AnemiaPhase 2NCT00303329
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
A Phase 2 study testing Deferasirox in people with Anemia and Hemosiderosis, run by Novartis Pharmaceuticals at 28 sites in 7 countries.
Trial summary
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Anemia, Hemosiderosis
- What is being tested?
- Deferasirox
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 184 participants
- Where
- 28 sitesin 7 countries
- Study dates
- Mar 2004to Oct 2008
- Phase
- Phase 2
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients completed the planned 12-month core study
- Female patients who have reached menarche and who are 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
- Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation
Who cannot join
- Pregnant or breast feeding patients
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
28 sites in 7 countries.
28 sites in 7 countries
Italy8 sites
Novartis Investigative Site
Bologna
Novartis Investigative Site
Brindisi
Novartis Investigative Site
Cagliari
Novartis Investigative Site
Genova
Novartis Investigative Site
Milan
Novartis Investigative Site
Pavia
Novartis Investigative Site
Rome
Novartis Investigative Site
Torino
United States5 sites
Childres's Hospital Boston
Boston, Massachusetts, 02115
New York Presbyterian Hospital
New York, New York, 10021
Children's Hospital and Research center at Oakland
Oakland, California, 94609-1809
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399
Stanford Hospital
Stanford, California, 94305
Belgium5 sites
Novartis Investigative Site
Bruges
Novartis Investigative Site
Brussels
Novartis Investigative Site
Ghent
Novartis Investigative Site
La Louvière
Novartis Investigative Site
Leuven
France4 sites
Novartis Investigative Site
Créteil
Novartis Investigative Site
Le Kremlin-Bicêtre
Novartis Investigative Site
Lille
Novartis Investigative Site
Troyes
Germany3 sites
Novartis Investigative Site
Düsseldorf
Novartis Investigative Site
Hanover
Novartis Investigative Site
Ulm
Canada2 sites
Novartis Investigative Site
Montreal
Novartis Investigative Site
Toronto
About this study
Official title: Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older. Patients who successfully completed the main trial may continue in the extension trial to receive chelation therapy with deferasirox for up to 3 years. Extension was prolonged to 4 years.
The objective of this study is to assess the long-term safety and efficacy of deferasirox in these patient groups.
- Conditions
- Anemia, Hemosiderosis
- Being tested
- Deferasirox
- Keywords
- β-thalassemia, rare chronic anemia, iron overload, deferasirox, chronic anemias, transfusional hemosiderosis
- Medical subject headings
- Anemia, Hemosiderosis, Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
Also called ICL670
Deferasirox available as 125 mg, 250 mg or 500 mg tablets
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | Deferasirox daily oral dose between 5-40 mg/kg/day Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.
Measured: Core study Baseline to the end of the study (up to 60 months)
Secondary outcomes (3)
The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study
Liver iron concentration was monitored at the start of the core study, the end of the core study, and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant.
Measured: Core study Baseline to end of extension study (up to 60 months)
The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study
Liver iron concentration was monitored at the end of the core study and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. Pediatric participants or participants with a medical contraindication to liver biopsy were allowed the use of SQUID in the extension study.
Measured: Core study Baseline to end of extension study (up to 60 months)
The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
Serum ferritin was monitored monthly and the dose of deferasirox was increased or decreased in steps of 5 to 10 mg/kg/day up to a maximum of 40 mg/kg/day if appropriate, every 3 months. If serum ferritin fell to 500 ng/mL or lower on two consecutive study visits, an interruption of treatment until serum ferritin was more than 500 ng/mL was considered.
Measured: Core study Baseline to end of extension study (up to 60 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2006 (estimated)
- Study startMar 2004
- Primary completionOct 2008
- Study completionOct 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 AnemiaStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 184 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
- NCT00303329
- Sponsor's protocol ID
- CICL670A0108E1
- Status last verified
- Apr 2011
- First submitted
- 14 Oct 2005
- Last updated
- 9 May 2011
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload
- ClinicalTrials.gov ID
- NCT00303329
- Acronym / sponsor ID
- CICL670A0108E1
- Conditions
- Anemia, Hemosiderosis
- Keywords
- β-thalassemia, rare chronic anemia, iron overload, deferasirox, chronic anemias, transfusional hemosiderosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 184 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Mar 2004
- Primary completion
- Oct 2008 (actual)
- Study completion
- Oct 2008 (actual)
- First posted
- 16 Mar 2006 (estimated)
- Results
- Posted 9 May 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older. Patients who successfully completed the main trial may continue in the extension trial to receive chelation therapy with deferasirox for up to 3 years. Extension was prolonged to 4 years.
The objective of this study is to assess the long-term safety and efficacy of deferasirox in these patient groups.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deferasirox | Experimental | Drug: DeferasiroxDeferasirox daily oral dose between 5-40 mg/kg/day |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients completed the planned 12-month core study
- Female patients who have reached menarche and who are 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
- Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation
Key exclusion criteria (2 listed)
- Pregnant or breast feeding patients
- 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 |
|---|---|---|
| DeferasiroxAlso: ICL670Deferasirox available as 125 mg, 250 mg or 500 mg tablets | Drug | Deferasirox |
Primary outcome
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
Time frame: Core study Baseline to the end of the study (up to 60 months)
Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.
3 secondary outcomes
The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study
Time frame: Core study Baseline to end of extension study (up to 60 months)
The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study
Time frame: Core study Baseline to end of extension study (up to 60 months)
The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
Time frame: Core study Baseline to end of extension study (up to 60 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
28 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Deferasirox in people with Anemia and Hemosiderosis, run by Novartis Pharmaceuticals at 28 sites in 7 countries.
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 2 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 9 May 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in Italy, United States, Belgium, France, Germany and 2 more countries. 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 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.
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 9 May 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 Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths (time frame: Core study Baseline to the end of the study (up to 60 months)). 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 9 May 2011.