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CompletedNCT01868477Updated Oct 31, 2018Results posted

Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome.

A Phase 2 interventional study of Deferasirox DFX, DT and Erythropoietin alpha in Low and Int 1-risk Myelodysplastic Syndrome, sponsored by Novartis Pharmaceuticals. Completed at 30 sites in 10 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-10-31.

Sponsored by Novartis Pharmaceuticals · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
28
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

The primary purpose of this trial was is to assess the effect of treatment with deferasirox combined with erythropoietin vs. erythropoietin alone on erythropoiesis in patients with low- and int-1-risk myelodysplastic syndrome. The addition of deferasirox to erythropoietin can lead to a potential synergism with the reduction of reactive oxygen species, through both the NF-kB pathway and the control of free toxic iron. This may create a better environment in the bone marrow for a better response with erythropoietin.

This study was designed to test in a prospective way the combination of deferasirox with erythropoietin in terms of their effect on hematopoiesis.

Read the detailed description

This study did not meet the original enrollment objective of 60 patients and was terminated without extending enrollment past original planned LPFV of 31-Oct-2016.

02

Conditions studied

  • Low and Int 1-risk Myelodysplastic Syndrome

Keywords

  • myelodysplastic syndrome
  • MDS
  • myelodysplasia
  • blood disorder
  • cytopenias
  • low blood counts
  • progressive bone marrow failure
  • adult
  • ICL570
  • deferasirox
  • erythropoietin
  • erythropoiesis
  • NF-kB pathway
  • hematopoiesis.
03

In context

Preleukemia

1,317 studies on the registry are indexed under Preleukemia; 57 are open to participants now.

This study's enrollment of 28 is below the median of 36 across 1,060 interventional studies indexed under Preleukemia.

Browse Preleukemia studies →

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.

Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  • Patients who had low- and Int-1-risk myelodysplastic syndrome
  • Documented diagnosis of the following:

Myelodysplastic syndrome that lasted ≥ 3 months and \< 3 years Disease must not have been secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases

  • A hemoglobin \< 10 g/dL and ≥ 8 g/dL
  • History of transfusions \< 10 RBC units and must not have been RBC transfusion dependent
  • 300 ng/mL \< serum ferritin \< 1,500 ng/mL (Values within 10% difference above 1500 ng/ml or 10% difference below 300 ng/ml could have been accepted at the investigator's discretion.
  • Endogenous erythropoietin levels \< 500 units/L
  • Serum creatinine ≤ 1.5 times upper limit of normal (ULN)
  • Creatinine clearance above the concentration limit in locally approved prescribing information (PI). Patients with creatinine clearance between 40 and less than 60 mL/min, who did not present with additional risk factors that might impair renal function, were eligible at the discretion of the investigator

Key Exclusion Criteria:

  • Patients who had MDS with isolated del(5q)
  • Patients who had received prior EPO treatment or other recombinant growth factors regardless of the outcome (Patient who had received prior EPO treatment or other recombinant growth factors for less than 4 weeks and not within 3 months before screening without a documented response are allowed)
  • Patients who had received steroids or immunosuppressive therapy for the improvement of hematological parameters (stable steroid treatment for adrenal failure or chronic medical conditions, and intermittent dexamethasone as antiemetics were allowed).
  • B12 and folate deficient patients with and without clinical symptoms (patients were rescreened after successful therapy of B12 and folate deficiency)
  • Uncontrolled seizures or uncontrolled hypertension
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
28 participants (actual)

Study arms

  • Experimental
    Erythropoietin alpha

    Patients will receive erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be switched to the combination arm. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study.

    Drug: Erythropoietin alpha

  • Experimental
    Deferasirox + Erythropoietin alpha

    Patients will receive deferasirox dispersible tablet (DT) 10 mg/kg/day or deferasirox film-coated tablet (FCT) 7 mg/kg/day in combination with erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, erythropoietin dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be discontinued from the study. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study. Patients will continue deferasirox treatment.

