A Phase 3 interventional study of Deferasirox in Thalassemia, Sickle Cell Disease and Diamond Blackfan Anemia, sponsored by Novartis Pharmaceuticals. Completed at 141 sites in 12 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2011-06-07.
Sponsored by Novartis Pharmaceuticals · Phase 3, Interventional, and Treatment
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators.
1,733 studies on the registry are indexed under Anemia; 246 are open to participants now.
This study's enrollment of 1,683 is above the median of 94 across 1,291 interventional studies indexed under Anemia.
Browse Anemia studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
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Cannot be adequately treated with a locally approved iron chelator due to one of the following reasons:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Starting dose was determined by the frequency of blood transfusions and recommended initial daily dose of deferasirox is 20 mg/kg body weight for patients receiving blood transfusion, 10 mg/kg for patients receiving less frequent transfusion/exchange transfusion and 30 mg/kg for patients receiving more frequent blood transfusions.
Drug: Deferasirox
125 mg, 250 mg and 500 mg tablets. Dosage was calculated based on participant's body weight. Tablets were dispersed in water, orange or apple juice and taken orally once a day.
Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.
Time frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
The Change in Serum Ferritin Values From Baseline Through Completion of the Study
The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (\<1000), (1000-\<2500), (2500-\<4000), (4000-\<5500), (5500-\<7000) and (\>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.
Time frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
| Milestone | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years |
|---|---|---|---|---|---|---|
| Started | 97 | 200 | 172 | 1164 | 43 | 7 |
| Deferasirox treatment | 97 | 200 | 172 | 1164 | 43 | 7 |
| Completed | 85 | 182 | 148 | 944 | 31 | 3 |
| Not completed | 12 | 18 | 24 | 220 | 12 | 4 |
| Withdrew: Adverse event | 4 | 5 | 10 | 72 | 5 | 2 |
| Withdrew: Abnormal laboratory value(s) | 3 | 5 | 4 | 27 | 1 | 2 |
| Withdrew: Unsatisfactory therapeutic effect | 0 | 2 | 2 | 17 | 0 | 0 |
| Withdrew: Condition no longer requires treatment | 3 | 0 | 0 | 11 | 1 | 0 |
| Withdrew: Protocol deviation | 0 | 0 | 1 | 11 | 0 | 0 |
| Withdrew: Withdrawal by subject | 2 | 3 | 3 | 61 | 5 | 0 |
| Withdrew: Lost to follow-up | 0 | 3 | 3 | 16 | 0 | 0 |
| Withdrew: Administrative problems | 0 | 0 | 0 | 1 | 0 | 0 |
| Withdrew: Death | 0 | 0 | 1 | 4 | 0 | 0 |
Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.
| Participants | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years |
|---|---|---|---|---|---|---|
| Number of deaths | 0 | 0 | 1 | 4 | 0 | 0 |
| Non-fatal SAEs | 10 | 22 | 27 | 129 | 4 | 2 |
| AEs leading to discontinuation | 4 | 5 | 10 | 75 | 5 | 2 |
| AEs leading dose adjustment/temporary interruption | 15 | 31 | 28 | 209 | 11 | 2 |
The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (\<1000), (1000-\<2500), (2500-\<4000), (4000-\<5500), (5500-\<7000) and (\>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.
| Participants | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years |
|---|---|---|---|---|---|---|
| Improvement | 17 | 27 | 26 | 206 | 4 | 2 |
| No Change | 63 | 106 | 87 | 570 | 31 | 3 |
| Worsening | 16 | 63 | 55 | 341 | 7 | 1 |
Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| All Participants | — | 194/1,683 (11.5%) | 85/1,683 (5.1%) |
| Event | All Participants |
|---|---|
| Sickle cell anaemia with crisisCongenital, familial and genetic disorders | 27/1683 |
| PyrexiaGeneral disorders | 9/1683 |
| Cardiac failureCardiac disorders | 6/1683 |
| CholelithiasisHepatobiliary disorders | 6/1683 |
| Non-cardiac chest painGeneral disorders | 5/1683 |
| Viral infectionInfections and infestations | 5/1683 |
| PregnancyPregnancy, puerperium and perinatal conditions | 5/1683 |
| RashSkin and subcutaneous tissue disorders | 5/1683 |
| SplenomegalyBlood and lymphatic system disorders | 4/1683 |
| GastritisGastrointestinal disorders | 4/1683 |
| Event | All Participants |
|---|---|
| RashSkin and subcutaneous tissue disorders | 85/1683 |
| Age Continuous(years) | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years | Total |
|---|---|---|---|---|---|---|---|
| Mean | 3.48 ± 1.13 | 8.52 ± 1.64 | 13.48 ± 1.20 | 28.91 ± 8.06 | 54.58 ± 3.92 | 70.14 ± 3.98 | 24.27 ± 12.63 |
| Sex: Female, Male(Participants) | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years | Total |
|---|---|---|---|---|---|---|---|
| Female | 44 | 101 | 81 | 633 | 32 | 5 | 896 |
| Male | 53 | 99 | 91 | 531 | 11 | 2 | 787 |
| Baseline Disease Characteristics(participants) | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years | Total |
|---|---|---|---|---|---|---|---|
| Beta-Thalassemia Major | 74 | 118 | 114 | 905 | 10 | 0 | 1221 |
| Beta-Thalassemia Intermedia | 1 | 22 | 16 | 94 | 20 | 3 | 156 |
| Sickle Cell Disease | 8 | 41 | 31 | 93 | 2 | 1 | 176 |
| Diamond-Blackfan Anemia | 4 | 7 | 8 | 24 | 0 | 0 | 43 |
| Other Diseases | 10 | 12 | 3 | 48 | 11 | 3 | 87 |
| Prior Chelation Drug Therapy(participants) | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years | Total |
|---|---|---|---|---|---|---|---|
| Deferoxamine | 80 | 167 | 137 | 745 | 31 | 5 | 1165 |
| Deferiprone | 1 | 8 | 11 | 146 | 6 | 1 | 173 |
| Deferoxamine and Deferiprone | 2 | 16 | 21 | 269 | 6 | 0 | 314 |
| Other Chelation Drug | 6 | 6 | 3 | 4 | 0 | 0 | 19 |
| Prior Chelatation Drug Information Missing | 8 | 3 | 0 | 0 | 0 | 1 | 12 |
| Reason for inadequate prior chelation therapy(participants) | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years | Total |
|---|---|---|---|---|---|---|---|
| Therapy non-compliance | 57 | 125 | 106 | 659 | 25 | 2 | 974 |
| Therapy contraindication | 1 | 7 | 6 | 29 | 1 | 1 | 45 |
| Therapy unacceptable toxicity | 11 | 29 | 22 | 131 | 6 | 1 | 200 |
| Therapy poor response | 20 | 34 | 38 | 267 | 8 | 0 | 367 |
| Therapy unacceptable discomfort | 2 | 2 | 0 | 78 | 3 | 2 | 87 |
| Reason for inadequate therapy information missing | 6 | 3 | 0 | 0 | 0 | 1 | 10 |
Showing the first 100 of 141 sites across 12 countries.
This study is completed, as verified in Jun 2011. You cannot join it, but the record below documents what was studied.
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Novartis Pharmaceuticals