Iron OverloadNCT01874405
Retrospective Evaluation of Adult and Pediatric Transfusion-dependent Patients Treated With Deferasirox Therapy
An observational study of Iron Overload, run by University of Campania Luigi Vanvitelli.
Trial summary
Iron overload is a leading cause of morbidity and mortality in transfusion-dependent patients. Deferasirox is the most promising iron chelator agent in several clinical scenarios. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Iron Overload
- How many people
- 80 participants
- Where
- No sites listed yet
- Study dates
- Mar 2003to Oct 2012
- Phase
- Observational
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Adult and pediatric transfusion- dependent patients (male and female) with different underlying cronic anemias who received iron chelation therapy with deferasirox during the observational study period and underwent at least 2 cardiac MRI scans at the Pozzuoli site. All consecutive patients visited at the participating sites starting from March 2003 to October 2012 will be entered in this observational study (chart review) provided all the inclusion an no exclusion criteria are met.
Who may be able to join
- Transfusion- dependent patients (> 2 years);
- Ongoing deferasirox therapy during the study period;
- ≥ 2 Magnetic Resonance scans (one at baseline and at least one post baseline - as per clinical need) during study period (this criteria is not mandatory for patients undergoing only the endocrine subanalysis and participating only to the cardiac analysis);
- Available medical history including relevant clinical and laboratory data (e.g serum ferritin, liver function tests, renal function tests, endocrine parameters ) at baseline before starting deferasirox treatment
Who cannot join
- Non transfusion- dependent patients;
- Other chelation therapy than deferasirox;
- Absence of complete medical history as above specified
About this study
Official title: Effectiveness of Deferasirox Therapy on Comprehensive Management of Iron Overload in Adult and Pediatric Transfusion-dependent Patients: a Long Term Retrospective Study
Iron overload is a leading cause of morbidity and mortality in transfusion-dependent patients. Deferasirox is the most promising iron chelator agent in several clinical scenarios. The investigators propose a retrospective study (chart review) to evaluate comprehensive iron overload management in transfusion-dependent patients treated with deferasirox for up to 5-10 years in a real clinical practice setting.
- Conditions
- Iron Overload
- Keywords
- Iron overload, thalassaemia, deferasirox, magnetic resonance, endocrine function
- Medical subject headings
- Iron Overload, Thalassemia
What is being measured
The primary outcome is the main question the study is designed to answer.
cardiac T2* in patients treated with deferasirox
change from baseline to end of study in cardiac T2*, as measured by Magnetic Resonance, in patients with iron overload (cardiac T2* <20 ms at baseline)
Measured: at least 1 year
cardiac T2* in patients treated with deferasirox
maintenance from baseline to end of study of cardiac T2* in not iron overloaded patients (cardiac T2* >20 ms at baseline)
Measured: at least 1 year
Secondary outcomes (3)
cardiac function in patient undergoing deferasirox treatment
change in left and right ejection fraction, telediastolic and telesystolic volumes, stroke volumes, cardiac output, myocardial mass, measured by Cardiac Magnetic Resonance, from baseline to end of study
Measured: at least 1 year
change in liver iron concentration
Measured: at least 1 year
maintenance of normal endocrine function in patients without endocrine dysfunction and improvement in disease severity in patients affected by endocrine dysfunction from baseline to end of study
Thyroid function (TSH, free triiodothyronine and free thyroxine serum free T4 levels), pancreatic cell function (basal glycemia, glycated hemoglobin level), bone mineral density (z-score) will be evaluated by the closest assessment to baseline (first deferasirox exposure) and to the end of study
Measured: at least 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2013 (estimated)
- Study startMar 2003
- Primary completionOct 2012
- Study completionOct 2012
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 Iron OverloadStudies 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
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Campania Luigi Vanvitelli
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Silverio Perrotta, University of Campania Luigi Vanvitelli
- Investigators
- Silverio Perrotta, MD, Principal investigator, University of Campania Luigi Vanvitelli
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01874405
- Sponsor's protocol ID
- DIPEDNA-2013-1
- Status last verified
- Jun 2013
- First submitted
- 26 May 2013
- Last updated
- 11 Jun 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Casale M, Citarella S, Filosa A, De Michele E, Palmieri F, Ragozzino A, Amendola G, Pugliese U, Tartaglione I, Della Rocca F, Cinque P, Nobili B, Perrotta S. Endocrine function and bone disease during long-term chelation therapy with deferasirox in patients with beta-thalassemia major. Am J Hematol. 2014 Dec;89(12):1102-6. doi: 10.1002/ajh.23844. Epub 2014 Sep 26.