    Drug: Deferasirox DFX, DT · Drug: Deferasirox DFX, FCT

Interventions

  • DrugDeferasirox DFX, DT

    provided as dispersible tablets for oral use in 125 and 250, 500 mg

  • DrugErythropoietin alpha
  • DrugDeferasirox DFX, FCT

    provided as film-coated tablet for oral use in 90, 180, 360 mg strengths

06

What researchers measure

Primary outcomes

  1. Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)

    Difference in percentage of patients achieving an erythroid response within 12 weeks of treatment between the two arms according to modified IWG 2006 criteria increase in hemoglobin (Hb) ≥ 1.5 g/dL. Erythroid response is defined as the increase in Hb from baseline ≥ 1.5 g/dL. Patients achieving erythroid response at least once within 12 weeks were considered responders

    Time frame: Baseline up to 12 weeks

Secondary outcomes

  1. Absolute Change From Baseline to Post-baseline Value for Hemoglobin(g/dL)(Full Analysis Set)

    Hematological response criteria defined as: Erythroid response: hemoglobin (Hb) increase from baseline \>= 1.5 g/dL (baseline \< 11 g/dL), neutrophil response: increase from baseline \>= 100% and increase \> 0.5 × 10\^9/L (baseline \<1 × 10\^9/L), platelet response: increase from baseline \>= 30 × 10\^9/L (baseline \<100 × 10\^9/L) according to modified IWG 2006 criteria

    Time frame: Baseline up to 24 weeks

  2. Summary of Hematologic Improvement in Patients Randomized to EPO+DFX and EPO Alone, Within 24 Weeks of Treatment (Full Analysis Set)

    Percentage of participants achieving an hematologic improvement defined as: neutrophil improvement: increase from baseline \>0.5 × 10\^9/L (baseline = 1.0 × 10\^9/L ), platelet improvement: increase from baseline ≥ 30 × 10\^9/L (baseline = 100 × 10\^9/L), hemoglobin improvement: Hb increase from baseline ≥ 1 g/dL (baseline\<11 g/dL)

    Time frame: Baseline up to 24 weeks

  3. Absolute Change in Hemoglobin Values up to 24 Weeks

    Absolute change in hemoglobin values for patients showing improvement: Hemoglobin improvement Hb increase from baseline ≥ 1 g/dL (baseline\<11 g/dL)

    Time frame: Baseline up to 24 weeks

  4. Absolute Change in Platelets and Neutrophil Levels up to 24 Weeks

    Absolute change in platelets and neutrophil levels for participants showing improvement: neutrophil improvement: increase from baseline \>0.5 × 10\^9/L (baseline = 1.0 × 10\^9/L ), platelet improvement: increase from baseline ≥ 30 × 10\^9/L (baseline = 100 × 10\^9/L)

    Time frame: Baseline up to 24 weeks

  5. Summary of Erythroid Response in Participants Randomized to EPO Alone at Baseline and Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)

    Erythroid response: hemoglobin increase from baseline \> = 1.5 g/dL (baseline \<11 g/dL)

    Time frame: Week 13 up to 24 weeks

  6. Summary of Erythroid Response Within 24 Weeks in Participants Randomized to EPO at Baseline and Not Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)

    Erythroid response: hemoglobin increase from baseline \> = 1.5 g/dL (baseline \<11 g/dL). Percentages are based on N. Confidence intervals are calculated using Clopper-Pearson method. Hemoglobin value is at time of first response

    Time frame: baseline up to 24 weeks

  7. Absolute Change in Serum Ferritin up to 24 Weeks for Erythropoietin Alpha Arm (Full Analysis Set)

    Absolute change in serum ferritin from baseline

    Time frame: Baseline up to 24 weeks

  8. Absolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)

    Absolute change in serum ferritin from baseline

    Time frame: Baseline up to 24 weeks

  9. Absolute Change in Serum Ferritin up to 24 Weeks for EPO+DFX at 12 Weeks Arm (Full Analysis Set)

    Absolute change in serum ferritin from baseline

    Time frame: Baseline up 24 weeks

  10. Absolute Change in Hemoglobin (Hb) From Baseline for Erythropoietin Alpha Arm (Full Analysis Set)

    This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy.

    Time frame: Baseline up to 24 weeks

  11. Absolute Change in Hemoglobin (Hb) From Baseline for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)

    This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy.

    Time frame: Baseline up to 24 weeks

  12. Absolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set)

    This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy. The time-course of Hb and its absolute changes from baseline was summarized by descriptive statistics by visit and erythroid response. Patients randomized to EPO and not switching after 12 weeks to EPO+DFX would consist of only responders.

