Beta Thalassemia Transfusion DependentPhase 2NCT00879242
Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec
A Phase 2 study testing Deferasirox in people with Beta Thalassemia Transfusion Dependent, run by Novartis Pharmaceuticals at 2 sites in Italy.
Trial summary
This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Beta Thalassemia Transfusion Dependent
- 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
- 20 participants
- Where
- 2 sitesItaly
- Study dates
- Feb 2009to May 2011
- Phase
- Phase 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- β-thalassemic patients receiving regular transfusions with an history of at least 20 transfusional events
- Patients naïve for deferasirox
- Serum ferritin levels ≥1000ng/ml (average of the last 6 months assays);
- Cardiac MRI T2* >5 and <20 msec.
- LVEF at MRI ≥56%
Who cannot join
- Patients that have already started deferasirox therapy
- Existing contraindications to the performance of MRI procedure, including presence of metallic prostheses not compatible with the MRI procedure other than those approved as safe for use in MRI scanners (e.g., some types of aneurysm clips, shrapnel in proximity to vital organs such as the retina), pacemaker, intractable claustrophobia and incapability to follow the instruction for the correct performance of the MRI (e.g. to maintain a supine position, to hold breath, etc.)
- Clinical conditions requiring intensive chelating therapy on the basis of Investigator's judgment
- Stable average ALT levels >300 U/L in the preceding 12 months
- Uncontrolled systemic hypertension
- Estimated creatinine clearance <60 ml/min
Show all 13Show fewer
- History of nephrotic syndrome
- History of clinically significant ocular toxicity related to the chelating therapy
- Psychiatric disorders or drug dependence that might impair the capability of the patient to provide their Informed Consent or to be administered the study treatment
- Known sensitivity to study drug(s) or class of study drug(s)
- Patients with severe medical condition(s) that in the view of the investigator prohibits participation in the study
- Use of any other investigational agent in the last 30 days.
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
2 sites in 1 country.
Italy2 sites
Novartis Investigative Site
Cagliari
Novartis Investigative Site
Orbassano
About this study
Official title: Multicenter, Open Label, Prospective Study to Evaluate the Efficacy and Safety of Deferasirox 30 mg/kg/Day for 52 Weeks, in Transfusion-dependent Beta-thalassemic Patients With Cardiac MRI T2* < 20 Msec
This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec.
- Conditions
- Beta Thalassemia Transfusion Dependent
- Being tested
- Deferasirox
- Keywords
- Thalassemia, cardiac MRI T2*
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
Dispersible tables of 125, 250 and 500 mg at the dose of 30 mg/kg/day, oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | 30 mg/kg/day. The daily dose can be increased to 40 mg/kg in case of unsatisfactory response after 12 weeks of treatment. Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in cardiac T2* value
Unsatisfactory response is a monthly MRI T2* improvement lower than 3% versus baseline.
Measured: 52 weeks
Secondary outcomes (2)
Change from baseline in cardiac functions parameters (LVEF, LVESV and LVEDV)
Measured: 52 weeks
Change from baseline liver T2* value
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2009 (estimated)
- Study startFeb 2009
- Primary completionMay 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Beta Thalassemia Transfusion DependentStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00879242
- Sponsor's protocol ID
- CICL670AIT07
- Other IDs
- EUDRACT Code :2008-003230-22
- Status last verified
- Nov 2011
- First submitted
- 8 Apr 2009
- Last updated
- 12 Dec 2016
- Results posted
- No
Publications
Piga A, Longo F, Origa R, Roggero S, Pinna F, Zappu A, Castiglioni C, Cappellini MD. Deferasirox for cardiac siderosis in beta-thalassaemia major: a multicentre, open label, prospective study. Br J Haematol. 2014 Nov;167(3):423-6. doi: 10.1111/bjh.12987. Epub 2014 Jun 30. No abstract available.
PubMed 24976388
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Open Label, Prospective Study to Evaluate the Efficacy and Safety of Deferasirox 30 mg/kg/Day for 52 Weeks, in Transfusion-dependent Beta-thalassemic Patients With Cardiac MRI T2* < 20 Msec
- ClinicalTrials.gov ID
- NCT00879242
- Acronym / sponsor ID
- CICL670AIT07
- Conditions
- Beta Thalassemia Transfusion Dependent
- Keywords
- Thalassemia, cardiac MRI T2*
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Feb 2009
- Primary completion
- May 2011 (actual)
- First posted
- 9 Apr 2009 (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 study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deferasirox | Experimental | Drug: Deferasirox30 mg/kg/day. The daily dose can be increased to 40 mg/kg in case of unsatisfactory response after 12 weeks of treatment. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- β-thalassemic patients receiving regular transfusions with an history of at least 20 transfusional events
- Patients naïve for deferasirox
- Serum ferritin levels ≥1000ng/ml (average of the last 6 months assays);
- Cardiac MRI T2* >5 and <20 msec.
- LVEF at MRI ≥56%
Key exclusion criteria (13 listed)
- Patients that have already started deferasirox therapy
- Existing contraindications to the performance of MRI procedure, including presence of metallic prostheses not compatible with the MRI procedure other than those approved as safe for use in MRI scanners (e.g., some types of aneurysm clips, shrapnel in proximity to vital organs such as the retina), pacemaker, intractable claustrophobia and incapability to follow the instruction for the correct performance of the MRI (e.g. to maintain a supine position, to hold breath, etc.)
- Clinical conditions requiring intensive chelating therapy on the basis of Investigator's judgment
- Stable average ALT levels >300 U/L in the preceding 12 months
- Uncontrolled systemic hypertension
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 |
|---|---|---|
| DeferasiroxDispersible tables of 125, 250 and 500 mg at the dose of 30 mg/kg/day, oral administration. | Drug | Deferasirox |
Primary outcome
Change from baseline in cardiac T2* value
Time frame: 52 weeks
Unsatisfactory response is a monthly MRI T2* improvement lower than 3% versus baseline.
2 secondary outcomes
Change from baseline in cardiac functions parameters (LVEF, LVESV and LVEDV)
Time frame: 52 weeks
Change from baseline liver T2* value
Time frame: 52 weeks
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 12 Dec 2016.
- Results publication: PubMed 24976388Piga A, Longo F, Origa R, Roggero S, Pinna F, Zappu A, Castiglioni C, Cappellini MD. Deferasirox for cardiac siderosis in beta-thalassaemia major: a multicentre, open label, prospective study. Br J Haematol. 2014 Nov;167(3):423-6. doi: 10.1111/bjh.12987. Epub 2014 Jun 30. No abstract available.
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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
2 sites in 1 country: 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 Beta Thalassemia Transfusion Dependent, run by Novartis Pharmaceuticals at 2 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 13 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 12 Dec 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 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?
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?
Change from baseline in cardiac T2* value (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00879242, which also lists 1 linked publication.
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 12 Dec 2016.