A Phase 2 interventional study of Deferasirox and Deferoxamine in Cardiac Iron Overload, sponsored by Novartis Pharmaceuticals. Completed at 15 sites in 8 countries. Open to participants aged 10 Years and older. Per ClinicalTrials.gov, last updated 2021-07-26.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment
This study will evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed be deferasirox monotherapy in patients with severe iron overload due to chronic blood transfusions.
156 studies on the registry are indexed under Iron Overload; 6 are open to participants now.
This study's enrollment of 60 is close to the median of 60 across 97 interventional studies indexed under Iron Overload.
Browse Iron Overload studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Deferoxamine combination followed by Deferasirox monotherapy
Drug: Deferasirox and Deferoxamine
Drug pages: Deferasirox · Deferoxamine
Change in Cardiac Iron Content 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.
Time frame: From Baseline to Month 12
Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 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.
Time frame: From the Months 6, 12, 18 and 24
Change in Cardiac Iron Content From Baseline to Month 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).
Time frame: From Baseline to Months 6, 18 and 24
Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 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.
Time frame: From the Months 6, 12, 18 and 24
Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 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.
Time frame: From the Months 6, 12, 18 and 24
Time to Achieve From Baseline (FAS) of at Least 10% at Month 24
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.
Time frame: At 24 months
Cardiac Iron Concentration Levels From Baseline and at Month 6, 12, 18 and 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.
Time frame: From the Baseline, Month 6, 12, 18 and Month 24
The study was conducted at 18 centers in 8 countries.
| Milestone | All Participants |
|---|---|
| Started | 60 |
| Completion of 12 months | 39 |
| Completion of 24 months | 34 |
| Completed | 34 |
| Not completed | 26 |
| Withdrew: Withdrawal by subject | 6 |
| Withdrew: Lost to follow-up | 6 |
| Withdrew: Adverse event | 5 |
| Withdrew: Abnormal test procedure result | 5 |
| Withdrew: Administrative problems | 2 |
| Withdrew: Death | 1 |
| Withdrew: Protocol deviation | 1 |
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.
| ratio | All Participants |
|---|---|
| Change in Cardiac Iron Content From Baseline to Month 12 | 1.09 (1.04 to 1.15) |
The number of evaluable participants at each visit were used as the denominator for the calculation of proportion at each visit.
| percentage of participants | All Participants |
|---|---|
| Month 6 | 12.50 (5.86 to 24.70) |
| Month12 | 19.23 (10.80 to 31.90) |
| Month 18 | 33.33 (19.75 to 50.39) |
| Month 24 | 47.22 (31.99 to 62.99) |
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).
| ratio | All Participants |
|---|---|
| Month 6 | 1.02 (0.98 to 1.07) |
| Month 18 | 1.17 (1.08 to 1.28) |
| Month 24 | 1.30 (1.17 to 1.44) |
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.
| Percent Ejection Fraction | All Participants |
|---|---|
| Baseline | 66.5 ± 5.32 |
| Month 6 | 0.1 ± 4.62 |
| Month 12 | -0.2 ± 4.84 |
| Month 18 | 0.6 ± 7.04 |
| Month 24 | 0.9 ± 5.98 |
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.
| Percent Ejection Fraction | All Participants |
|---|---|
| Baseline | 67.1 ± 5.55 |
| Month 6 | -1.2 ± 5.35 |
| Month 12 | -1.6 ± 4.40 |
| Month 18 | -2.1 ± 6.10 |
| Month 24 | -1.4 ± 4.25 |
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.
| milliseconds/ms | All Participants |
|---|---|
| Time to Achieve From Baseline (FAS) of at Least 10% at Month 24 | 722.0 (520.0 to NA) |
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.
| mg Fe/g dw | All Participants |
|---|---|
| Baseline | 4.18 ± 1.045 |
| Month 6 | 4.31 ± 1.442 |
| Month 12 | 3.93 ± 1.429 |
| Month 18 | 3.51 ± 1.348 |
| Month 24 | 3.14 ± 1.381 |
Collected over All Adverse events are reported in this record from First Patient First Treatment until Last Patient Last Visit, up to 24 months with 28 days of Follow up. Non-serious events are listed at a 2% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| All Participants | 1/60 (1.7%) | 17/60 (28.3%) | 52/60 (86.7%) |
| Event | All Participants |
|---|---|
| Abdominal painGastrointestinal disorders | 2/60 |
| AnaemiaBlood and lymphatic system disorders | 1/60 |
| Dermoid cystCongenital, familial and genetic disorders | 1/60 |
| Irritable bowel syndromeGastrointestinal disorders | 1/60 |
| Pancreatitis acuteGastrointestinal disorders | 1/60 |
| Reflux gastritisGastrointestinal disorders | 1/60 |
| VomitingGastrointestinal disorders | 1/60 |
| AstheniaGeneral disorders | 1/60 |
| PyrexiaGeneral disorders | 1/60 |
| Biliary colicHepatobiliary disorders | 1/60 |
| Event | All Participants |
|---|---|
| PyrexiaGeneral disorders | 14/60 |
| Back painMusculoskeletal and connective tissue disorders | 12/60 |
| Upper respiratory tract infectionInfections and infestations | 10/60 |
| NasopharyngitisInfections and infestations | 9/60 |
| HeadacheNervous system disorders | 9/60 |
| Abdominal painGastrointestinal disorders | 8/60 |
| DiarrhoeaGastrointestinal disorders | 8/60 |
| NauseaGastrointestinal disorders | 8/60 |
| InfluenzaInfections and infestations | 8/60 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 8/60 |
| Age, Continuous(years) | All Participants |
|---|---|
| Mean | 22.8 ± 7.33 |
| Sex: Female, Male(Participants) | All Participants |
|---|---|
| Female | 32 |
| Male | 28 |
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.
Data sharing details ↗ · www.clinicalstudydatarequest.com
This study is completed, as verified in Jul 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Deferasirox · Deferoxamine