Myelodysplastic SyndromesPhase 4NCT00117507
Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients
A Phase 4 study testing Deferasirox in people with Myelodysplastic Syndromes and Iron Overload, run by Novartis Pharmaceuticals at 3 sites in United States.
Trial summary
Thirty patients were to be enrolled and 24 patients were actually enrolled into this open-label, single-arm trial designed to assess the safety and tolerability of oral deferasirox in adult transfusion dependent myelodysplastic syndrome (MDS) patients with iron overload. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Iron Overload
- What is being tested?
- Deferasirox
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 3 sitesUnited States
- Study dates
- Sep 2005to Jan 2008
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Male or female patients with low or intermediate (INT-1) risk MDS, determined via IPSS criteria, with transfusional iron overload. NOTE: Bone marrow morphology and cytogenetic studies completed within 3 months prior to screening can be used if the patient has been hematologically stable. Every attempt to obtain cytogenetics studies should be made; however, if there is culture failure, repeat marrow aspiration will not be mandated. In this case, RAEB with less than 11% marrow blasts will be accepted.
- Patients on chelation therapy at the time of screening required a 1-day wash out prior to the first dose of study drug.
- Age: greater than or equal to 18 years
- Serum ferritin:
- For entry into the screening period: serum ferritin greater than or equal to 1000 µg/mL on at least two occasions, at least two weeks apart, during the prior year. Samples must be obtained in the absence of concomitant infection;
- For enrollment into the study: serum ferritin greater than or equal to 1000 µg/mL at screening (via the central lab) obtained in the absence of concomitant infection
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- A lifetime minimum of 20 previous packed red cell transfusions
- Life expectancy greater than or equal to 6 months
- Women must have a negative serum or urine pregnancy test and use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined by amenorrhea for at least 12 months).
- Able to provide written informed consent
Who cannot join
- Serum creatinine greater than 2 × upper limit of normal (ULN)
- ALT or AST greater than 5 × ULN.
- Clinical or laboratory evidence of active hepatitis B or hepatitis C (HBsAg in the absence of HBsAb -OR- HCV Ab positive with HCV RNA positive and ALT above the normal range)
- Significant proteinuria as indicated by a urinary protein/creatinine ratio greater than 0.5 mg/mg in a non-first void urine sample during screening (or alternatively in two of three samples obtained for screening)
- History of HIV positive test result (ELISA or Western blot)
- ECOG performance status greater than 2
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- Uncontrolled systemic hypertension
- Unstable cardiac disease not controlled by standard medical therapy
- Third degree atrioventricular (AV) block or QT interval prolongation above the normal range
- History of clinically relevant ocular toxicity related to iron chelation
- Pregnancy or breast feeding
- Treatment with a systemic investigational drug within the past 4 weeks or a topical investigational drug within the past 7 days.
- Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
- inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
- major gastrointestinal tract surgery, such as gastrectomy, gastroenterostomy, or bowel resection;
- pancreatic injury or pancreatitis or indications of impaired pancreatic function/injury, as indicated by abnormal lipase or amylase;
- urinary obstruction or difficulty in voiding.
- History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Karmanos Cancer Center
Detroit, Michigan, 48201
MD Anderson Cancer Center
Houston, Texas, 77030-4009
Stanford University Medical Center
Stanford, California, 94305-5821
About this study
Official title: An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-Risk and INT-1 Myelodysplastic Patients
Thirty patients were to be enrolled and 24 patients were actually enrolled into this open-label, single-arm trial designed to assess the safety and tolerability of oral deferasirox in adult transfusion dependent myelodysplastic syndrome (MDS) patients with iron overload. Patients enrolled in this study had low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria. All patients initiated treatment with 20mg/kg/day deferasirox.
Deferasirox were administered orally once per day for 12 months.
Read the detailed description
Patients were screened for eligibility to determine if they meet all inclusion/exclusion criteria. The screening period were up to 4 weeks. Patient's baseline LIC will be determined non-invasively by means of MRI R2 analysis. In addition, blood and urine samples will be taken for the determination of baseline safety data.
- Conditions
- Myelodysplastic Syndromes, Iron Overload
- Being tested
- Deferasirox
- Keywords
- MDS, Myelodysplastic Syndrome, ICL-670, ICL-670 and Myelodysplastic Syndrome
- Medical subject headings
- Myelodysplastic Syndromes, Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
Also called Chelator, Iron chelator
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | Participants received deferasirox 20mg/kg/day OD for 12 months. Deferasirox was taken every morning 30 minutes before breakfast, if possible consistently around the same time between 7:00 and 9:00 AM. The tablets was dropped into water or orange juice and gently stirred for 1 to 3 minutes until completely dispersed. Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events and Serious Adverse Events
An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a subject or clinical investigation subject after providing written informed consent for participation in the study. Any sign or symptom that occured from first dose of study treatment until end of study treatment.
Measured: Up To Week 52
Secondary outcomes (7)
Absolute Change in Serum Ferritin From Baseline to Week 52
Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670).
Measured: Baseline to Week 52
Absolute Change in Liver Iron Concentration (LIC) From Baseline to End of Study
LIC was assessed using magnetic resonance imaging (MRI) mean liver proton transverse relaxation rates (R2).
Measured: Baseline to Week 52
To Evaluate Change in Transfusion Requirements
Change in transfusion requirements from baseline.
Measured: Baseline to Week 52
Absolute Change in Serum Erythropoietin
Absolute Change in Serum Erythropoietin from baseline.
