A Phase 2 interventional study of Deferasirox in Myelodysplastic Syndrome and Iron Overload, sponsored by Novartis Pharmaceuticals. Completed at 48 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-08-16.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment
The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.
Study entry requires a diagnosis of low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria and serum ferritin ≥ 1000 ng/mL. Patients must have had at least 30 prior red blood cell transfusions. Deferasirox will be administered at an initial dose of 20 mg/kg orally once per day. Patient transfusion history and at least three complete blood count (CBC) values must be available for the 12 weeks prior to study registration for patients with MDS and chronic iron overload from blood transfusions.
1,997 studies on the registry are indexed under Myelodysplastic Syndromes; 324 are open to participants now.
This study's enrollment of 176 is above the median of 40 across 1,611 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
For entry into the screening period, serum ferritin ≥ 1000 ng/mL on at least two occasions, at least two weeks apart, during the prior year.
Serum ferritin ≥ 1000 ng/mL at screening via the central lab.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Evaluate the safety and tolerability of deferasirox 20 mg/kg/day over one year in patients with MDS
Drug: Deferasirox
20 mg/kg/day over one year in patients with MDS
Also known as: ICL670A, chelator, desferal, iron chelation
Number of Participants Reporting Adverse Events
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the study, whether or not considered related to the participant's participation in the study. Serious Adverse Events include adverse events that result in either of death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect.
Time frame: up to 53 Weeks
Change in Serum Ferritin From Baseline to Weeks 13, 25, 37 and 53
Change in levels of serum ferritin from baseline to 3, 6, 9 and 12 months (Weeks 13, 25, 37 and 53).
Time frame: From Baseline to Weeks 13, 25, 37 and 53
Change in Labile Plasma Iron (LPI)
LPI represents the component of non-transferrin bound iron and is an indicator of iron overload. The blood sample for LPI determinations was collected at Week 1 (prior to first dose) and at Weeks 13, 25, 37 and 49. LPI was calculated as 1 LPI unit = the quantity of reactive oxygen species produced by approximately 1.5 μM Fe
Time frame: From Baseline to Weeks 13, 25, 37 and 49
Directly Chelatable Iron (DCI)
The blood sample for DCI determinations were collected at Week 1 (prior to first dose) and at Weeks 13, 25, 37 and 49.
Time frame: From Baseline to Weeks 13, 25, 37 and 49
Total Iron Levels
Levels of non-transferrin bound iron (NTBI) and serum iron from baseline to 3,6,9 and 12 months (Weeks 13, 25, 37 and 49) were assessed.
Time frame: From Baseline to Weeks 13, 25, 37, 49 and 53
Serum Transferrin Levels
Serum transferrin from baseline to 3, 6, 9 and 12 months of treatment (Visit Weeks 13, 25, 37 and 49) were assessed.
Time frame: From Baseline to Weeks 13, 25, 37, 49 and 53
Transferrin Saturation
Levels of transferrin saturation from baseline to 3, 6, 9 and 12 months.
Time frame: From Baseline to Weeks 13, 25, 37, 49 and 53
Transfusion Requirements
Number of participants receiving transfusions, the summarized during the study.
Time frame: up to 1 year
Frequency of Hematologic Improvement During the Study
Hematologic responses defined by International Working Group response criteria in myelodysplasia. Hematologic Improvement (HI) responses, at least 9 weeks defined as: Erythroid response (pretreatment, \<11 g/dL): Hgb increase by 1.5 g/dL; Relevant reduction units of Red blood cell (RBC) transfusions by absolute number at least 4 RBC transfusions/8 week compared with pretreatment transfusion number in previous 8 weeks; Only RBC transfusions for Hgb of 9.0 g/dL pretreatment count in RBC transfusion response evaluation; Platelet response (pretreatment,\<100x10\^9/L): If starting with \>20x10\^9/L platelets: absolute increase 30x10\^9/L, Increase from baseline \<20 x10\^9/L to \>20x10\^9/L and by =/\> 100%; Neutrophil response (pretreatment, \<1.0x10\^9/L): =/\> 100% increase \& absolute increase \>0.5x10\^9/L; Progression or relapse after HI: At least 1 of the following: =/\>50% decrement from max response levels in granulocytes or platelets; Reduction in Hgb by 1.5 g/dL; or Transfusion dependence.
