Trial · NCT00631163
Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis
NCT00631163, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2021-06-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis. Trials, record NCT00631163. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00631163
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis
- NCT number
- NCT00631163
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 102 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Chronic Anemia, Transfusional Hemosiderosis
- Interventions
- Deferasirox (ICL670)
- First posted
- 2008-03-07
- Last updated
- 2021-06-29
- Other registries' IDs
- eudract:2006-003337-32
- Results first posted
- 2021-06-29
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1 | From the Baseline, Year 1 (End of core study) | Liver iron concentration (LIC), a predictor of iron burden, was measured using relaxation rate magnetic resonance imaging (R2-MRI) technique. Relaxation rate was determined as R2 = 1/relaxation time (T2). The baseline value of LIC of participants was categorized as \< 7, ≥ 7 to \< 15, and ≥ 15 milligram of iron/tissue dry weight (mg Fe/g dw). A negative change from baseline favored study treatment in reducing LIC. |
| Secondary | Absolute Change From Baseline in Liver Iron Concentration (LIC) to End of Year 2 | From the Baseline to End of Year 2 (End of extension study) | Liver Iron Concentration (LIC), a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC of participants was categorized as \< 7, ≥ 7 to \< 15, and ≥ 15 mg Fe/g dw. A negative change from baseline favoured study treatment in reducing LIC. |
| Secondary | Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup | From the Baseline, End of Year 1 (End of core study), End of Year 2 (End of extension study) | Liver Iron Concentration (LIC), a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC was \< 7, ≥ 7 to \< 15, and ≥ 15 mg Fe/g dw. A negative change from baseline favoured study treatment in reducing LIC. |
| Secondary | Absolute Change From Baseline in Serum Ferritin Levels to Year 2 | From the Baseline up to Year 2 (End of extension study) | Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content. |
| Secondary | Absolute Serum Ferritin Levels Over 2 Years | From the Baseline, Year 1 (End of core study), Year 2 (End of extension study) | Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content. |
| Secondary | Total Body Iron Elimination Rate (TBIE), Iron Intake, Iron Excretion/Iron Intake and Chelation Efficiency After 2 Years | From the Baseline, Year 2 (End of extension study) | Total body iron excretion (TBIE)was used to investigate the chelation efficacy of Deferasirox therapy. TBIE rate was estimated based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores. |
| Secondary | Correlation of LIC and Serum Ferritin at Core and Extension Study | From the Baseline, Year 1 (End of core study), Year 2 (End of extension study) | LIC, a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC was \< 7, ≥ 7 to \< 15, and ≥ 15 mg Fe/g dw. Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content. The correlation between absolute change in LIC and absolute change in serum ferritin was determined. |
| Secondary | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESI), Discontinuation and Interruption | From the Baseline up to Year 2 (End of extension study) | Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require hospitalisation, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Death was defined as a fatal event leading to permanent cessation of all vital functions of the body. |
| Secondary | Number of Participants With Clinically Significant Ophthalmological Abnormalities | At 2 years (End of extension study) | Clinically significant changes in left eye and right eye were assessed by the investigator based on methods like visual acuity, slit lamp examination, tonometry and fundus oculi. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1 | From the Baseline, Year 1 (End of core study) | The analysis was performed in per-protocol population in core study (PP1 Set), comprising of all enrolled participants who had LIC assessments at baseline and Year 1. | mg Fe/g dw | MEAN |
| SECONDARY | Absolute Change From Baseline in Liver Iron Concentration (LIC) to End of Year 2 | From the Baseline to End of Year 2 (End of extension study) | The analysis was performed in per-protocol population in core study (PP2 Set), comprising of all enrolled participants who had LIC assessments at baseline and at end of the extension phase. | mg Fe/g dw | MEAN |
| SECONDARY | Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup | From the Baseline, End of Year 1 (End of core study), End of Year 2 (End of extension study) | The analysis was performed in PP1 set in Japanese subgroup defined as all participants who were enrolled in Japan for core study (Year 1) and PP2 set in Japanese subgroup for extension study (Year 2). Here, 'n' signifies the participants reviewed for Liver Iron Concentration (LIC) in Japanese subgroup for each group, respectively. | mg Fe/g dw | MEAN |
| SECONDARY | Absolute Change From Baseline in Serum Ferritin Levels to Year 2 | From the Baseline up to Year 2 (End of extension study) | The analysis was performed in PP2 Set population and Japanese subgroup. Here, Number of subjects analyzed signifies the participants assessed for serum ferritin during the study for each arm, respectively. | nanogram(s)/millilitre | MEAN |
