Myelodysplastic SyndromesPhase 4NCT00564941
Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
A Phase 4 study testing deferasirox in people with Myelodysplastic Syndromes and Beta-Thalassemia, run by Novartis Pharmaceuticals at 4 sites in Hungary.
Trial summary
This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Myelodysplastic Syndromes, Beta-Thalassemia
- What is being tested?
- deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 309 participants
- Where
- 4 sitesHungary
- Study dates
- Dec 2007to Jul 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- In- or outpatients with myelodysplasia / with risk of low or intermedier-1 according to the International Prognostic Scoring System (IPSS) confirmed by bone marrow evaluation within 3 month/ or beta thalassaemia major patients, who have chronic iron overload, as a consequence of frequent blood transfusion
- Serum ferritin> 1800 µg/L
- Age: 18-80 years
- men and women
- Chronic iron overload caused by at least 30 units and maximum 100 units of blood of packed red blood cells
- Deferoxamin therapy is contraindicated or inadequate or unable to use in the recommended dose due to intolerability or other reason
- Eastern Cooperative Oncology Group (ECOG) performance status score between 0-2
- written informed consent
Who cannot join
- beta thalassaemia minor,
- haemosiderosis caused by other than chronic transfusional iron overload,
- patients with impaired renal function (Creatinin clearance< 60 ml/ min),
- pregnancy,
- lactation,
- patient of childbearing potential unwilling to use contraceptive precautions
Show all 16Show fewer
- known hypersensitivity to deferasirox or any ingredients,
- impaired hepatic function (SGOT,SGPT 5x above UNL).
- Patients severely ill due to underlying disease progression or other severe concomitant disease.
- Patients with poor prognosis of karyotype
- patients with malabsorption caused by inflammatory bowel disease, gastrectomy, pancreatitis or other medical condition
- History of nephrotic syndrome
- Significant proteinuria
- Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
- Patients with positive test to HIV
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
4 sites in 1 country.
Hungary4 sites
Novartis Investigative Site
Budapest
Novartis Investigative Site
Debrecen
Novartis Investigative Site
Pécs
Novartis Investigative Site
Szeged
About this study
This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
- Conditions
- Myelodysplastic Syndromes, Beta-Thalassemia
- Being tested
- deferasirox
- Keywords
- Myelodysplastic Syndromes, beta-Thalassemia, Iron Overload, Iron Chelating Agents, deferasirox
- Medical subject headings
- Myelodysplastic Syndromes, beta-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.
This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
Measured: monthly during the therapy and at the end of the treatment (aftr 9 months therapy)
Secondary outcomes (1)
patient's compliance during the study assessed by the number of the unused tablets returned by the patient safety assessed by patient laboratory data, adverse events, serious adverse events
Measured: during the treatment (9 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2007 (estimated)
- Study startDec 2007
- Primary completionJul 2011
- Study completionJul 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies 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
- 309 participants (estimated)
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
- NCT00564941
- Sponsor's protocol ID
- CICL670AHU02
- Status last verified
- Feb 2017
- First submitted
- 28 Nov 2007
- Last updated
- 24 Feb 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
- ClinicalTrials.gov ID
- NCT00564941
- Acronym / sponsor ID
- CICL670AHU02
- Conditions
- Myelodysplastic Syndromes, Beta-Thalassemia
- Keywords
- Myelodysplastic Syndromes, beta-Thalassemia, Iron Overload, Iron Chelating Agents, deferasirox
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 309 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Dec 2007
- Primary completion
- Jul 2011 (actual)
- Study completion
- Jul 2011 (actual)
- First posted
- 29 Nov 2007 (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 safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic 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
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- In- or outpatients with myelodysplasia / with risk of low or intermedier-1 according to the International Prognostic Scoring System (IPSS) confirmed by bone marrow evaluation within 3 month/ or beta thalassaemia major patients, who have chronic iron overload, as a consequence of frequent blood transfusion
- Serum ferritin> 1800 µg/L
- Age: 18-80 years
- men and women
- Chronic iron overload caused by at least 30 units and maximum 100 units of blood of packed red blood cells
Key exclusion criteria (16 listed)
- beta thalassaemia minor,
- haemosiderosis caused by other than chronic transfusional iron overload,
- patients with impaired renal function (Creatinin clearance< 60 ml/ min),
- pregnancy,
- lactation,
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
This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
Time frame: monthly during the therapy and at the end of the treatment (aftr 9 months therapy)
1 secondary outcome
patient's compliance during the study assessed by the number of the unused tablets returned by the patient safety assessed by patient laboratory data, adverse events, serious adverse events
Time frame: during the treatment (9 months)
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 24 Feb 2017.
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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
4 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 Syndromes and Beta-Thalassemia, run by Novartis Pharmaceuticals at 4 sites in Hungary.
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 8 inclusion and 16 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 24 Feb 2017. 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 Hungary. 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?
This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload (time frame: monthly during the therapy and at the end of the treatment (aftr 9 months therapy)). 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 24 Feb 2017.