Measured: Baseline to Week 52
Absolute Change in Urinary Hepcidin
Absolute Change in Urinary Hepcidin from baseline
Measured: Baseline to Week 52
Absolute Change in Transferrin Saturation
Transferrin Saturation was assessed using magnetic resonance imaging (MRI) mean liver proton transverse relaxation rates (R2)
Measured: Baseline to Week 52
Labile Plasma Iron (LPI)
LPI represents the component of non-transferrin bound iron and is an indicator of iron overload. The outcome was reported as LPI Unit, where, 1 LPI unit = the quantity of reactive oxygen species produced by approximately 1.5 μM Fe.
Measured: Baseline to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2005 (estimated)
- Study startSep 2005
- Primary completionJan 2008
- Study completionJan 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- 24 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
- NCT00117507
- Sponsor's protocol ID
- CICL670AUS02
- Status last verified
- Jun 2021
- First submitted
- 30 Jun 2005
- Last updated
- 24 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Greenberg PL, Koller CA, Cabantchik ZI, Warsi G, Glynos T, Paley C, Schiffer C. Prospective assessment of effects on iron-overload parameters of deferasirox therapy in patients with myelodysplastic syndromes. Leuk Res. 2010 Dec;34(12):1560-5. doi: 10.1016/j.leukres.2010.06.013. Epub 2010 Jul 8.
PubMed 20615548
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-Risk and INT-1 Myelodysplastic Patients
- ClinicalTrials.gov ID
- NCT00117507
- Acronym / sponsor ID
- CICL670AUS02
- Conditions
- Myelodysplastic Syndromes, Iron Overload
- Keywords
- MDS, Myelodysplastic Syndrome, ICL-670, ICL-670 and Myelodysplastic Syndrome
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Sep 2005
- Primary completion
- Jan 2008 (actual)
- Study completion
- Jan 2008 (actual)
- First posted
- 7 Jul 2005 (estimated)
- Results
- Posted 24 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Thirty patients were to be enrolled and 24 patients were actually enrolled into this open-label, single-arm trial designed to assess the safety and tolerability of oral deferasirox in adult transfusion dependent myelodysplastic syndrome (MDS) patients with iron overload. Patients enrolled in this study had low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria. All patients initiated treatment with 20mg/kg/day deferasirox.
Deferasirox were administered orally once per day for 12 months.
Detailed description
Patients were screened for eligibility to determine if they meet all inclusion/exclusion criteria. The screening period were up to 4 weeks. Patient's baseline LIC will be determined non-invasively by means of MRI R2 analysis. In addition, blood and urine samples will be taken for the determination of baseline safety data.
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: DeferasiroxParticipants received deferasirox 20mg/kg/day OD for 12 months. Deferasirox was taken every morning 30 minutes before breakfast, if possible consistently around the same time between 7:00 and 9:00 AM. The tablets was dropped into water or orange juice and gently stirred for 1 to 3 minutes until completely dispersed. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Male or female patients with low or intermediate (INT-1) risk MDS, determined via IPSS criteria, with transfusional iron overload. NOTE: Bone marrow morphology and cytogenetic studies completed within 3 months prior to screening can be used if the patient has been hematologically stable. Every attempt to obtain cytogenetics studies should be made; however, if there is culture failure, repeat marrow aspiration will not be mandated. In this case, RAEB with less than 11% marrow blasts will be accepted.
- Patients on chelation therapy at the time of screening required a 1-day wash out prior to the first dose of study drug.
- Age: greater than or equal to 18 years
- Serum ferritin:
- A lifetime minimum of 20 previous packed red cell transfusions
Key exclusion criteria (18 listed)
- Serum creatinine greater than 2 × upper limit of normal (ULN)
- ALT or AST greater than 5 × ULN.
- Clinical or laboratory evidence of active hepatitis B or hepatitis C (HBsAg in the absence of HBsAb -OR- HCV Ab positive with HCV RNA positive and ALT above the normal range)
- Significant proteinuria as indicated by a urinary protein/creatinine ratio greater than 0.5 mg/mg in a non-first void urine sample during screening (or alternatively in two of three samples obtained for screening)
- History of HIV positive test result (ELISA or Western blot)
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: Chelator, Iron chelator | Drug | Deferasirox |
Primary outcome
Number of Participants With Adverse Events and Serious Adverse Events
Time frame: Up To Week 52
An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a subject or clinical investigation subject after providing written informed consent for participation in the study. Any sign or symptom that occured from first dose of study treatment until end of study treatment.
7 secondary outcomes
Absolute Change in Serum Ferritin From Baseline to Week 52
Time frame: Baseline to Week 52
Absolute Change in Liver Iron Concentration (LIC) From Baseline to End of Study
Time frame: Baseline to Week 52
To Evaluate Change in Transfusion Requirements
Time frame: Baseline to Week 52
Absolute Change in Serum Erythropoietin
Time frame: Baseline to Week 52
Absolute Change in Urinary Hepcidin
Time frame: Baseline to Week 52
Absolute Change in Transferrin Saturation
Time frame: Baseline to Week 52
Labile Plasma Iron (LPI)
Time frame: Baseline to Week 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 20615548Greenberg PL, Koller CA, Cabantchik ZI, Warsi G, Glynos T, Paley C, Schiffer C. Prospective assessment of effects on iron-overload parameters of deferasirox therapy in patients with myelodysplastic syndromes. Leuk Res. 2010 Dec;34(12):1560-5. doi: 10.1016/j.leukres.2010.06.013. Epub 2010 Jul 8.
- Results for CICL670AUS02 from the Novartis Clinical Trials websiteLink 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
3 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 Iron Overload, run by Novartis Pharmaceuticals at 3 sites in United States.
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 18 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 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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?
Yes. Results are posted in the registry (first posted 24 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Adverse Events and Serious Adverse Events (time frame: Up To Week 52). 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 NCT00117507, 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 24 Jun 2021.