| SECONDARY | Absolute Serum Ferritin Levels Over 2 Years | From the Baseline, Year 1 (End of core study), Year 2 (End of extension study) | The analysis was performed in PP1 set population for core study (Year 1) and PP2 set population for extension study (Year 2) and Japanese subgroup. Here, Number of participants analysed signifies the subjects assessed for serum ferritin during the study for each arm, respectively. | nanogram(s)/millilitre | MEAN |
| SECONDARY | Total Body Iron Elimination Rate (TBIE), Iron Intake, Iron Excretion/Iron Intake and Chelation Efficiency After 2 Years | From the Baseline, Year 2 (End of extension study) | The analysis was performed in PP2 set population and Japanese/tim subgroup. Here, Number of participants analyzed included all participants who were evaluable for the specified categories. | mg/kg/day | MEAN |
| SECONDARY | Correlation of LIC and Serum Ferritin at Core and Extension Study | From the Baseline, Year 1 (End of core study), Year 2 (End of extension study) | The analysis was performed in PP1 set for core study (Year 1) and PP2 set for extension study (Year 2). Here, Number of subjects analysed signifies the subjects assessed for LIC and serum ferritin during the study for each arm, respectively. | Correlation coefficient | NUMBER |
| SECONDARY | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESI), Discontinuation and Interruption | From the Baseline up to Year 2 (End of extension study) | The analysis was performed in the safety set (SAF) population, defined as subjects who received at least one dose of study drug, which was defined as at least one administration record with a valid date and an actual total daily dose administrated above zero, and Japanese sub-group. | participants | NUMBER |
| SECONDARY | Number of Participants With Clinically Significant Ophthalmological Abnormalities | At 2 years (End of extension study) | The analysis was performed in the SAF population, defined as subjects who received at least one dose of study drug, which was defined as at least one administration. These participants comprise 2 year completer groups | participants | NUMBER |
Source ClinicalTrials.gov
Results by group33
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1 | Deferasirox | -10.9 | Standard Deviation 11.86 | mg Fe/g dw | |
| Absolute Change From Baseline in Liver Iron Concentration (LIC) to End of Year 2 | Deferasirox | -13.5 | Standard Deviation 14.10 | mg Fe/g dw | |
| Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup | Deferasirox | Year 1 | -13.9 | Standard Deviation 10.21 | mg Fe/g dw |
| Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup | Deferasirox | Year 2 | -18.4 | Standard Deviation 12.48 | mg Fe/g dw |
| Absolute Change From Baseline in Serum Ferritin Levels to Year 2 | All Randomized Participants | -677.9 | Standard Deviation 4462.11 | nanogram(s)/millilitre | |
| Absolute Change From Baseline in Serum Ferritin Levels to Year 2 | Japanese Participants | -892.8 | Standard Deviation 5724.89 | nanogram(s)/millilitre | |
| Absolute Serum Ferritin Levels Over 2 Years | All Randomized Participants | Year 1 | 2653.3 | Standard Deviation 2952.07 | nanogram(s)/millilitre |
| Absolute Serum Ferritin Levels Over 2 Years | Japanese Participants | Year 1 | 2903.5 | Standard Deviation 3376.00 | nanogram(s)/millilitre |
| Absolute Serum Ferritin Levels Over 2 Years | All Randomized Participants | Year 2 | 2092.4 | Standard Deviation 2287.11 | nanogram(s)/millilitre |
| Absolute Serum Ferritin Levels Over 2 Years | Japanese Participants | Year 2 | 2114.8 | Standard Deviation 2391.31 | nanogram(s)/millilitre |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Number of Participants in Year 1 | STARTED | Deferasirox | 102 | |
| Number of Participants in Year 1 | COMPLETED | Deferasirox | 68 | |
| Number of Participants in Year 1 | NOT COMPLETED | Deferasirox | 34 | |
| Number of Participants in Year 2 | STARTED | Deferasirox | 57 | |
| Number of Participants in Year 2 | COMPLETED | Deferasirox | 52 | |
| Number of Participants in Year 2 | NOT COMPLETED | Deferasirox | 5 |
Source ClinicalTrials.gov
Adverse events555
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | ANAEMIA | Blood and lymphatic system disorders | Myelodysplastic Syndrome | 5 | 42 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Aplastic Anemia | 1 | 29 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Other | 1 | 31 | |
| other | APLASTIC ANAEMIA | Blood and lymphatic system disorders | Myelodysplastic Syndrome | 0 | 42 | |
| other | APLASTIC ANAEMIA | Blood and lymphatic system disorders | Aplastic Anemia | 2 | 29 | |
| other | APLASTIC ANAEMIA | Blood and lymphatic system disorders | Other | 0 | 31 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | Myelodysplastic Syndrome | 0 | 42 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | Aplastic Anemia | 2 | 29 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | Other | 1 | 31 | |
| other | THROMBOCYTOPENIA | Blood and lymphatic system disorders | Myelodysplastic Syndrome | 3 | 42 |
1 to 10 of 555Source ClinicalTrials.gov
See all 555 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Myelodysplastic Syndrome | 2 | 42 |
| Total, all-cause mortality | Aplastic Anemia | 3 | 29 |
| Total, all-cause mortality | Other | 1 | 31 |
| Total, other adverse events | Myelodysplastic Syndrome | 39 | 42 |
| Total, other adverse events | Aplastic Anemia | 25 | 29 |
| Total, other adverse events | Other | 25 | 31 |
| Total, serious adverse events | Myelodysplastic Syndrome | 22 | 42 |
| Total, serious adverse events | Aplastic Anemia | 14 | 29 |
| Total, serious adverse events | Other | 10 | 31 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26138856 | RESULT |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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