Trial · NCT01459718
Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload
NCT01459718, Phase 2, Terminated, Novartis Pharmaceuticals; results first posted 2019-10-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload. Trials, record NCT01459718. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01459718
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload
- NCT number
- NCT01459718
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 32 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Transfusion-dependent β-thalassemia Patients, Cardiac Iron Overload
- Interventions
- Deferasirox, Deferoxamine (DFO)
- First posted
- 2011-10-26
- Last updated
- 2019-10-23
- Other registries' IDs
- eudract:2009-018091-34
- Results first posted
- 2019-10-23
- Limitations and caveats
- Study was terminated. Efficacy was not powered for analysis due to the low enrollment of only 13 patients.
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients Achieving a Complete Response (CR) | 24 months | Complete Response is defined as patients that stop intensive deferasirox -DFO treatment, at any time point during the 24 months of study, based on an improvement in the cardiac Magnetic Resonance Imaging T2 star technique (MRI T2\*) value being \>10ms, and continue to be treated with deferasirox monotherapy without any further need for reverting back to intensive iron chelation treatment during the 24 months of study. |
| Primary | Number of Patients Achieving a Partial Response (PR) | 24 months | Partial Response is defined as patients that stop intensive deferasirox -DFO treatment at any time point during the 24 months study and transition to receive deferasirox monotherapy, but due to a deterioration in cardiac MRI T2\* to a value \< 10 ms revert back to intensive deferasirox -DFO iron chelation therapy during the 24 months of study. |
| Primary | Number of Patients With Stable Disease (SD) | 24 months | Stable Disease is defined as those patients that never achieved an improvement in the cardiac MRI T2\* to values \>10ms during the 24 months of study. |
| Secondary | Change From Baseline in Cardiac Iron Overload of Patients in Intensive Iron Chelation Therapy Consisting of Deferasirox-DFO and After Transition to Deferasirox Monotherapy | Baseline, 6, 12, 18, 24 months | Cardiac iron overload was determined by cardiac MRI T2\*. Cardiac iron overload also was measured by the monthly velocity of heart MRI T2\*. |
| Secondary | Time to Response | 24 months | Time to response was defined as the time from baseline when the participant had severe cardiac iron overload to the time when the participant achieved mild/moderate cardiac overload (T2\*\>10 milliseconds \[ms\]). |
| Secondary | Change From Baseline in Liver Iron Concentration (LIC) | Baseline, 6, 12, 18, 24 months | Change from baseline in LIC was determined by change in liver MRI T2\*. |
| Secondary | Correlation Between Change From Baseline in Serum Ferritin and LIC Levels | Baseline, 6, 12, 18, 24 months | Spearman correlation coefficients between serum ferritin and LIC changes from baseline levels were reported. |
| Secondary | Left Ventricular Ejection Fraction (LVEF) | 6, 12, 18, 24 months | LVEF % was measured by cardiac magnetic resonance (CMR). |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Patients Achieving a Complete Response (CR) | 24 months | The full analysis set included all the participants entered in the study with at least a valid post-baseline assessment of the primary efficacy variable. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Patients Achieving a Partial Response (PR) | 24 months | The full analysis set included all the participants entered in the study with at least a valid post-baseline assessment of the primary efficacy variable. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Patients With Stable Disease (SD) | 24 months | The full analysis set included all the participants entered in the study with at least a valid post-baseline assessment of the primary efficacy variable. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Cardiac Iron Overload of Patients in Intensive Iron Chelation Therapy Consisting of Deferasirox-DFO and After Transition to Deferasirox Monotherapy | Baseline, 6, 12, 18, 24 months | The full analysis set included all the participants entered in the study with at least a valid post-baseline assessment of the primary efficacy variable. Here 'n' number analyzed signifies number of participants evaluable at each time point. | Milliseconds (ms) | MEAN |
| SECONDARY | Time to Response | 24 months | The full analysis set included all participants entered in the study with at least a valid post-baseline assessment of the primary efficacy variable. Here 'N' (overall number of participants analyzed) signifies number of participants evaluable for this outcome measure at the specified time-point. | ms | MEAN |
