Thalassemia MajorPhase 4NCT00105495
Efficacy Study in Removing Excess Iron From the Heart
A Phase 4 study testing Ferriprox (deferiprone) and Desferal (deferoxamine) in people with Thalassemia Major and Hemosiderosis, run by ApoPharma at 4 sites in 2 countries.
Trial summary
The purpose of this study is to determine whether deferiprone has superior efficacy in removing excess iron from the heart when compared with deferoxamine. Read more about this study
- Who is it for?
- Ages 18 to 36 years
- Conditions the study is for
- Thalassemia Major, Hemosiderosis
- What is being tested?
- Ferriprox (deferiprone) and Desferal (deferoxamine)
- How many people
- 60 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Dec 2002to Oct 2004
- Phase
- Phase 4
- 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 to 36 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Diagnosis of thalassemia major as confirmed by laboratory and clinical criteria
- Participants who are well transfused-maintaining a mean pre-transfusion Hb (hemoglobin) no less than 9 g/dL.
- Between 18 and 36 years of age.
- Receiving ongoing chelation therapy with deferoxamine for at least the past five years. Those who have been exposed to deferiprone for
- ≤ 6 months but not within the last 2 years prior to commencement of this study will be considered eligible to participate.
- Abnormal heart MRI T2* greater than or equal to 8 ms and < 20 ms.
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- If female, fertile, and is neither pregnant nor lactating, confirms she will use an effective method of contraception for the length of the trial and has a negative pregnancy test immediately prior to commencement of study drug OR has had a tubal ligation OR a hysterectomy OR is post menopausal (at least 1 year no menses prior to enrollment in the study) OR their only sexual partner has been sterilized (if male).
- If male and fertile, he confirms that he and/or his partner will use an effective method of contraception for the length of the trial.
- Provide a signed and witnessed written informed consent obtained prior to the first study intervention.
Who cannot join
- Have anemia other than thalassemia.
- HIV antibody positive.
- Clinical evidence of cardiomyopathy as shown by LV Shortening Fraction < 30 % and/or CMR derived LV (left ventricular) Ejection Fraction < 56 %.
- Severe/significant arrhythmia, including those who have had atrial fibrillation (participants with occasional ectopic beats and normal echo can be included) or those requiring treatment.
- Previously discontinued therapy with deferiprone or deferoxamine because of an adverse drug reaction to either chelator.
- Have received deferiprone in the last five years. However those who have been exposed to deferiprone for ≤ 6 months but not within the last 2 years prior to commencement of this study will be considered eligible to participate.
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- Evidence of abnormal liver function (liver enzymes > 3 times upper limit of normal - entry may be delayed until return to normal).
- Have disorders associated with neutropenia (ANC < 1.5 x 10^9/L) or thrombocytopenia (platelet count <50 x 10^9/L) in the twelve months prior to start of study medication, except for participants who have been treated with interferon and in whom the ANC has fully recovered. Participants with neutropenia or thrombocytopenia in the last year, which resolved with splenectomy, may be considered for this study.
- Those who refuse to participate in the screening procedures or who are unable to participate in screening procedures or who are unable to comply with requirements of the protocol.
- Receiving other investigational products.
- Those in the opinion of the Investigator, who represent poor medical, psychological or psychiatric risks for whom participation in an investigational trial would be unwise.
- Those who are pregnant, breastfeeding or planning to become pregnant during the study period.
- Metallic objects in his/her body, such as artificial joints, inner ear (cochlear) implants, brain aneurysm clips, pacemakers, and metallic foreign bodies in the eye or other body areas.
- History of malignancy.
- Participants with claustrophobia.
- History of alcohol or drug abuse.
- Participants who are, in the opinion of the Investigator, excessively obese.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Greece2 sites
1st Department of Pediatrics, Athens University, Aghia Sophia Children's Hospital
Athens, 11527
Aghia Sophia Children's Hospital
Athens, 11527
Italy2 sites
Ospedale Regionale Microcitemie, Dipartimento di Scienze
Cagliari, Sardinia, 09100
Dipartimento di Scienze e Dell' Adolescenza, University of Turin
Turin, 10126
About this study
Official title: Randomized Trial Comparing the Relative Efficacy of Deferiprone to That of Deferoxamine in Removing Excess Cardiac Iron in Thalassemia Major Patients
The purpose of this study is to determine whether deferiprone has superior efficacy in removing excess iron from the heart when compared with deferoxamine.
Read the detailed description
This study is a multi-center, randomized, open-label, controlled clinical trial. The study population is participants with thalassemia major who are receiving regular chelation therapy with deferoxamine. A total of sixty (60) participants will be enrolled among the investigative sites.
The primary objective of this study is to determine whether deferiprone exhibits superior efficacy in removing excess iron from the heart compared to that of the standard therapy, deferoxamine.
The secondary objective is to evaluate the relative efficacy of deferiprone with respect to that of deferoxamine as assessed by serum ferritin concentration and liver iron concentration.
The primary efficacy measure in this study will be the participants' cardiac iron status, as determined by heart MRI T2* assessments.
The secondary efficacy measure will be by serum ferritin concentration and liver iron concentration. This will be measured by the Superconducting Quantum-Interference Device (SQUID) BioSusceptometer.
The duration of treatment is 12 months.
