Trial · NCT00873041
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
NCT00873041, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2012-09-25
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study. Trials, record NCT00873041. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00873041
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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 Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
- NCT number
- NCT00873041
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 166 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Non-transfusion Dependent Thalassemia
- Interventions
- deferasirox, placebo
- First posted
- 2009-04-01
- Last updated
- 2013-07-09
- Other registries' IDs
- eudract:2007-007000-15
- Results first posted
- 2012-09-25
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52 | Baseline, Week 52 | LIC was measured by magnetic resonance imaging technique at baseline and Week 52. Estimates were obtained from an Analysis of Covariance (ANCOVA) model for change in LIC between baseline and Week 52 with treatment as factor and baseline LIC as covariate. |
| Primary | Extension Study: Percentage of Participants Reaching a Liver Iron Concentration (LIC) < 5 mg Fe/g dw From Core Baseline to End of Extension Study | Core Baseline to End of Extension Study (up to 24 months) | Liver iron concentration was measured at Core Baseline and at the end of the Extension Study. Magnetic Resonance Imaging (MRI) scans were analyzed at a central laboratory to determine the LIC value. The percentage of participants with LIC \< 5 mgFe/g dw (milligram iron/gram dry weight) change from Baseline at the end of the Extension Study is reported. |
| Secondary | Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24 | Baseline, Week 24 | LIC was measured by magnetic resonance imaging technique at baseline and Week 24. Estimates were obtained from an Analysis of Covariance (ANCOVA) model for change in LIC between baseline and Week 24 with treatment as factor and baseline LIC as covariate. |
| Secondary | Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter | Baseline, (Day 286 to End of Study [Day 365]) | Baseline serum ferritin average was the average of all available ferritin values from screening to last sample prior to the first intake of study drug. Fourth quarter serum ferritin average was the average of all serum ferritin values obtained within days 286- End of Study. Change from baseline: fourth quarter serum ferritin average - baseline serum ferritin average. |
| Secondary | Core Study: Change in Serum Ferritin Between Baseline and Second Quarter | Baseline, (Day 106 to Day 195) | Baseline serum ferritin average was the average of all available ferritin values from screening to last sample prior to the first intake of study drug. Second quarter serum ferritin average was the average of all serum ferritin values obtained within days 106-195. Change from baseline: second quarter serum ferritin average - baseline serum ferritin average. |
| Secondary | Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe | 52 Weeks | Percentage of Participants with Mild, Moderate and Severe adverse events (AE) any primary system organ class regardless of study drug relationship. A patient with multiple occurrences of an AE is counted only once in the AE category for that treatment. A patient with multiple severity ratings for an AE while on a treatment is only counted once under the maximum rating. |
| Secondary | Core Study: Change in Liver Iron Concentration (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24 | Baseline, Week 24, Week 52 | LIC was measured by magnetic resonance imaging technique at baseline, Week 24 and Week 52. Dose Doubling (Dose Increases) began at Week 24. |
| Secondary | Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration) | Baseline, 52 weeks | The correlation between serum ferritin and LIC was investigated using a scatter plot with a regression line for the following cases: * Baseline serum ferritin versus baseline LIC * Serum ferritin difference from baseline at fourth quarter versus difference from baseline in LIC at Week 52. A value of 1.0 indicates a perfect correlation. |
| Secondary | Core Study: Change From Baseline in Hemoglobin at Month 12 | Baseline, Month 12 | Blood was collected for Hemoglobin at baseline and Month 12. Change from baseline= Month 12 hemoglobin - baseline hemoglobin. |
| Secondary | Core Study: Change From Baseline in Transferrin Saturation at Month 12 | Baseline, Month 12 | Blood was collected for transferrin saturation at Baseline and Month 12. Change from baseline= Month 12 transferrin saturation - baseline transferrin saturation. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| SECONDARY | Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24 | Baseline, Week 24 | Full Analysis Set. The last available post-baseline LIC was carried forward if no LIC value was available at Week 24. Only patients with both baseline and at least one post-baseline value were included for this analysis. | mg iron (Fe)/g dry weight (dw) | LEAST_SQUARES_MEAN |
| SECONDARY | Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter | Baseline, (Day 286 to End of Study [Day 365]) | Full Analysis set (all randomized patients). Only participants with both baseline and post-baseline values are included in analyses. If serum ferritin was missing during the fourth quarter, the last available average of serum ferritin per quarter was used for the calculation of the change from baseline. | μg/L | MEAN |
| SECONDARY | Core Study: Change in Serum Ferritin Between Baseline and Second Quarter | Baseline, (Day 106 to Day 195) | Full Analysis set (all randomized patients). Only participants with both baseline and post-baseline values are included in analyses. If serum ferritin was missing during the second quarter, the last available average of serum ferritin per quarter was used for the calculation of the change from baseline. | μg/L | MEAN |
