Myelodysplastic SyndromesPhase 4NCT00481143
Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndrome and Transfusion-dependent Iron Overload
A Phase 4 study testing ICL670/Deferasirox in people with Myelodysplastic Syndromes and Transfusion Dependent Iron Overload, run by Novartis Pharmaceuticals at 1 site in Germany.
Trial summary
The purpose of this study is to investigate the effects of iron chelation using deferasirox in low and INT-1 risk (referring to the international prognostic scoring system, IPSS) MDS patients who show signs of iron overload due to repeated blood transfusions. This trial is not recruiting patients in the United States. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Transfusion Dependent Iron Overload
- What is being tested?
- ICL670/Deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 63 participants
- Where
- 1 siteGermany
- Study dates
- May 2007to Nov 2010
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- MDS patients presenting with low or intermediate-1 IPSS risk and transfusional iron overload
- Patients of either gender and age ≥ 18 years
- History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs)
- Patients can be either naïve to iron chelation or have had prior treatment with deferoxamine (DFO) or deferiprone (L1)
- Females of childbearing potential must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
Who cannot join
- Non-transfusion related iron overload
- Treatment with deferasirox (ICL670) before study start
- Patients with a concomitant malignant disease
- Patients with out of range lab values
- History of nephrotic syndrome
- Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
Show all 12Show fewer
- Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
- Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days
- Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug.
- Patients with active uncontrolled infectious disease
- Pregnancy or breast feeding
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 in Patients Diagnosed With Low and INT-1 Risk Myelodysplastic Syndrome (MDS) and Transfusion-dependent Iron Overload
The purpose of this study is to investigate the effects of iron chelation using deferasirox in low and INT-1 risk (referring to the international prognostic scoring system, IPSS) MDS patients who show signs of iron overload due to repeated blood transfusions.
This trial is not recruiting patients in the United States.
- Being tested
- ICL670/Deferasirox
- Keywords
- Myelodysplastic Syndrome, MDS, iron overload, chelation, chelators, ICL670, Deferasirox, serum ferritin, LIC, transfusional hemosiderosis, Refractory Anemia, RA, Refractory Anemia with Ringed Sideroblasts, RARS, blood transfusions, Mydelodysplastic Syndrome(s)
- Medical subject headings
- Myelodysplastic Syndromes, Iron Overload, Anemia, Refractory
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ICL670/DeferasiroxDrug
Also called ICL670
| Study group | Role | What they receive |
|---|---|---|
| deferasirox | Experimental | Not described Receives: Drug: ICL670/Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox
Measured: 52 weeks
Secondary outcomes (1)
Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs)
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 posted1 Jun 2007 (estimated)
- Study startMay 2007
- Primary completionNov 2010
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 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
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmeceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00481143
- Sponsor's protocol ID
- CICL670ADE03
- Status last verified
- Nov 2016
- First submitted
- 31 May 2007
- Last updated
- 31 May 2017
- Results posted
- No
Publications
Nolte F, Hochsmann B, Giagounidis A, Lubbert M, Platzbecker U, Haase D, Luck A, Gattermann N, Taupitz M, Baier M, Leismann O, Junkes A, Schumann C, Hofmann WK, Schrezenmeier H. Results from a 1-year, open-label, single arm, multi-center trial evaluating the efficacy and safety of oral Deferasirox in patients diagnosed with low and int-1 risk myelodysplastic syndrome (MDS) and transfusion-dependent iron overload. Ann Hematol. 2013 Jan;92(2):191-8. doi: 10.1007/s00277-012-1594-z. Epub 2012 Oct 17.
PubMed 23073603
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 in Patients Diagnosed With Low and INT-1 Risk Myelodysplastic Syndrome (MDS) and Transfusion-dependent Iron Overload
- ClinicalTrials.gov ID
- NCT00481143
- Acronym / sponsor ID
- CICL670ADE03
- Keywords
- Myelodysplastic Syndrome, MDS, iron overload, chelation, chelators, ICL670, Deferasirox, serum ferritin
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- May 2007
- Primary completion
- Nov 2010 (actual)
- First posted
- 1 Jun 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.
The purpose of this study is to investigate the effects of iron chelation using deferasirox in low and INT-1 risk (referring to the international prognostic scoring system, IPSS) MDS patients who show signs of iron overload due to repeated blood transfusions.
This trial is not recruiting patients in the United States.
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: ICL670/Deferasirox |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- MDS patients presenting with low or intermediate-1 IPSS risk and transfusional iron overload
- Patients of either gender and age ≥ 18 years
- History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs)
- Patients can be either naïve to iron chelation or have had prior treatment with deferoxamine (DFO) or deferiprone (L1)
- Females of childbearing potential must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
Key exclusion criteria (12 listed)
- Non-transfusion related iron overload
- Treatment with deferasirox (ICL670) before study start
- Patients with a concomitant malignant disease
- Patients with out of range lab values
- History of nephrotic syndrome
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 |
|---|---|---|
| ICL670/DeferasiroxAlso: ICL670 | Drug | deferasirox |
Primary outcome
To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox
Time frame: 52 weeks
1 secondary outcome
Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs)
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 31 May 2017.
- Results publication: PubMed 23073603Nolte F, Hochsmann B, Giagounidis A, Lubbert M, Platzbecker U, Haase D, Luck A, Gattermann N, Taupitz M, Baier M, Leismann O, Junkes A, Schumann C, Hofmann WK, Schrezenmeier H. Results from a 1-year, open-label, single arm, multi-center trial evaluating the efficacy and safety of oral Deferasirox in patients diagnosed with low and int-1 risk myelodysplastic syndrome (MDS) and transfusion-dependent iron overload. Ann Hematol. 2013 Jan;92(2):191-8. doi: 10.1007/s00277-012-1594-z. Epub 2012 Oct 17.
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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 Pharmeceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing ICL670/Deferasirox in people with Myelodysplastic Syndromes and Transfusion Dependent Iron Overload, run by Novartis Pharmaceuticals at 1 site in Germany.
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 12 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 31 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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 assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox (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 NCT00481143, 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 31 May 2017; AzTrials last refreshed its copy on 8 Nov 2026.