Myelodysplastic SyndromePhase 4MACS1574NCT01326845
Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study
A Phase 4 study testing Deferasirox in people with Myelodysplastic Syndrome and Transfusional Iron Overload, run by Novartis Pharmaceuticals at 10 sites in 4 countries.
Trial summary
The objective of the study is to evaluate and compare the frequency and severity of GI adverse events in different dose administration regimens. The patient population consists of low or intermediate (int-1) risk myelodysplastic syndrome (MDS) patients with transfusional iron overload. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, Transfusional Iron Overload
- What is being tested?
- Deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 10 sitesin 4 countries
- Study dates
- Dec 2011to Sep 2012
- 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
- Written informed consent prior to any screening procedures
- Male or female patients ≥ 18 years of age
- Patient must weigh between 45-135 kg
- Patients with low or intermediate (int-1) risk MDS, as determined by IPSS score or RA, RARS by WHO criteria. IPSS must be confirmed by a bone marrow examination within 6 months prior to study entry and must be hematologically stable
- Deferasirox naïve:
- Sexually active pre-menopausal female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation
Who cannot join
- History or current GI disease
- Systemic diseases which could prevent study treatments
- Left ventricular ejection fraction< 50 % by echo cardiography
- Serum creatinine > 1.2 x ULN at screening
- Platelet counts < 25x 109/L except in cases where guidance is already given in the local deferasirox label
- AST or ALT > 2.5 xULN at screening
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
10 sites in 4 countries.
10 sites in 4 countries
France5 sites
Novartis Investigative Site
Brest, 29200
Novartis Investigative Site
Caen, Cedex, 14033
Novartis Investigative Site
Limoges, 87042
Novartis Investigative Site
Pessac, 33604
Novartis Investigative Site
Vandœuvre-lès-Nancy, 54511
Italy3 sites
Novartis Investigative Site
Alessandria, AL, 15100
Novartis Investigative Site
Roma, 00133
Novartis Investigative Site
Torino, TO, 10126
About this study
Official title: A Multicenter, Randomized, Comparative Study of Different Deferasirox Administration Regimens on Gastrointestinal (GI) Tolerability in Low or Intermediate (Int-1) Risk MDS Myelodysplastic Syndrome Patients With Transfusional Iron Overload.
The objective of the study is to evaluate and compare the frequency and severity of GI adverse events in different dose administration regimens. The patient population consists of low or intermediate (int-1) risk myelodysplastic syndrome (MDS) patients with transfusional iron overload. The study patients are randomized to either a morning dose of 20 mg/kg/day deferasirox or an evening dose of the same. Patients are then followed up for 6 months for any GI events and are assessed using patient reported outcomes tools e.g. a patient diary.
- Being tested
- Deferasirox
- Keywords
- Gastrointestinal adverse events,, myelodysplastic syndrome,, transfusional iron overload diarrhea,, constipation abdominal pain,, vomiting
- Medical subject headings
- Myelodysplastic Syndromes, Vomiting
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 am | Experimental | Deferasirox 20 mg/kg/day taken in the morning, 30 minutes before food Receives: Drug: Deferasirox |
| Deferasirox pm | Experimental | Deferasirox 20 mg/kg/day taken in the evening, no less than 2 hours after the last food intake or at least 30 minutes before the evening meal Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Difference in the Frequency of Overall Newly Occurring GI Adverse Events (AEs) in the Two Treatment Arms
Study was prematurely terminated and not powered for efficacy. Frequency of GI AEs during the overall study period is available in the AE tables reported in the safety section.
Measured: 3 months
Secondary outcomes (8)
Difference in Frequency of Overall Newly Occurring GI AEs Between the Two Treatment Groups at Month 6.
Study was prematurely terminated and not powered for efficacy.
Measured: 6 months
Difference in Frequency of Specific Commonly Reported GI AEs Between the Two Treatment Groups
Study was prematurely terminated and not powered for efficacy.
Measured: months 3 and 6.
Difference in Severity of Overall GI AEs Between the Two Treatment Groups
Study was prematurely terminated and not powered for efficacy.
Measured: months 3 and 6.
Difference in Severity of Specific Commonly Reported GI Symptoms Between the Two Treatment Groups
Study was prematurely terminated and not powered for efficacy.
Measured: months 3 and 6
Difference in Frequency and Severity of All Non-GI AEs Between the Two Treatment Groups
Study was prematurely terminated and not powered for efficacy.
Measured: months 3 and 6
the Difference Between the Time From Baseline to the First Occurrence of GI AEs Between the Two Treatment Groups
Study was prematurely terminated and not powered for efficacy.
Measured: 3 months, 6 months
Difference in Severity of GI Symptoms, Bowel Habits and Level of Satisfaction From the Patient's Perspective Between the Two Treatment Groups
Measured: 3 months, 6 months
Difference in Reducing Serum Ferritin After Each Month of Study Drug Administration Between the Two Groups
Study was prematurely terminated and not powered for efficacy.