| SECONDARY | Change From Baseline in Liver Iron Concentration (LIC) | Baseline, 6, 12, 18, 24 months | The full analysis set included all the participants entered in the study with at least a valid post-baseline assessment of the primary efficacy variable. Here 'n' number analyzed signifies number of participants evaluable at each time point. | mg of iron/gram of dry weight of liver | MEAN |
| SECONDARY | Correlation Between Change From Baseline in Serum Ferritin and LIC Levels | Baseline, 6, 12, 18, 24 months | Full analysis set included all participants entered in study with at least a valid post-baseline assessment of the primary efficacy variable. Here 'N' (overall number of participants analyzed) signifies number of participants evaluable for this outcome measure and 'n' (number analyzed) signifies number of participants evaluable at each time point. | Spearman correlation coefficient | NUMBER |
| SECONDARY | Left Ventricular Ejection Fraction (LVEF) | 6, 12, 18, 24 months | The full analysis set included all the participants entered in the study with at least a valid post-baseline assessment of the primary efficacy variable. Here 'n' number analyzed signifies number of participants evaluable at each time point. | Percentage of ejection fraction | MEAN |
Source ClinicalTrials.gov
Results by group21
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Patients Achieving a Complete Response (CR) | Deferasirox / Deferasirox + Deferoxamine (DFO) | 4 | Participants | ||
| Number of Patients Achieving a Partial Response (PR) | Deferasirox / Deferasirox + Deferoxamine (DFO) | 0 | Participants | ||
| Number of Patients With Stable Disease (SD) | Deferasirox / Deferasirox + Deferoxamine (DFO) | 8 | Participants | ||
| Change From Baseline in Cardiac Iron Overload of Patients in Intensive Iron Chelation Therapy Consisting of Deferasirox-DFO and After Transition to Deferasirox Monotherapy | Deferasirox / Deferasirox + Deferoxamine (DFO) | 6 months | 0.1 | Standard Deviation 1.4 | Milliseconds (ms) |
| Change From Baseline in Cardiac Iron Overload of Patients in Intensive Iron Chelation Therapy Consisting of Deferasirox-DFO and After Transition to Deferasirox Monotherapy | Deferasirox / Deferasirox + Deferoxamine (DFO) | 12 months | 0.7 | Standard Deviation 1.9 | Milliseconds (ms) |
| Change From Baseline in Cardiac Iron Overload of Patients in Intensive Iron Chelation Therapy Consisting of Deferasirox-DFO and After Transition to Deferasirox Monotherapy | Deferasirox / Deferasirox + Deferoxamine (DFO) | 18 months | 1.3 | Standard Deviation 2.4 | Milliseconds (ms) |
| Change From Baseline in Cardiac Iron Overload of Patients in Intensive Iron Chelation Therapy Consisting of Deferasirox-DFO and After Transition to Deferasirox Monotherapy | Deferasirox / Deferasirox + Deferoxamine (DFO) | 24 months | 2.4 | Standard Deviation 3.9 | Milliseconds (ms) |
| Time to Response | Deferasirox / Deferasirox + Deferoxamine (DFO) | 13.0 | Standard Deviation 1.1 | ms | |
| Change From Baseline in Liver Iron Concentration (LIC) | Deferasirox / Deferasirox + Deferoxamine (DFO) | 6 months | -5 | Standard Deviation 7.7 | mg of iron/gram of dry weight of liver |
| Change From Baseline in Liver Iron Concentration (LIC) | Deferasirox / Deferasirox + Deferoxamine (DFO) | 12 months | -10.3 | Standard Deviation 9.2 | mg of iron/gram of dry weight of liver |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Deferasirox / Deferasirox + Deferoxamine (DFO) | 13 | |
| Overall Study | Safety Analysis Set | Deferasirox / Deferasirox + Deferoxamine (DFO) | 13 | |
| Overall Study | COMPLETED | Deferasirox / Deferasirox + Deferoxamine (DFO) | 10 | |
| Overall Study | NOT COMPLETED | Deferasirox / Deferasirox + Deferoxamine (DFO) | 3 |
Source ClinicalTrials.gov
Adverse events86
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 | Thrombocytosis | Blood and lymphatic system disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 | |
| other | Angina pectoris | Cardiac disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 | |
| other | Atrial fibrillation | Cardiac disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 | |
| other | Sinus tachycardia | Cardiac disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 | |
| other | Ear pain | Ear and labyrinth disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 | |
| other | Hypoacusis | Ear and labyrinth disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 | |
| other | Tinnitus | Ear and labyrinth disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 | |
| other | Hyperthyroidism | Endocrine disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 | |
| other | Abdominal distension | Gastrointestinal disorders | Deferasirox / Deferasirox + Deferoxamine (DFO) | 1 | 13 |
1 to 10 of 86Source ClinicalTrials.gov
See all 86 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Deferasirox / Deferasirox + Deferoxamine (DFO) | ||
| Total, other adverse events | Deferasirox / Deferasirox + Deferoxamine (DFO) | 12 | 13 |
| Total, serious adverse events | Deferasirox / Deferasirox + Deferoxamine (DFO) | 5 | 13 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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GET https://conductscience.org/api/trials/v1/NCT01459718/results?page=2