    Time frame: Baseline up to 24 weeks

07

Results

Posted Oct 31, 2018

Participant flow

Participant flow — Overall Study
MilestoneErythropoietin AlphaDeferasirox + Erythropoietin Alpha
Started1211
Switched to epo+dfx50
Not switched to epo+dfx70
Completed86
Not completed45
Withdrew: Adverse event24
Withdrew: Disease progression11
Withdrew: Withdrawal by subject10

Outcome measures

PrimaryDifference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)

Difference in percentage of patients achieving an erythroid response within 12 weeks of treatment between the two arms according to modified IWG 2006 criteria increase in hemoglobin (Hb) ≥ 1.5 g/dL. Erythroid response is defined as the increase in Hb from baseline ≥ 1.5 g/dL. Patients achieving erythroid response at least once within 12 weeks were considered responders

Time frame:
Baseline up to 12 weeks
Reported as:
Number · percentage of participants
Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)
percentage of participantsErythropoietin AlphaDeferasirox + Erythropoietin Alpha
Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)41.7 (15.2 to 72.3)27.3 (6.02 to 61.0)
Statistical analysis
  • Erythropoietin Alpha vs Deferasirox + Erythropoietin Alpha · Mean difference (final values): 14.4 · 95% CI -24.0 to 48.16
SecondaryAbsolute Change From Baseline to Post-baseline Value for Hemoglobin(g/dL)(Full Analysis Set)

Hematological response criteria defined as: Erythroid response: hemoglobin (Hb) increase from baseline \>= 1.5 g/dL (baseline \< 11 g/dL), neutrophil response: increase from baseline \>= 100% and increase \> 0.5 × 10\^9/L (baseline \<1 × 10\^9/L), platelet response: increase from baseline \>= 30 × 10\^9/L (baseline \<100 × 10\^9/L) according to modified IWG 2006 criteria

Time frame:
Baseline up to 24 weeks
Reported as:
Mean · g/dL
Absolute Change From Baseline to Post-baseline Value for Hemoglobin(g/dL)(Full Analysis Set)
g/dLErythropoietin AlphaDeferasirox + Erythropoietin Alpha
Absolute Change From Baseline to Post-baseline Value for Hemoglobin(g/dL)(Full Analysis Set)1.8 ± 0.212.1 ± 0.61
SecondarySummary of Hematologic Improvement in Patients Randomized to EPO+DFX and EPO Alone, Within 24 Weeks of Treatment (Full Analysis Set)

Percentage of participants achieving an hematologic improvement defined as: neutrophil improvement: increase from baseline \>0.5 × 10\^9/L (baseline = 1.0 × 10\^9/L ), platelet improvement: increase from baseline ≥ 30 × 10\^9/L (baseline = 100 × 10\^9/L), hemoglobin improvement: Hb increase from baseline ≥ 1 g/dL (baseline\<11 g/dL)

Time frame:
Baseline up to 24 weeks
Reported as:
Number · percentage of participants
Summary of Hematologic Improvement in Patients Randomized to EPO+DFX and EPO Alone, Within 24 Weeks of Treatment (Full Analysis Set)
percentage of participantsErythropoietin AlphaDeferasirox + Erythropoietin Alpha
Hematologic improvement10045.5
Neutropil improvement66.780.0
Platelet improvement50.080.0
Hemoglobin improvement66.760.0
SecondaryAbsolute Change in Hemoglobin Values up to 24 Weeks

Absolute change in hemoglobin values for patients showing improvement: Hemoglobin improvement Hb increase from baseline ≥ 1 g/dL (baseline\<11 g/dL)

Time frame:
Baseline up to 24 weeks
Reported as:
Mean · g/dL
Absolute Change in Hemoglobin Values up to 24 Weeks
g/dLErythropoietin AlphaDeferasirox + Erythropoietin Alpha
Absolute Change in Hemoglobin Values up to 24 Weeks1.3 ± 0.371.4 ± 0.55
SecondaryAbsolute Change in Platelets and Neutrophil Levels up to 24 Weeks

Absolute change in platelets and neutrophil levels for participants showing improvement: neutrophil improvement: increase from baseline \>0.5 × 10\^9/L (baseline = 1.0 × 10\^9/L ), platelet improvement: increase from baseline ≥ 30 × 10\^9/L (baseline = 100 × 10\^9/L)