Time frame: up to 1 year
Trough Plasma Deferasirox Concentration
The Pharmacokinetic (PK) parameters were assessed at Week 13, 25, 37 and 49 using Liquid Chromatography with tandem mass spectrometry (LC-MS/MS) method.
Time frame: At Week 13, 25, 37 and 49
Treatment Compliance to Deferasirox
Treatment compliance was assessed using records of study medication used, dosages administered, and intervals between visits during the study. Drug accountability was noted by the field monitor during site visits and at the completion of the trial. Participants were asked to return all unused medication at monthly visits.
Time frame: up to 1 year
The Prevalence of Hereditary Hemochromatosis Gene (HFE) Gene Mutations
HFE (hemochromatosis) gene mutations in the Myelodysplastic Syndrome (MDS) population, the trial included testing for the C282Y, H63D and S65C HFE gene mutations. One blood sample was collected at Baseline (Week 1), or, if that visit was missing, the sample was taken at Week 13 (Month 3). Only one blood sample was to be taken from each participant.
Time frame: up to Week 13 (Month 3)
The study was conducted at 39 centers in the United States and 6 centers in Canada
| Milestone | Deferasirox |
|---|---|
| Started | 173 |
| Completed | 95 |
| Not completed | 78 |
| Withdrew: Adverse event | 27 |
| Withdrew: Abnormal laboratory value | 15 |
| Withdrew: Withdrawal by subject | 16 |
| Withdrew: Death | 17 |
| Withdrew: Unsatisfactory therapeutic effect | 2 |
| Withdrew: Participant condition no longer requires study drug | 1 |
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the study, whether or not considered related to the participant's participation in the study. Serious Adverse Events include adverse events that result in either of death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect.
| Participants | Deferasirox |
|---|---|
| Number of Participants Reporting Adverse Events | 171 |
Change in levels of serum ferritin from baseline to 3, 6, 9 and 12 months (Weeks 13, 25, 37 and 53).
| μg/L | Deferasirox |
|---|---|
| Baseline | 2771.5 (863 to 9993) |
| Week 13 | -146.5 (-6679 to 3260) |
| Week 25 | -167.5 (-5034 to 2265) |
| Week 37 | -505.0 (-3699 to 4132) |
| Week 53 | -592.0 (-5752 to 5948) |
LPI represents the component of non-transferrin bound iron and is an indicator of iron overload. The blood sample for LPI determinations was collected at Week 1 (prior to first dose) and at Weeks 13, 25, 37 and 49. LPI was calculated as 1 LPI unit = the quantity of reactive oxygen species produced by approximately 1.5 μM Fe
| LPI Unit | Deferasirox |
|---|---|
| Baseline | 0.30 (0.0 to 3.6) |
| Week 13 | -0.10 (-3.3 to 2.2) |
| Week 25 | -0.20 (-2.4 to 1.2) |
| Week 37 | -0.30 (-3.4 to 0.6) |
| Week 49 | -0.45 (-3.6 to 0.9) |
The blood sample for DCI determinations were collected at Week 1 (prior to first dose) and at Weeks 13, 25, 37 and 49.
| µM | Deferasirox |
|---|---|
| Baseline | 0.00 (0.0 to 1.3) |
| Week 13 | 0.00 (-0.5 to 2.2) |
| Week 25 | 0.00 (-0.6 to 2.6) |
| Week 37 | 0.00 (-0.6 to 1.4) |
| Week 49 | 0.00 (-0.6 to 1.6) |
Levels of non-transferrin bound iron (NTBI) and serum iron from baseline to 3,6,9 and 12 months (Weeks 13, 25, 37 and 49) were assessed.
