Non-alcoholic SteatohepatitisPhase 1/2DEFINENCT01278056
The Impact of Deferasirox on Non-Alcoholic-Steatohepatitis
A Phase 1/2 study testing Exjade in people with Non-alcoholic Steatohepatitis and Increased Iron Storage / Disturbed Distribution, run by Crolll Gmbh at 9 sites in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non-alcoholic Steatohepatitis, Increased Iron Storage / Disturbed Distribution
- What is being tested?
- Exjade
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 9 sitesGermany
- Study dates
- Mar 2010to Jul 2012
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with elevated liver enzymes
- Elevated serum ferritin (females > 300 ng/ml, males > 450 ng/ml)
- Liver Histology consistent with a diagnosis of NASH
Who cannot join
- 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
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- 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).
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
Germany9 sites
Zollernalbklinikum
Balingen/Hechingen, 72336
Charité, Virchow Klinikum
Berlin, 13353
Klinikum der J. W. Goethe-Universität, Med. Klinik I
Frankfurt, 60590
Universitätsklinikum Halle, Klinik & Poliklinik für Innere Medizin I
Halle, 06097
Universitätsklinikum des Saarlandes
Homburg/Saar, 66421
Universitätsklinikum Magdeburg, Klinik für Gastroenterologie, Hepatologie und Infektiologie
Magdeburg, 39120
Universitätsklinikum Mainz, I. Medizinische Klinik und Poliklinik
Mainz, 55131
Universitätsklinikum Regensburg, Klinik und Poliklinik für Innere Med. I
Regensburg, 93042
Universitätsklinikum Tübingen, Medizinische Klinik IV
Tübingen, 72076
About this study
Official title: The Impact of Deferasirox on Non-Alcoholic-Steatohepatitis (NASH) - a Prospective Open Label Phase I/II Trial
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.
Read more
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.
- Being tested
- Exjade
- Keywords
- Non-alcoholic steatohepatitis (NASH) and increased iron storage / disturbed distribution
- Medical subject headings
- Non-alcoholic Fatty Liver Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExjadeDrug
Also called Deferasirox, ICL670
Two dose escalating cohorts of oral administration in Phase I. Phase II: oral administration of the maximum tolerated dose.
| Study group | Role | What they receive |
|---|---|---|
| Exjade | Experimental | Not described Receives: Drug: Exjade |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of deferasirox in all patients (Phase I)
Safety and tolerability assessments will consist of evaluating (serious) adverse events, laboratory parameters including hematology, chemistry; vital signs and physical examinations according to CTC.
Measured: Phase I: 12 weeks of treatment
Changes in liver histology in all patients (Phase II)
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.
Measured: Phase II: 48 weeks of treatment
Secondary outcomes (2)
Phase I: e.g. changes in liver enzymes, serum ferritin, and hemoglobin levels
Measured: Phase I: 12 weeks of treatment
Phase II: e.g. changes in MRI and histology based assessment of hepatic steatosis, fibrosis and iron content
Measured: Phase II: 48 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2011 (estimated)
- Study startMar 2010
- Primary completionMar 2012
- Study completionJul 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non-alcoholic SteatohepatitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Crolll Gmbh
- Sponsor type
- University, hospital or other
- Collaborators
- Estimate, GmbH, University of Magdeburg
- Responsible party
- Sponsor
- Investigators
- Gerhard Treiber, PD Dr. med., Principal investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01278056
- Sponsor's protocol ID
- CICL670EDE08T
- Acronym
- DEFINE
- Status last verified
- Jul 2012
- First submitted
- 14 Jan 2011
- Last updated
- 20 Jul 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Jul 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Impact of Deferasirox on Non-Alcoholic-Steatohepatitis (NASH) - a Prospective Open Label Phase I/II Trial
- ClinicalTrials.gov ID
- NCT01278056
- Acronym / sponsor ID
- DEFINE / CICL670EDE08T
- Keywords
- Non-alcoholic steatohepatitis (NASH) and increased iron storage / disturbed distribution
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Crolll GmbhUniversity, hospital or other
- Collaborators
- Estimate, GmbH, University of Magdeburg
- Study start
- Mar 2010
- Primary completion
- Mar 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 17 Jan 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Exjade | Experimental | Drug: Exjade |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Patients with elevated liver enzymes
- Elevated serum ferritin (females > 300 ng/ml, males > 450 ng/ml)
- Liver Histology consistent with a diagnosis of NASH
Key exclusion criteria (22 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ExjadeAlso: Deferasirox, ICL670Two dose escalating cohorts of oral administration in Phase I. Phase II: oral administration of the maximum tolerated dose. | Drug | Exjade |
Primary outcome
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.
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.
2 secondary outcomes
Phase I: e.g. changes in liver enzymes, serum ferritin, and hemoglobin levels
Time frame: Phase I: 12 weeks of treatment
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2012.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gerhard Treiber, PD Dr. med. principal investigator
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Exjade in people with Non-alcoholic Steatohepatitis and Increased Iron Storage / Disturbed Distribution, run by Crolll Gmbh at 9 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Jul 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of deferasirox in all patients (Phase I) (time frame: Phase I: 12 weeks of treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2012.