The Impact of Deferasirox on Non-Alcoholic-Steatohepatitis

Phase 1

Completed

• Conditions: Non-alcoholic Steatohepatitis; Increased Iron Storage / Disturbed Distribution
• Interventions: Drug: Exjade

• Registration Number: NCT01278056
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-03
• Study First Submitted: 2011-01-14
• Study First Submitted QC: 2011-01-14
• Study First Posted: 2011-01-17
• Primary Completion: 2012-03
• Status Verified: 2012-07
• Study Completion: 2012-07
• Last Update Submitted: 2012-07-19
• Last Update Posted: 2012-07-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Exjade	Exjade	-

Primary Outcome Measures

Name	Time	Method
Safety and tolerability of deferasirox in all patients (Phase I)	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.
Changes in liver histology in all patients (Phase II)	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

Name	Time	Method
Phase I: e.g. changes in liver enzymes, serum ferritin, and hemoglobin levels	Phase I: 12 weeks of treatment
Phase II: e.g. changes in MRI and histology based assessment of hepatic steatosis, fibrosis and iron content	Phase II: 48 weeks of treatment

Trial Locations

Locations (9)

Universitätsklinikum Mainz, I. Medizinische Klinik und Poliklinik; 🇩🇪 Mainz, Germany

Universitätsklinikum des Saarlandes; 🇩🇪 Homburg/Saar, Germany

Universitätsklinikum Magdeburg, Klinik für Gastroenterologie, Hepatologie und Infektiologie; 🇩🇪 Magdeburg, Germany

Universitätsklinikum Regensburg, Klinik und Poliklinik für Innere Med. I; 🇩🇪 Regensburg, Germany

Universitätsklinikum Halle, Klinik & Poliklinik für Innere Medizin I; 🇩🇪 Halle, Germany

Universitätsklinikum Tübingen, Medizinische Klinik IV; 🇩🇪 Tübingen, Germany

Charité, Virchow Klinikum; 🇩🇪 Berlin, Germany

Klinikum der J. W. Goethe-Universität, Med. Klinik I; 🇩🇪 Frankfurt, Germany

Zollernalbklinikum; 🇩🇪 Balingen/Hechingen, Germany