Time frame:
Baseline up to 24 weeks
Reported as:
Mean · 10^9 cells/L
Absolute Change in Platelets and Neutrophil Levels up to 24 Weeks
10^9 cells/LErythropoietin AlphaDeferasirox + Erythropoietin Alpha
Platelets58.7 ± 23.9366.3 ± 22.74
Neutrophils1.2 ± 1.162.4 ± 1.57
SecondarySummary of Erythroid Response in Participants Randomized to EPO Alone at Baseline and Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)

Erythroid response: hemoglobin increase from baseline \> = 1.5 g/dL (baseline \<11 g/dL)

Time frame:
Week 13 up to 24 weeks
Reported as:
Number · participants
Summary of Erythroid Response in Participants Randomized to EPO Alone at Baseline and Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)
participantsEPO+DFX (12 Weeks)
Summary of Erythroid Response in Participants Randomized to EPO Alone at Baseline and Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)0
SecondarySummary of Erythroid Response Within 24 Weeks in Participants Randomized to EPO at Baseline and Not Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)

Erythroid response: hemoglobin increase from baseline \> = 1.5 g/dL (baseline \<11 g/dL). Percentages are based on N. Confidence intervals are calculated using Clopper-Pearson method. Hemoglobin value is at time of first response

Time frame:
baseline up to 24 weeks
Reported as:
Number · percentage of participants
Summary of Erythroid Response Within 24 Weeks in Participants Randomized to EPO at Baseline and Not Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)
percentage of participantsEPO (24 Weeks)
Summary of Erythroid Response Within 24 Weeks in Participants Randomized to EPO at Baseline and Not Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)71.4 (47.8 to 100.0)
SecondaryAbsolute Change in Serum Ferritin up to 24 Weeks for Erythropoietin Alpha Arm (Full Analysis Set)

Absolute change in serum ferritin from baseline

Time frame:
Baseline up to 24 weeks
Reported as:
Median · ng/mL
Absolute Change in Serum Ferritin up to 24 Weeks for Erythropoietin Alpha Arm (Full Analysis Set)
ng/mLErythropoietin Alpha
Responders - Week 5-98.5 (-323 to -73.5)
Responders - Week 9-79.0 (-381 to 54.0)
Responders - Week 1324.8 (-179 to 104)
Responders - Week 17-57.8 (-140 to 258.0)
Responders - Week 21-39.8 (-44.0 to -35.5)
Non-responders - Week 5-352 (-523 to -182)
Non-responders - Week 9-189 (-572 to 194.5)
Non-responders - Week 13-44.5 (-621 to 531.5)
SecondaryAbsolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)

Absolute change in serum ferritin from baseline

Time frame:
Baseline up to 24 weeks
Reported as:
Median · ng/mL
Absolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)
ng/mLDeferasirox + Erythropoietin Alpha
Responders - Week 5-82.5 (-243 to 1068)
Responders - Week 9-139 (-292 to 702.0)
Responders - Week 13|-121 (-338 to 0.0)
Responders - Week 1716.5 (-143 to 722.0)
Responders - Week 21-95.5 (-189 to 173.0)
Non-responders - Week 5-38.0 (-315 to 111.0)
Non-responders - Week 9|-144 (-435 to 1.0)
Non-responders - Week 13|-155 (-225 to -127)
Non-responders - Week 17-123 (-154 to -91.0)
Non-responders - Week 21-291 (-291 to -291)
SecondaryAbsolute Change in Serum Ferritin up to 24 Weeks for EPO+DFX at 12 Weeks Arm (Full Analysis Set)

Absolute change in serum ferritin from baseline

Time frame:
Baseline up 24 weeks
Reported as:
Median · ng/mL
Absolute Change in Serum Ferritin up to 24 Weeks for EPO+DFX at 12 Weeks Arm (Full Analysis Set)
ng/mLEPO+DFX at 12
Responders - Week 5-116 (-116 to -116)
Responders - Week 9-136 (-136 to -136)
Responders - Week 1359.5 (59.5 to 59.5)
Responders - Week 1774.5 (74.5 to 74.5)
Non-responders - Week 5-68.3 (-144 to 221.3)
Non-responders - Week 9-148 (-319 to 321.3)
Non-responders - Week 13220.4 (-228 to 635.3)
Non-responders - Week 17-16.6 (-28.5 to -4.7)
Non-responders - Week 21-10.5 (-463 to 367.3)
SecondaryAbsolute Change in Hemoglobin (Hb) From Baseline for Erythropoietin Alpha Arm (Full Analysis Set)

This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy.