| μg/dL | Deferasirox |
|---|---|
| Baseline | 194.0 (48.0 to 409.0) |
| Week 13 | 230.50 (54.0 to 863.0) |
| Week 25 | 222.0 (27.0 to 497.0) |
| Week 37 | 212.0 (53.0 to 480.0) |
| Week 49 | 218.0 (48.0 to 929.0) |
| Week 53 | 221.00 (16.0 to 455.0) |
Serum transferrin from baseline to 3, 6, 9 and 12 months of treatment (Visit Weeks 13, 25, 37 and 49) were assessed.
| mg/dL | Deferasirox |
|---|---|
| Baseline | 156.0 (83.0 to 244.0) |
| Week 13 | 161.0 (108.0 to 257.0) |
| Week 25 | 160.0 (107.0 to 266.0) |
| Week 37 | 158.0 (88.0 to 272.0) |
| Week 49 | 160.0 (90.0 to 289.0) |
| Week 53 | 161.00 (99.0 to 253.0) |
Levels of transferrin saturation from baseline to 3, 6, 9 and 12 months.
| percentage of saturation | Deferasirox |
|---|---|
| Baseline | 91.00 (20.0 to 94.0) |
| Week 13 | -11.00 (-73.0 to 57.0) |
| Week 25 | -11.00 (-66.0 to 58.0) |
| Week 37 | -12.50 (-68.0 to 57.0) |
| Week 49 | -12.00 (-72.0 to 65.0) |
| Week 53 | -18.00 (-69.0 to 64.0) |
Number of participants receiving transfusions, the summarized during the study.
| Participants | Deferasirox |
|---|---|
| Screening | 163 |
| Week 1 through 13 | 136 |
| Week 14 through 26 | 119 |
| Week 27 through 39 | 98 |
| Week 40 through 52 | 72 |
Hematologic responses defined by International Working Group response criteria in myelodysplasia. Hematologic Improvement (HI) responses, at least 9 weeks defined as: Erythroid response (pretreatment, \<11 g/dL): Hgb increase by 1.5 g/dL; Relevant reduction units of Red blood cell (RBC) transfusions by absolute number at least 4 RBC transfusions/8 week compared with pretreatment transfusion number in previous 8 weeks; Only RBC transfusions for Hgb of 9.0 g/dL pretreatment count in RBC transfusion response evaluation; Platelet response (pretreatment,\<100x10\^9/L): If starting with \>20x10\^9/L platelets: absolute increase 30x10\^9/L, Increase from baseline \<20 x10\^9/L to \>20x10\^9/L and by =/\> 100%; Neutrophil response (pretreatment, \<1.0x10\^9/L): =/\> 100% increase \& absolute increase \>0.5x10\^9/L; Progression or relapse after HI: At least 1 of the following: =/\>50% decrement from max response levels in granulocytes or platelets; Reduction in Hgb by 1.5 g/dL; or Transfusion dependence.
| Participants | Deferasirox |
|---|---|
| Response (Yes) | 7 |
| Response (No) | 74 |
| Missing | 10 |
The Pharmacokinetic (PK) parameters were assessed at Week 13, 25, 37 and 49 using Liquid Chromatography with tandem mass spectrometry (LC-MS/MS) method.
| μmol/L | Deferasirox |
|---|---|
| Week 13 | 21.200 (0.00 to 209.0) |
| Week 25 | 26.700 (0.00 to 331.0) |
| Week 37 | 25.200 (0.00 to 242.0) |
| Week 49 | 30.700 (0.00 to 223.0) |
Treatment compliance was assessed using records of study medication used, dosages administered, and intervals between visits during the study. Drug accountability was noted by the field monitor during site visits and at the completion of the trial. Participants were asked to return all unused medication at monthly visits.
