened):

  • Alcohol intake > 140 g/week
  • Established liver cirrhosis Child Pugh B or C
  • Copresence of other causes of chronic liver disease
  • Anemia < 10 g/dl
  • Any elevation of liver enzymes > 5 ULN (ALAT, ASAT, g-GT), > 2.5 ULN (other), > 1.5 (Bilirubin)
  • Serum creatinine > 1.4 mg/dl or Ccr < 60 ml/min
  • Hemochromatosis
  • Known allergy or contraindication to the administration of Deferasirox
  • Sexually active pre-menopausal female patients who are unable to use a highly effective method of birth control. An exception is made for those who have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation.
  • Patients with impaired coagulation
  • History of blood transfusion during the 6 months prior to study entry
  • Oral iron supplementation within the last 4 weeks of study entry
  • Treatment with phlebotomy within 2 weeks of screening visit
  • Desferal treatment within 1 month of the screening visit
  • Patients currently or previously treated with deferiprone or Deferasirox
  • Presence of a surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any study drug
  • Positive HIV serology
  • Patients with active inflammatory diseases that may interfere with the accurate measurement of serum ferritin
  • Patients with a diagnosis of a clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation
  • Pregnant or breast feeding patients
  • Patients treated with systemic investigational drug within 4 weeks prior or with topical investigational drug within 7 days prior to the screening visit
  • Medications with proven or suspected influence on NASH such as glitazones, statins, or metformin are no exclusion criteria for study entry (insulin is not regarded to interfere with NASH).

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 identifier (NCT number): NCT01278056

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Balingen/Hechingen, Germany, 72336
Charité, Virchow Klinikum
Berlin, Germany, 13353
Klinikum der J. W. Goethe-Universität, Med. Klinik I
Frankfurt, Germany, 60590
Universitätsklinikum Halle, Klinik & Poliklinik für Innere Medizin I
Halle, Germany, 06097
Universitätsklinikum des Saarlandes
Homburg/Saar, Germany, 66421
Universitätsklinikum Magdeburg, Klinik für Gastroenterologie, Hepatologie und Infektiologie
Magdeburg, Germany, 39120
Universitätsklinikum Mainz, I. Medizinische Klinik und Poliklinik
Mainz, Germany, 55131
Universitätsklinikum Regensburg, Klinik und Poliklinik für Innere Med. I
Regensburg, Germany, 93042
Universitätsklinikum Tübingen, Medizinische Klinik IV
Tübingen, Germany, 72076
Sponsors and Collaborators
Crolll Gmbh
Estimate, GmbH
University of Magdeburg
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Principal Investigator: Gerhard Treiber, PD Dr. med.

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Responsible Party: Crolll Gmbh Identifier: NCT01278056     History of Changes
Other Study ID Numbers: CICL670EDE08T
First Posted: January 17, 2011    Key Record Dates
Last Update Posted: July 20, 2012
Last Verified: July 2012

Keywords provided by Crolll Gmbh:
Non-alcoholic steatohepatitis (NASH) and increased iron storage / disturbed distribution

Additional relevant MeSH terms:
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Fatty Liver
Non-alcoholic Fatty Liver Disease
Liver Diseases
Digestive System Diseases
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action