Time frame:
Baseline up to 24 weeks
Reported as:
Median · g/dL
Absolute Change in Hemoglobin (Hb) From Baseline for Erythropoietin Alpha Arm (Full Analysis Set)
g/dLErythropoietin Alpha
Responders - Week 51.5 (1.1 to 3.2)
Responders - Week 91.9 (1.3 to 4.4)
Responders - Week 131.7 (1.5 to 3.4)
Responders - Week 171.6 (-0.3 to 1.8)
Responders - Week 210.8 (-0.7 to 1.8)
Non-responders - Week 5-0.9 (-1.7 to -0.1)
Non-responders - Week 9-1.7 (-2.0 to -1.4)
Non-responders - Week 13-2.5 (-2.8 to -2.1)
SecondaryAbsolute Change in Hemoglobin (Hb) From Baseline for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)

This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy.

Time frame:
Baseline up to 24 weeks
Reported as:
Median · g/dL
Absolute Change in Hemoglobin (Hb) From Baseline for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)
g/dLDeferasirox + Erythropoietin Alpha
Responders - Week 50.7 (0.6 to 2.0)
Responders - Week 91.6 (1.0 to 2.6)
Responders - Week 132.9 (2.8 to 3.0)
Responders - Week 172.4 (0.6 to 3.0)
Responders - Week 211.7 (-1.3 to 2.4)
Non-responders - Week 5-0.1 (-2.3 to 0.1)
Non-responders - Week 90.0 (-0.8 to 0.5)
Non-responders - Week 130.2 (0.1 to 0.5)
Non-responders - Week 17-0.5 (-0.5 to -0.5)
Non-responders - Week 21-0.6 (-0.6 to -0.6)
SecondaryAbsolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set)

This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy. The time-course of Hb and its absolute changes from baseline was summarized by descriptive statistics by visit and erythroid response. Patients randomized to EPO and not switching after 12 weeks to EPO+DFX would consist of only responders.

Time frame:
Baseline up to 24 weeks
Reported as:
Median · g/dL
Absolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set)
g/dLEPO+DFX at 12 Weeks
Responders - Week 51.2 (1.2 to 1.2)
Responders - Week 9|1.8 (1.8 to 1.8)
Responders - Week 13|0.7 (0.7 to 0.7)
Responders - Week 17|-0.6 (-0.6 to -0.6)
Non-responders - Week 50.3 (-0.5 to 0.6)
Non-responders - Week 9|0.5 (-0.4 to 0.8)
Non-responders - Week 130.4 (0.2 to 1.0)
Non-responders - Week 17|0.0 (-0.1 to 0.9)
Non-responders - Week 210.0 (-0.7 to 0.8)

Adverse events

Collected over Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All Adverse events are reported in this record from First Patient First Treatment until Last Patient Last Visit up to approximately 24 weeks. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
EPO A0/7 (0%)1/7 (14.3%)4/7 (57.1%)
EPO+DFX DT0/10 (0%)1/10 (10%)10/10 (100%)
EPO+DFX FCT0/1 (0%)1/1 (100%)1/1 (100%)
Switched to DFX+EPO After 12 Weeks0/5 (0%)1/5 (20%)5/5 (100%)
Most frequent serious events
Most frequent serious events
EventEPO AEPO+DFX DTEPO+DFX FCTSwitched to DFX+EPO After 12 Weeks
DiverticulitisInfections and infestations0/70/101/10/5
SyncopeNervous system disorders0/70/101/10/5
TachycardiaCardiac disorders0/70/100/11/5
Inguinal herniaGastrointestinal disorders0/70/100/11/5
ArthralgiaMusculoskeletal and connective tissue disorders1/70/100/11/5
PyrexiaGeneral disorders1/70/100/10/5
C-reactive protein increasedInvestigations1/70/100/10/5
Back painMusculoskeletal and connective tissue disorders1/70/100/10/5
Musculoskeletal painMusculoskeletal and connective tissue disorders1/70/100/10/5
Femoral neck fractureInjury, poisoning and procedural complications0/71/100/10/5
Most frequent other events
Showing 10 of 41
Most frequent other events
EventEPO AEPO+DFX DTEPO+DFX FCTSwitched to DFX+EPO After 12 Weeks
DiarrhoeaGastrointestinal disorders0/73/101/10/5
Localised infectionInfections and infestations0/70/100/12/5
AnaemiaBlood and lymphatic system disorders2/72/100/11/5
VertigoEar and labyrinth disorders0/70/100/11/5
Abdominal pain upperGastrointestinal disorders0/70/100/11/5
Inguinal herniaGastrointestinal disorders0/70/100/11/5
ToothacheGastrointestinal disorders0/70/100/11/5
Injection site bruisingGeneral disorders0/70/100/11/5
Oedema peripheralGeneral disorders0/70/100/11/5
PyrexiaGeneral disorders0/70/100/11/5

