Myelodysplastic SyndromePhase 4NCT01250951
This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
A Phase 4 study testing Deferasirox in people with Myelodysplastic Syndrome and Thalassemia, run by Novartis Pharmaceuticals at 2 sites in Russia.
Trial summary
This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, Thalassemia
- What is being tested?
- Deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 111 participants
- Where
- 2 sitesRussia
- Study dates
- Dec 2009to Sep 2011
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥ 2 years
- Primary Diagnosis: Myelodysplastic Syndrome (presenting with low or intermediate-1 IPSS risk), thalassemia or rare anemias patients (anemia Diamond-Blackfan, Fanconi's anemia, Sideroblastic anemia, Red cell aplasia)
- ECOG Performance Status ≤ 2
- Transfusion overload confirmed with ferritin level >1000 µg/l.
- No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure).
- Serum creatine level > ULN
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- No proteinuria
- Liver enzymes level < 5 ULN.
- No pregnancy or lactation
- Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents.
Who cannot join
- Age < 2 years
- No iron overload (Ferritin level <1000 µg/l).
- Primary iron overload (hereditary hemochromatosis)
- Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure)
- Elevated serum creatinine > ULN or/and proteinuria
- Liver enzymes level >5 ULN.
- Pregnancy or lactation.
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
2 sites in 1 country.
Russia2 sites
Novartis Investigative Site
Moscow
Novartis Investigative Site
Saint Petersburg
About this study
Official title: 1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.
- Conditions
- Myelodysplastic Syndrome, Thalassemia
- Being tested
- Deferasirox
- Keywords
- Iron Overload, hemotranfusion, deferasirox, ferritin, Rare anemia, Transfusional Iron Overload
- Medical subject headings
- Myelodysplastic Syndromes, Thalassemia, 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
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | Not described Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade
Measured: Baseline assessment is followed by monthly assessments for up to 1 year
Secondary outcomes (3)
changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination
Measured: Baseline assessment is followed by monthly assessments for up to 1 year.
changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade
Measured: at baseline and 1 at year (at the end of study).
Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination.
Measured: From the start of study up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Dec 2010 (estimated)
- Study startDec 2009
- Primary completionJul 2011
- Study completionSep 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 Myelodysplastic SyndromeStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 111 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
- NCT01250951
- Sponsor's protocol ID
- CICL670ARU01
- Status last verified
- Dec 2011
- First submitted
- 11 Nov 2010
- Last updated
- 12 Dec 2016
- Results posted
- No
Publications
Pennell DJ, Porter JB, Cappellini MD, El-Beshlawy A, Chan LL, Aydinok Y, Elalfy MS, Sutcharitchan P, Li CK, Ibrahim H, Viprakasit V, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood. 2010 Mar 25;115(12):2364-71. Epub 2009 Dec 8. Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, Gattermann N, Giraudier S, Lee JW, Chan LL, Lin KH, Rose C, Taher A, Thein SL, Viprakasit V, Habr D, Domokos G, Roubert B, Kattamis A; EPIC Study Investigators. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica. 2010 Apr;95(4):557-66. Epub 2009 Nov 30.
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
- 1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
- ClinicalTrials.gov ID
- NCT01250951
- Acronym / sponsor ID
- CICL670ARU01
- Conditions
- Myelodysplastic Syndrome, Thalassemia
- Keywords
- Iron Overload, hemotranfusion, deferasirox, ferritin, Rare anemia, Transfusional Iron Overload
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 111 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Dec 2009
- Primary completion
- Jul 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 1 Dec 2010 (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 evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.
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: Deferasirox |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Age ≥ 2 years
- Primary Diagnosis: Myelodysplastic Syndrome (presenting with low or intermediate-1 IPSS risk), thalassemia or rare anemias patients (anemia Diamond-Blackfan, Fanconi's anemia, Sideroblastic anemia, Red cell aplasia)
- ECOG Performance Status ≤ 2
- Transfusion overload confirmed with ferritin level >1000 µg/l.
- No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure).
Key exclusion criteria (8 listed)
- Age < 2 years
- No iron overload (Ferritin level <1000 µg/l).
- Primary iron overload (hereditary hemochromatosis)
- Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure)
- Elevated serum creatinine > ULN or/and proteinuria
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: ICL670 | Drug | Deferasirox |
Primary outcome
changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade
Time frame: Baseline assessment is followed by monthly assessments for up to 1 year
3 secondary outcomes
changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination
Time frame: Baseline assessment is followed by monthly assessments for up to 1 year.
changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade
Time frame: at baseline and 1 at year (at the end of study).
Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination.
Time frame: From the start of study up to 1 year
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 publicationPennell DJ, Porter JB, Cappellini MD, El-Beshlawy A, Chan LL, Aydinok Y, Elalfy MS, Sutcharitchan P, Li CK, Ibrahim H, Viprakasit V, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood. 2010 Mar 25;115(12):2364-71. Epub 2009 Dec 8. Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, Gattermann N, Giraudier S, Lee JW, Chan LL, Lin KH, Rose C, Taher A, Thein SL, Viprakasit V, Habr D, Domokos G, Roubert B, Kattamis A; EPIC Study Investigators. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica. 2010 Apr;95(4):557-66. Epub 2009 Nov 30.
- DeferasiroxLink provided by the sponsor.
- ThalassemiaLink provided by the sponsor.
- Myelodisplastic SyndromeLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
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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 4 study testing Deferasirox in people with Myelodysplastic Syndrome and Thalassemia, run by Novartis Pharmaceuticals at 2 sites in Russia.
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 10 inclusion and 8 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 Russia. 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?
changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade (time frame: Baseline assessment is followed by monthly assessments for up to 1 year). 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 NCT01250951, 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.