| SECONDARY | Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe | 52 Weeks | Safety Analysis Set included all randomized participants who received treatment. | Percentage of participants | NUMBER |
| SECONDARY | Core Study: Change in Liver Iron Concentration (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24 | Baseline, Week 24, Week 52 | Full Analysis Set. The last available post-baseline LIC was carried forward if no LIC value was available at Week 24 and Week 52. Only patients with dose increases after week 24, with both baseline and at least one post-baseline value were included for this analysis. | mg iron (Fe)/g dry weight (dw) | MEAN |
| SECONDARY | Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration) | Baseline, 52 weeks | Participants from the Full Analysis Set (all randomized participants). | Correlation coefficient | NUMBER |
| SECONDARY | Core Study: Change From Baseline in Hemoglobin at Month 12 | Baseline, Month 12 | Full Analysis Set (all randomized participants). Only patients with a value both at baseline and at considered timepoint are included in analyses. | g/L | MEAN |
| SECONDARY | Core Study: Change From Baseline in Transferrin Saturation at Month 12 | Baseline, Month 12 | Full Analysis Set (all randomized patients). Only patients with a value both at baseline and at considered timepoint are included in the analyses. | Percent saturation | MEAN |
| SECONDARY | Core Study: Change in Liver Iron Concentration (LIC) in Placebo Patients From Baseline to Week 52 | Baseline, Week 52 | Safety Analysis Set. The last available post-baseline LIC was carried forward if no LIC value was available at Week 52. Only patients with both baseline and at least one post-baseline value were included for this analysis. | mg iron (Fe)/g dry weight (dw) | MEAN |
| SECONDARY | Core Study: Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results | 52 Weeks | Safety Set included all randomized participants who received treatment. | Percentage of participants | NUMBER |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group86
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24 | 5 mg/kg/Day Deferasirox | -0.87 | Standard Error 0.448 | mg iron (Fe)/g dry weight (dw) | |
| Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24 | 10 mg/kg/Day Deferasirox | -0.90 | Standard Error 0.450 | mg iron (Fe)/g dry weight (dw) | |
| Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24 | Placebo | -0.24 | Standard Error 0.439 | mg iron (Fe)/g dry weight (dw) | |
| Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter | 5 mg/kg/Day Deferasirox | -130.47 | Standard Deviation 260.555 | μg/L | |
| Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter | 10 mg/kg/Day Deferasirox | -249.16 | Standard Deviation 389.356 | μg/L | |
| Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter | Placebo | 128.63 | Standard Deviation 249.689 | μg/L | |
| Core Study: Change in Serum Ferritin Between Baseline and Second Quarter | 5 mg/kg/Day Deferasirox | 8.20 | Standard Deviation 244.443 | μg/L | |
| Core Study: Change in Serum Ferritin Between Baseline and Second Quarter | 10 mg/kg/Day Deferasirox | -17.75 | Standard Deviation 368.812 | μg/L | |
| Core Study: Change in Serum Ferritin Between Baseline and Second Quarter | Placebo | 106.45 | Standard Deviation 330.217 | μg/L | |
| Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe | 5 mg/kg/Day Deferasirox | Mild | 36.4 | Percentage of participants |
1 to 10 of 86Source ClinicalTrials.gov
See all 86 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Core Study | STARTED | 5 mg/kg/Day Deferasirox | 55 | |
| Core Study | STARTED | 10 mg/kg/Day Deferasirox | 55 | |
| Core Study | STARTED | Placebo/Deferasirox | 56 | |
| Core Study | COMPLETED | 5 mg/kg/Day Deferasirox | 48 | |
| Core Study | COMPLETED | 10 mg/kg/Day Deferasirox | 49 | |
| Core Study | COMPLETED | Placebo/Deferasirox | 51 | |
| Core Study | NOT COMPLETED | 5 mg/kg/Day Deferasirox | 7 | |
| Core Study | NOT COMPLETED | 10 mg/kg/Day Deferasirox | 6 | |
| Core Study | NOT COMPLETED | Placebo/Deferasirox | 5 | |
| Extension Study | STARTED | 5 mg/kg/Day Deferasirox | 41 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events264
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 | Deferasirox 5 mg/kg/Day | 6 | 55 | |
| other | Anaemia | Blood and lymphatic system disorders | Deferasirox 10 mg/kg/Day | 4 | 55 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo/Deferasirox Any Dose | 3 | 56 | |
| other | Conjunctivitis | Eye disorders | Deferasirox 5 mg/kg/Day | 3 | 55 | |
| other | Conjunctivitis | Eye disorders | Deferasirox 10 mg/kg/Day | 1 | 55 | |
| other | Conjunctivitis | Eye disorders | Placebo/Deferasirox Any Dose | 1 | 56 | |
| other | Abdominal pain | Gastrointestinal disorders | Deferasirox 5 mg/kg/Day | 4 | 55 | |
| other | Abdominal pain | Gastrointestinal disorders | Deferasirox 10 mg/kg/Day | 4 | 55 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo/Deferasirox Any Dose | 9 | 56 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Deferasirox 5 mg/kg/Day | 4 | 55 |
1 to 10 of 264Source ClinicalTrials.gov
See all 264 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Deferasirox 5 mg/kg/Day | ||
| Total, all-cause mortality | Deferasirox 10 mg/kg/Day | ||
| Total, all-cause mortality | Placebo/Deferasirox Any Dose | ||
| Total, other adverse events | Deferasirox 5 mg/kg/Day | 37 | 55 |
| Total, other adverse events | Deferasirox 10 mg/kg/Day | 45 | 55 |
| Total, other adverse events | Placebo/Deferasirox Any Dose | 48 | 56 |
| Total, serious adverse events | Deferasirox 5 mg/kg/Day | 11 | 55 |
| Total, serious adverse events | Deferasirox 10 mg/kg/Day | 12 | 55 |
| Total, serious adverse events | Placebo/Deferasirox Any Dose | 16 | 56 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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.
For software
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The record and each of its lists are JSON, twenty rows a page. No key.
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