Baseline characteristics

Age, Continuous
Age, Continuous(years)Erythropoietin AlphaDeferasirox + Erythropoietin AlphaTotal
Mean74.5 ± 5.8471.1 ± 7.5472.9 ± 6.77
Sex: Female, Male
Sex: Female, Male(Participants)Erythropoietin AlphaDeferasirox + Erythropoietin AlphaTotal
Female8513
Male4610
Race/Ethnicity, Customized
Race/Ethnicity, Customized(participants)Erythropoietin AlphaDeferasirox + Erythropoietin AlphaTotal
Caucasian8715
Asian448
08

Study locations

30 sites
  • Novartis Investigative Site
    Oran, 31000, Algeria
  • Novartis Investigative Site
    Sidi Bel abbes, 22000, Algeria
  • Novartis Investigative Site
    Caba, Buenos Aires C1425DND, Argentina
  • Novartis Investigative Site
    La Plata, Buenos Aires B1900AWT, Argentina
  • Novartis Investigative Site
    Vancouver, British Columbia V6Z1Y6, Canada
  • Novartis Investigative Site
    Hamilton, Ontario L8V 5C2, Canada
  • Novartis Investigative Site
    Toronto, Ontario M4N 3M5, Canada
  • Novartis Investigative Site
    Beijing, Beijing 100730, China
  • Novartis Investigative Site
    Guangzhou, Guangdong 51000, China
  • Novartis Investigative Site
    Nanjing, Jiangsu, China
  • Novartis Investigative Site
    Chengdu, Sichuan 610041, China
  • Novartis Investigative Site
    Hangzhou, Zhejiang 310003, China
  • Novartis Investigative Site
    Shanghai, 200025, China
  • Novartis Investigative Site
    Berlin, 12203, Germany
  • Novartis Investigative Site
    Dresden, 01307, Germany
  • Novartis Investigative Site
    Duesseldorf, 40225, Germany
  • Novartis Investigative Site
    Lütten-Klein, 18107, Germany
  • Novartis Investigative Site
    Wuerzburg, 97080, Germany
  • Novartis Investigative Site
    Cagliari, CA 09126, Italy
  • Novartis Investigative Site
    Reggio Calabria, RC 89124, Italy
  • Novartis Investigative Site
    Roma, RM 00161, Italy
  • Novartis Investigative Site
    Seoul, Korea 06351, Korea, Republic of
  • Novartis Investigative Site
    Badalona, Catalunya 08916, Spain
  • Novartis Investigative Site
    Girona, Catalunya 17007, Spain
  • Novartis Investigative Site
    Hospitalet de LLobregat, Catalunya 08907, Spain
  • Novartis Investigative Site
    Gothenburg, 413 45, Sweden
  • Novartis Investigative Site
    Linköping, SE-581 85, Sweden
  • Novartis Investigative Site
    Lulea, SE 971 80, Sweden
  • Novartis Investigative Site
    Stockholm, SE-141 86, Sweden
  • Novartis Investigative Site
    Oldham, Lancashire OL1 2JH, United Kingdom
09

References and documents

Study documents

  • Study protocol · Aug 6, 2015
  • Statistical analysis plan · Jun 12, 2017

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 31, 2018, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01868477
Lead sponsor
Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
Jun 4, 2013
Start date
Jan 28, 2014
Primary completion
Mar 22, 2017
Completion
Apr 5, 2017
Results posted
Oct 31, 2018
Last update
Oct 31, 2018

Study contacts

Novartis Pharmaceuticals
study director · Novartis Pharmaceuticals

Oversight

Data monitoring committee
No
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Not currently enrolling

This study is completed, as verified in Oct 2018. You cannot join it, but the record below documents what was studied.

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Other studies from this sponsor

Novartis Pharmaceuticals

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