PubMed 25197009
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness of Deferasirox Therapy on Comprehensive Management of Iron Overload in Adult and Pediatric Transfusion-dependent Patients: a Long Term Retrospective Study
- ClinicalTrials.gov ID
- NCT01874405
- Acronym / sponsor ID
- DIPEDNA-2013-1
- Conditions
- Iron Overload
- Keywords
- Iron overload, thalassaemia, deferasirox, magnetic resonance, endocrine function
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- University of Campania Luigi VanvitelliUniversity, hospital or other
- Study start
- Mar 2003
- Primary completion
- Oct 2012 (actual)
- Study completion
- Oct 2012 (actual)
- First posted
- 11 Jun 2013 (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.
Iron overload is a leading cause of morbidity and mortality in transfusion-dependent patients. Deferasirox is the most promising iron chelator agent in several clinical scenarios. The investigators propose a retrospective study (chart review) to evaluate comprehensive iron overload management in transfusion-dependent patients treated with deferasirox for up to 5-10 years in a real clinical practice setting.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Adult and pediatric transfusion- dependent patients (male and female) with different underlying cronic anemias who received iron chelation therapy with deferasirox during the observational study period and underwent at least 2 cardiac MRI scans at the Pozzuoli site. All consecutive patients visited at the participating sites starting from March 2003 to October 2012 will be entered in this observational study (chart review) provided all the inclusion an no exclusion criteria are met.
Key inclusion criteria (4 listed)
- Transfusion- dependent patients (> 2 years);
- Ongoing deferasirox therapy during the study period;
- ≥ 2 Magnetic Resonance scans (one at baseline and at least one post baseline - as per clinical need) during study period (this criteria is not mandatory for patients undergoing only the endocrine subanalysis and participating only to the cardiac analysis);
- Available medical history including relevant clinical and laboratory data (e.g serum ferritin, liver function tests, renal function tests, endocrine parameters ) at baseline before starting deferasirox treatment
Key exclusion criteria (3 listed)
- Non transfusion- dependent patients;
- Other chelation therapy than deferasirox;
- Absence of complete medical history as above specified
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
cardiac T2* in patients treated with deferasirox
Time frame: at least 1 year
change from baseline to end of study in cardiac T2*, as measured by Magnetic Resonance, in patients with iron overload (cardiac T2* <20 ms at baseline)
cardiac T2* in patients treated with deferasirox
Time frame: at least 1 year
maintenance from baseline to end of study of cardiac T2* in not iron overloaded patients (cardiac T2* >20 ms at baseline)
3 secondary outcomes
cardiac function in patient undergoing deferasirox treatment
Time frame: at least 1 year
change in liver iron concentration
Time frame: at least 1 year
maintenance of normal endocrine function in patients without endocrine dysfunction and improvement in disease severity in patients affected by endocrine dysfunction from baseline to end of study
Time frame: at least 3 years
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 11 Jun 2013.
- Derived from this study: PubMed 25197009Casale M, Citarella S, Filosa A, De Michele E, Palmieri F, Ragozzino A, Amendola G, Pugliese U, Tartaglione I, Della Rocca F, Cinque P, Nobili B, Perrotta S. Endocrine function and bone disease during long-term chelation therapy with deferasirox in patients with beta-thalassemia major. Am J Hematol. 2014 Dec;89(12):1102-6. doi: 10.1002/ajh.23844. Epub 2014 Sep 26.
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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.
Silverio Perrotta, MD principal investigator
University of Campania Luigi Vanvitelli
Questions about this trial
What is this clinical trial studying?
An observational study of Iron Overload, run by University of Campania Luigi Vanvitelli.
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 4 inclusion and 3 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 Jun 2013. Status can change between updates, so confirm with the study contact before making plans.
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.
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?
cardiac T2* in patients treated with deferasirox (time frame: at least 1 year). 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?
On the ClinicalTrials.gov record for NCT01874405, 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 11 Jun 2013.