Measured: 3 months, 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2011 (estimated)
- Study startDec 2011
- Primary completionSep 2012
- Study completionSep 2012
Why it stopped: The trial was terminated due to insufficient enrollment.
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.
- 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 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
- NCT01326845
- Sponsor's protocol ID
- CICL670A2417
- Other IDs
- 2011-001077-13
- Acronym
- MACS1574
- Status last verified
- Mar 2017
- First submitted
- 30 Mar 2011
- Last updated
- 3 Mar 2017
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Comparative Study of Different Deferasirox Administration Regimens on Gastrointestinal (GI) Tolerability in Low or Intermediate (Int-1) Risk MDS Myelodysplastic Syndrome Patients With Transfusional Iron Overload.
- ClinicalTrials.gov ID
- NCT01326845
- Acronym / sponsor ID
- MACS1574 / CICL670A2417
- Keywords
- Gastrointestinal adverse events,, myelodysplastic syndrome,, transfusional iron overload diarrhea,, constipation abdominal pain,, vomiting
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The trial was terminated due to insufficient enrollment.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Dec 2011
- Primary completion
- Sep 2012 (actual)
- Study completion
- Sep 2012 (actual)
- First posted
- 31 Mar 2011 (estimated)
- Results
- Posted 15 Nov 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of the study is to evaluate and compare the frequency and severity of GI adverse events in different dose administration regimens. The patient population consists of low or intermediate (int-1) risk myelodysplastic syndrome (MDS) patients with transfusional iron overload. The study patients are randomized to either a morning dose of 20 mg/kg/day deferasirox or an evening dose of the same. Patients are then followed up for 6 months for any GI events and are assessed using patient reported outcomes tools e.g. a patient diary.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Deferasirox am | Experimental | Drug: DeferasiroxDeferasirox 20 mg/kg/day taken in the morning, 30 minutes before food |
| Deferasirox pm | Experimental | Drug: DeferasiroxDeferasirox 20 mg/kg/day taken in the evening, no less than 2 hours after the last food intake or at least 30 minutes before the evening meal |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Written informed consent prior to any screening procedures
- Male or female patients ≥ 18 years of age
- Patient must weigh between 45-135 kg
- Patients with low or intermediate (int-1) risk MDS, as determined by IPSS score or RA, RARS by WHO criteria. IPSS must be confirmed by a bone marrow examination within 6 months prior to study entry and must be hematologically stable
- Sexually active pre-menopausal female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation
Key exclusion criteria (7 listed)
- History or current GI disease
- Systemic diseases which could prevent study treatments
- Left ventricular ejection fraction< 50 % by echo cardiography
- Serum creatinine > 1.2 x ULN at screening
- Platelet counts < 25x 109/L except in cases where guidance is already given in the local deferasirox label
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 am, Deferasirox pm |
Primary outcome
Difference in the Frequency of Overall Newly Occurring GI Adverse Events (AEs) in the Two Treatment Arms
Time frame: 3 months
Study was prematurely terminated and not powered for efficacy. Frequency of GI AEs during the overall study period is available in the AE tables reported in the safety section.
8 secondary outcomes
Difference in Frequency of Overall Newly Occurring GI AEs Between the Two Treatment Groups at Month 6.
Time frame: 6 months
Difference in Frequency of Specific Commonly Reported GI AEs Between the Two Treatment Groups
Time frame: months 3 and 6.
Difference in Severity of Overall GI AEs Between the Two Treatment Groups
Time frame: months 3 and 6.
Difference in Severity of Specific Commonly Reported GI Symptoms Between the Two Treatment Groups
Time frame: months 3 and 6
Difference in Frequency and Severity of All Non-GI AEs Between the Two Treatment Groups
Time frame: months 3 and 6
the Difference Between the Time From Baseline to the First Occurrence of GI AEs Between the Two Treatment Groups
Time frame: 3 months, 6 months
Difference in Severity of GI Symptoms, Bowel Habits and Level of Satisfaction From the Patient's Perspective Between the Two Treatment Groups
Time frame: 3 months, 6 months
Difference in Reducing Serum Ferritin After Each Month of Study Drug Administration Between the Two Groups
Time frame: 3 months, 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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
10 sites in 4 countries: 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 Transfusional Iron Overload, run by Novartis Pharmaceuticals at 10 sites in 4 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 6 inclusion and 7 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: stopped early, as of the sponsor's last update on 3 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in France, Italy, South Africa, Spain. 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 15 Nov 2013). 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?
Difference in the Frequency of Overall Newly Occurring GI Adverse Events (AEs) in the Two Treatment Arms (time frame: 3 months). 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 3 Mar 2017.