The Impact of Deferasirox on Non-Alcoholic-Steatohepatitis

NCT ID: NCT01278056

Last Updated: 2012-07-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-03-31
• Study Completion Date: 2012-07-31

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.

Conditions

Non-alcoholic Steatohepatitis; Increased Iron Storage / Disturbed Distribution

Keywords

Non-alcoholic steatohepatitis (NASH) and increased iron storage / disturbed distribution

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Exjade

Group Type: EXPERIMENTAL

Exjade

Intervention Type: DRUG

Two dose escalating cohorts of oral administration in Phase I. Phase II: oral administration of the maximum tolerated dose.

Interventions

Exjade

Two dose escalating cohorts of oral administration in Phase I. Phase II: oral administration of the maximum tolerated dose.

Intervention Type: DRUG

Other Intervention Names

Deferasirox, ICL670

Eligibility Criteria

Inclusion Criteria

• 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

• 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Estimate, GmbH

INDUSTRY

Sponsor Role: collaborator

University of Magdeburg

OTHER

Sponsor Role: collaborator

Crolll Gmbh

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Gerhard Treiber, PD Dr. med.

Role: PRINCIPAL_INVESTIGATOR

Locations

Zollernalbklinikum

Balingen/Hechingen, , Germany

Charité, Virchow Klinikum

Berlin, , Germany

Klinikum der J. W. Goethe-Universität, Med. Klinik I

Frankfurt, , Germany

Universitätsklinikum Halle, Klinik & Poliklinik für Innere Medizin I

Halle, , Germany

Universitätsklinikum des Saarlandes

Homburg/Saar, , Germany

Universitätsklinikum Magdeburg, Klinik für Gastroenterologie, Hepatologie und Infektiologie

Magdeburg, , Germany

Universitätsklinikum Mainz, I. Medizinische Klinik und Poliklinik

Mainz, , Germany

Universitätsklinikum Regensburg, Klinik und Poliklinik für Innere Med. I

Regensburg, , Germany

Universitätsklinikum Tübingen, Medizinische Klinik IV

Tübingen, , Germany

Countries

Germany

Other Identifiers

CICL670EDE08T

Identifier Type: -

Identifier Source: org_study_id