| participants | Deferasirox |
|---|---|
| 20 to < 40 % | 2 |
| 40 - < 60 % | 1 |
| 60 - < 80 % | 4 |
| 80 - < 100 % | 89 |
| 100 - < 120 % | 65 |
| ≥ 120 % | 12 |
HFE (hemochromatosis) gene mutations in the Myelodysplastic Syndrome (MDS) population, the trial included testing for the C282Y, H63D and S65C HFE gene mutations. One blood sample was collected at Baseline (Week 1), or, if that visit was missing, the sample was taken at Week 13 (Month 3). Only one blood sample was to be taken from each participant.
| Participants | Deferasirox |
|---|---|
| C282Y — Negative | 85 |
| C282Y — Positive Heterozygous | 9 |
| H63D — Negative | 70 |
| H63D — Positive Heterozygous | 24 |
| S65C — Negative | 92 |
| S65C — Positive Heterozygous | 2 |
Collected over All Adverse Event Were Collected From Start of Treatment up to 53 Weeks. Non-serious events are listed at a 2% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Deferasirox | 17/173 (9.8%) | 78/173 (45.1%) | 166/173 (96%) |
| Enrolled, Not Treated | 1/3 (33.3%) | 0/3 (0%) | 0/3 (0%) |
| Event | Deferasirox | Enrolled, Not Treated |
|---|---|---|
| PneumoniaInfections and infestations | 12/173 | 0/3 |
| Febrile neutropeniaBlood and lymphatic system disorders | 7/173 | 0/3 |
| PyrexiaGeneral disorders | 7/173 | 0/3 |
| CellulitisInfections and infestations | 6/173 | 0/3 |
| Renal failure acuteRenal and urinary disorders | 6/173 | 0/3 |
| DiarrhoeaGastrointestinal disorders | 5/173 | 0/3 |
| Oedema peripheralGeneral disorders | 5/173 | 0/3 |
| Urinary tract infectionInfections and infestations | 5/173 | 0/3 |
| DehydrationMetabolism and nutrition disorders | 5/173 | 0/3 |
| AnaemiaBlood and lymphatic system disorders | 4/173 | 0/3 |
| Event | Deferasirox | Enrolled, Not Treated |
|---|---|---|
| DiarrhoeaGastrointestinal disorders | 101/173 | 0/3 |
| FatigueGeneral disorders | 68/173 | 0/3 |
| NauseaGastrointestinal disorders | 60/173 | 0/3 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 36/173 | 0/3 |
| Oedema peripheralGeneral disorders | 35/173 | 0/3 |
| Blood creatinine increasedInvestigations | 34/173 | 0/3 |
| RashSkin and subcutaneous tissue disorders | 32/173 | 0/3 |
| ConstipationGastrointestinal disorders | 30/173 | 0/3 |
| PyrexiaGeneral disorders | 28/173 | 0/3 |
| AnorexiaMetabolism and nutrition disorders | 24/173 | 0/3 |
The full analysis set (FAS) population consisted of all participants who received at least 1 dose of Deferasirox.
| Age, Continuous(years) | Deferasirox |
|---|---|
| Mean | 69.9 ± 11.45 |
| Sex: Female, Male(Participants) | Deferasirox |
|---|---|
| Female | 70 |
| Male | 103 |
| Race/Ethnicity, Customized(Participants) | Deferasirox |
|---|---|
| Caucasian | 159 |
| Black | 4 |
| Oriental | 4 |
| Other | 6 |
Ran from Jul 2005 to primary completion in Mar 2008, 32 months.
Results were first posted in Aug 2021, 13 years 4 months after primary completion.
Of 52 industry-sponsored phase 2 interventional studies under Myelodysplastic Syndromes, half reached primary completion within 40 months of starting (the median), counting those still running; a quarter within 30 and three quarters within 62. This study's 32 months is below that.
Counting every sponsor, half of 279 studies reached it within 66 months; a quarter within 40 and three quarters within 104.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jul 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Deferasirox