Liver Iron OverloadPhase 2/3NCT01033747
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
A Phase 2/3 study testing Deferasirox in people with Liver Iron Overload, run by Novartis Pharmaceuticals at 4 sites in Italy.
Trial summary
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Liver Iron Overload
- What is being tested?
- Deferasirox
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 4 sitesItaly
- Study dates
- Feb 2003to Jan 2008
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Currently participating in the 9-month comparative prolongation of extension phase of the original study.
- Patients currently participating in the food-effect sub-study, according to amendment 3.
- Ability to provide written informed consent prior to participation in this non-comparative extension study.
- Female patients sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
- Body weight of at least 35 kg.
Who cannot join
- Pregnant or breastfeeding patients.
- History of non-compliance to medical regimens and patients who are considered potentially unreliable.
- Proteinuria > 300 mg/L second void morning urine.
- Patients with serum creatinine above the upper limit normal.
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
4 sites in 1 country.
Italy4 sites
Novartis Investigative Site
Cagliari
Novartis Investigative Site
Genova
Novartis Investigative Site
Milan
Novartis Investigative Site
Torino
About this study
Official title: A 5-year Open Label, Non-comparative Extension to a Randomized, Open-label, Phase IIa Study to Evaluate Safety, Tolerability and the Effects on Liver Iron Concentration of Repeated Doses of 10 and 20 mg/kg/Day of Deferasirox in Comparison With 40 mg/kg/Day Deferoxamine in Patients With Transfusion-dependent Iron Overload
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
- Conditions
- Liver Iron Overload
- Being tested
- Deferasirox
- Keywords
- iron overload, iron chelation therapy, B-thalassemia
- Medical subject headings
- Iron Overload, beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
10 mg/kg or 30 mg/kg orally daily
DeferasiroxDrug
5 mg/kg or 30 mg/kg orally daily
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | Deferasirox group consists of all participants who were initially randomized to 10 and 20 mg/kg/day deferasirox orally daily in the main study and remained on the same treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative study Receives: Drug: Deferasirox |
| Deferasirox Crossover | Experimental | Deferasirox Crossover group consists of participants who were initially randomized to 40 mg/kg/day deferoxamine (DFO)subcutaneously in the main study and comparative prolongation study and crossed over to 5mg/kg/day to 30 mg/kg/day deferasirox orally daily at the beginning of the 5-year non-comparative extension study Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox
The mean percentage change in liver iron content (LIC) as assessed by superconducting quantum interference device (SQUID) was evaluated by comparing the LIC at the start of Deferasirox treatment to the LIC at the end of the 5 year extension study for participants who were treated with Deferasirox for more than 3.5 years. LIC is expressed in milligrams of iron per gram of liver dry weight (mgFe/g dw). Relative change = 1- (Change in LIC from Baseline/Baseline level) x 100.
Measured: Baseline to 7 Years
Secondary outcomes (1)
Relative Change in Serum Ferritin From Baseline to 3.5 Years
The mean percentage change in serum ferritin was evaluated by comparing the serum ferritin level at the start of Deferasirox treatment to the serum ferritin level collected 18 months following the start of the extension study. Serum ferritin is measured in micrograms per Liter. Relative Change = 1- (Change in ferritin level from Baseline/Baseline level) x 100.
Measured: Baseline to 3.5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2009 (estimated)
- Study startFeb 2003
- Primary completionJan 2008
- Study completionJan 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Liver Iron OverloadStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01033747
- Sponsor's protocol ID
- CICL670A0105E2
- Status last verified
- Jul 2011
- First submitted
- 16 Oct 2009
- Last updated
- 11 Aug 2011
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 5-year Open Label, Non-comparative Extension to a Randomized, Open-label, Phase IIa Study to Evaluate Safety, Tolerability and the Effects on Liver Iron Concentration of Repeated Doses of 10 and 20 mg/kg/Day of Deferasirox in Comparison With 40 mg/kg/Day Deferoxamine in Patients With Transfusion-dependent Iron Overload
- ClinicalTrials.gov ID
- NCT01033747
- Acronym / sponsor ID
- CICL670A0105E2
- Conditions
- Liver Iron Overload
- Keywords
- iron overload, iron chelation therapy, B-thalassemia
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Feb 2003
- Primary completion
- Jan 2008 (actual)
- Study completion
- Jan 2008 (actual)
- First posted
- 16 Dec 2009 (estimated)
- Results
- Posted 11 Aug 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Deferasirox | Experimental | Drug: DeferasiroxDeferasirox group consists of all participants who were initially randomized to 10 and 20 mg/kg/day deferasirox orally daily in the main study and remained on the same treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative study |
| Deferasirox Crossover | Experimental | Drug: DeferasiroxDeferasirox Crossover group consists of participants who were initially randomized to 40 mg/kg/day deferoxamine (DFO)subcutaneously in the main study and comparative prolongation study and crossed over to 5mg/kg/day to 30 mg/kg/day deferasirox orally daily at the beginning of the 5-year non-comparative extension study |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patients Currently participating in the 9-month comparative prolongation of extension phase of the original study.
- Patients currently participating in the food-effect sub-study, according to amendment 3.
- Ability to provide written informed consent prior to participation in this non-comparative extension study.
- Female patients sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
- Body weight of at least 35 kg.
Key exclusion criteria (5 listed)
- Pregnant or breastfeeding patients.
- History of non-compliance to medical regimens and patients who are considered potentially unreliable.
- Proteinuria > 300 mg/L second void morning urine.
- Patients with serum creatinine above the upper limit normal.
- Other protocol-defined inclusion/exclusion criteria may apply.
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 |
|---|---|---|
| Deferasirox10 mg/kg or 30 mg/kg orally daily | Drug | Deferasirox |
| Deferasirox5 mg/kg or 30 mg/kg orally daily | Drug | Deferasirox Crossover |
Primary outcome
The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox
Time frame: Baseline to 7 Years
The mean percentage change in liver iron content (LIC) as assessed by superconducting quantum interference device (SQUID) was evaluated by comparing the LIC at the start of Deferasirox treatment to the LIC at the end of the 5 year extension study for participants who were treated with Deferasirox for more than 3.5 years. LIC is expressed in milligrams of iron per gram of liver dry weight (mgFe/g dw). Relative change = 1- (Change in LIC from Baseline/Baseline level) x 100.
1 secondary outcome
Relative Change in Serum Ferritin From Baseline to 3.5 Years
Time frame: Baseline to 3.5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Liver Iron Overload
RecruitingObservational study
Fully Automated High-Throughput Quantitative MRI of the Liver
Healthy, Iron Overload
RecruitingObservational study
Impact of Iron Overload on the Incidence of Liver Complications in Long-Term Survivors (≥10 Years) of Allogeneic Hematopoietic Stem-Cell Transplantation.
Iron Overload, Hemosiderosis
RecruitingObservational study
An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis
Chronic Liver Disease
RecruitingObservational study
China Registry for Genetic / Metabolic Liver Diseases
Genetic/Metabolic Liver Diseases
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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Deferasirox in people with Liver Iron Overload, run by Novartis Pharmaceuticals at 4 sites in Italy.
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 5 inclusion and 5 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 11 Aug 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Italy. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Aug 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox (time frame: Baseline to 7 Years). 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 11 Aug 2011.