Trial · NCT01254227
Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron Overload
NCT01254227, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2021-06-24
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron Overload. Trials, record NCT01254227. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01254227
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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 Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron Overload
- NCT number
- NCT01254227
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 60 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Cardiac Iron Overload
- Interventions
- Deferasirox and Deferoxamine
- First posted
- 2010-12-06
- Last updated
- 2021-07-26
- Other registries' IDs
- eudract:2010-021062-29
- Results first posted
- 2021-06-24
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Cardiac Iron Content From Baseline to Month 12 | From Baseline to Month 12 | Cardiac T2\* is the most sensitive and reproducible test in detecting myocardial iron load. A cardiac T2\* value of \<10 ms is defined as severe cardiac iron overload. Participants who do not have baseline T2\* or do not have any post-baseline T2\* are excluded from the analysis. |
| Secondary | Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24 | From the Months 6, 12, 18 and 24 | The number of evaluable participants at each visit were used as the denominator for the calculation of proportion at each visit. |
| Secondary | Change in Cardiac Iron Content From Baseline to Month 6,18 and 24 | From Baseline to Months 6, 18 and 24 | The change in cardiac iron content was calculated as ratio of Cardiac T2\* at different time points; the efficacy endpoint analyses were performed on the Full Analysis Set (FAS). |
| Secondary | Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24 | From the Months 6, 12, 18 and 24 | Magnetic resonance imaging (MRI)-measured cardiac T2\* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2\* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader. |
| Secondary | Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 6, 12, 18 and 24 | From the Months 6, 12, 18 and 24 | Magnetic resonance imaging (MRI)-measured cardiac T2\* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2\* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader. |
| Secondary | Time to Achieve From Baseline (FAS) of at Least 10% at Month 24 | At 24 months | Time from date of start of study treatment to date when first achieving T2\* ≥ 10 ms (but at least 10% relative increase from baseline) was summarized using the reverse Kaplan-Meier estimates (1 - Kaplan-Meier estimates) for the FAS. |
| Secondary | Cardiac Iron Concentration Levels From Baseline and at Month 6, 12, 18 and 24 | From the Baseline, Month 6, 12, 18 and Month 24 | Cardiac iron concentration (mg Fe/g dw) was quantified using the formula (cardiac iron concentration (mg Fe/g dw) = 45 \* T2\* (ms) \^ (-1.22) and analyzed over time. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Cardiac Iron Content From Baseline to Month 12 | From Baseline to Month 12 | The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint. | ratio | GEOMETRIC_MEAN |
| SECONDARY | Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24 | From the Months 6, 12, 18 and 24 | The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint. Here, Number of participants analyzed included all participants who were evaluable for the specified timepoints. | percentage of participants | GEOMETRIC_MEAN |
| SECONDARY | Change in Cardiac Iron Content From Baseline to Month 6,18 and 24 | From Baseline to Months 6, 18 and 24 | The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint. Here, Number of participants analyzed included all participants who were evaluable for the specified timepoints. | ratio | GEOMETRIC_MEAN |
| SECONDARY | Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24 | From the Months 6, 12, 18 and 24 | The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint. | Percent Ejection Fraction | MEAN |
| SECONDARY | Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 6, 12, 18 and 24 | From the Months 6, 12, 18 and 24 | The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint. | Percent Ejection Fraction | MEAN |
| SECONDARY | Time to Achieve From Baseline (FAS) of at Least 10% at Month 24 | At 24 months | The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint. | milliseconds/ms | MEDIAN |
| SECONDARY | Cardiac Iron Concentration Levels From Baseline and at Month 6, 12, 18 and 24 | From the Baseline, Month 6, 12, 18 and Month 24 | The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint. | mg Fe/g dw | MEAN |
Source ClinicalTrials.gov
Results by group24
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Cardiac Iron Content From Baseline to Month 12 | All Participants | 1.09 | 95% Confidence Interval 1.04 to 1.15 | ratio | |
| Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24 | All Participants | Month 6 | 12.50 | 95% Confidence Interval 5.86 to 24.70 | percentage of participants |
| Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24 | All Participants | Month12 | 19.23 | 95% Confidence Interval 10.80 to 31.90 | percentage of participants |
| Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24 | All Participants | Month 18 | 33.33 | 95% Confidence Interval 19.75 to 50.39 | percentage of participants |
| Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24 | All Participants | Month 24 | 47.22 | 95% Confidence Interval 31.99 to 62.99 | percentage of participants |
| Change in Cardiac Iron Content From Baseline to Month 6,18 and 24 | All Participants | Month 6 | 1.02 | 95% Confidence Interval 0.98 to 1.07 | ratio |
| Change in Cardiac Iron Content From Baseline to Month 6,18 and 24 | All Participants | Month 18 | 1.17 | 95% Confidence Interval 1.08 to 1.28 | ratio |
| Change in Cardiac Iron Content From Baseline to Month 6,18 and 24 | All Participants | Month 24 | 1.30 | 95% Confidence Interval 1.17 to 1.44 | ratio |
| Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24 | All Participants | Baseline | 66.5 | Standard Deviation 5.32 | Percent Ejection Fraction |
| Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24 | All Participants | Month 6 | 0.1 | Standard Deviation 4.62 | Percent Ejection Fraction |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | All Participants | 60 | |
| Overall Study | Completion of 12 Months | All Participants | 39 | |
| Overall Study | Completion of 24 Months | All Participants | 34 | |
| Overall Study | COMPLETED | All Participants | 34 | |
| Overall Study | NOT COMPLETED | All Participants | 26 |
Source ClinicalTrials.gov
Adverse events93
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 | All Participants | 2 | 60 | |
| other | Atrioventricular block first degree | Cardiac disorders | All Participants | 3 | 60 | |
| other | Palpitations | Cardiac disorders | All Participants | 2 | 60 | |
| other | Sinus tachycardia | Cardiac disorders | All Participants | 2 | 60 | |
| other | Tachycardia | Cardiac disorders | All Participants | 2 | 60 | |
| other | Conductive deafness | Ear and labyrinth disorders | All Participants | 2 | 60 | |
| other | Deafness bilateral | Ear and labyrinth disorders | All Participants | 2 | 60 | |
| other | Deafness neurosensory | Ear and labyrinth disorders | All Participants | 3 | 60 | |
| other | Eustachian tube dysfunction | Ear and labyrinth disorders | All Participants | 2 | 60 | |
| other | Conjunctivitis | Eye disorders | All Participants | 2 | 60 |
1 to 10 of 93Source ClinicalTrials.gov
See all 93 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | All Participants | 1 | 60 |
| Total, other adverse events | All Participants | 52 | 60 |
| Total, serious adverse events | All Participants | 17 | 60 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 25934475 | DERIVED |
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01254227
GET https://conductscience.org/api/trials/v1/NCT01254227/results?page=2