- Conditions
- Thalassemia Major, Hemosiderosis
- Being tested
- Ferriprox (deferiprone), Desferal (deferoxamine)
- Keywords
- Iron Overload, Thalassemia, Haemosiderosis, Cardiac, Deferiprone, Chelation
- Medical subject headings
- beta-Thalassemia, Hemosiderosis, Iron Overload, Thalassemia, Hemochromatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ferriprox (deferiprone)Drug
Desferal (deferoxamine)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine whether deferiprone exhibits superior efficacy in removing excess iron from the heart compared to that of deferoxamine, as reflected by MRI T2* assessments in the heart in participants treated with either chelator
Secondary outcomes (1)
To evaluate the relative efficacy of deferiprone with respect to that of deferoxamine as assessed by serum ferritin concentration and liver iron concentration (LIC)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2005 (estimated)
- Study startDec 2002
- Study completionOct 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia MajorStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants
Sponsor and investigators
- Lead sponsor
- ApoPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Renzo Galanello, M.D., Principal investigator, Ospedale Regionale Microcitemie, Cagliari, ItalyAntonio Piga, M.D., Principal investigator, Dipartimento di Scienze Pediatriche e Dell'Adolescenza, University of Turin, Turin, ItalyMarkissia Karagiorga, M.D., Principal investigator, Aghia Sophia Children's Hospital, Athens, GreeceVassilis Ladis, M.D., Principal investigator, 1st Department of Pediatrics, Athens University, Aghia Sophia Children's Hospital, Athens, Greece
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00105495
- Sponsor's protocol ID
- LA16-0102
- Status last verified
- Oct 2006
- First submitted
- 15 Mar 2005
- Last updated
- 5 Jan 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Trial Comparing the Relative Efficacy of Deferiprone to That of Deferoxamine in Removing Excess Cardiac Iron in Thalassemia Major Patients
- ClinicalTrials.gov ID
- NCT00105495
- Acronym / sponsor ID
- LA16-0102
- Conditions
- Thalassemia Major, Hemosiderosis
- Keywords
- Iron Overload, Thalassemia, Haemosiderosis, Cardiac, Deferiprone, Chelation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants
- Lead sponsor
- ApoPharmaIndustry (pharma, biotech, devices)
- Study start
- Dec 2002
- Study completion
- Oct 2004
- First posted
- 16 Mar 2005 (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.
The purpose of this study is to determine whether deferiprone has superior efficacy in removing excess iron from the heart when compared with deferoxamine.
Detailed description
This study is a multi-center, randomized, open-label, controlled clinical trial. The study population is participants with thalassemia major who are receiving regular chelation therapy with deferoxamine. A total of sixty (60) participants will be enrolled among the investigative sites.
The primary objective of this study is to determine whether deferiprone exhibits superior efficacy in removing excess iron from the heart compared to that of the standard therapy, deferoxamine.
The secondary objective is to evaluate the relative efficacy of deferiprone with respect to that of deferoxamine as assessed by serum ferritin concentration and liver iron concentration.
The primary efficacy measure in this study will be the participants' cardiac iron status, as determined by heart MRI T2* assessments.
The secondary efficacy measure will be by serum ferritin concentration and liver iron concentration. This will be measured by the Superconducting Quantum-Interference Device (SQUID) BioSusceptometer.
The duration of treatment is 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 36 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Diagnosis of thalassemia major as confirmed by laboratory and clinical criteria
- Participants who are well transfused-maintaining a mean pre-transfusion Hb (hemoglobin) no less than 9 g/dL.
- Between 18 and 36 years of age.
- Receiving ongoing chelation therapy with deferoxamine for at least the past five years. Those who have been exposed to deferiprone for
- ≤ 6 months but not within the last 2 years prior to commencement of this study will be considered eligible to participate.
Key exclusion criteria (17 listed)
- Have anemia other than thalassemia.
- HIV antibody positive.
- Clinical evidence of cardiomyopathy as shown by LV Shortening Fraction < 30 % and/or CMR derived LV (left ventricular) Ejection Fraction < 56 %.
- Severe/significant arrhythmia, including those who have had atrial fibrillation (participants with occasional ectopic beats and normal echo can be included) or those requiring treatment.
- Previously discontinued therapy with deferiprone or deferoxamine because of an adverse drug reaction to either chelator.
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 |
|---|---|---|
| Ferriprox (deferiprone) | Drug | – |
| Desferal (deferoxamine) | Drug | – |
Primary outcome
To determine whether deferiprone exhibits superior efficacy in removing excess iron from the heart compared to that of deferoxamine, as reflected by MRI T2* assessments in the heart in participants treated with either chelator
1 secondary outcome
To evaluate the relative efficacy of deferiprone with respect to that of deferoxamine as assessed by serum ferritin concentration and liver iron concentration (LIC)
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 5 Jan 2016.
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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.
Renzo Galanello, M.D. principal investigator
Ospedale Regionale Microcitemie, Cagliari, Italy
Antonio Piga, M.D. principal investigator
Dipartimento di Scienze Pediatriche e Dell'Adolescenza, University of Turin, Turin, Italy
Markissia Karagiorga, M.D. principal investigator
Aghia Sophia Children's Hospital, Athens, Greece
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Ferriprox (deferiprone) and Desferal (deferoxamine) in people with Thalassemia Major and Hemosiderosis, run by ApoPharma at 4 sites in 2 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 9 inclusion and 17 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 5 Jan 2016. 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 Greece, 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
To determine whether deferiprone exhibits superior efficacy in removing excess iron from the heart compared to that of deferoxamine, as reflected by MRI T2* assessments in the heart in participants treated with either chelator. 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 5 Jan 2016.