Trial · NCT00303329
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
NCT00303329, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2011-05-09
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients. Trials, record NCT00303329. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00303329
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
- NCT number
- NCT00303329
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 184 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Anemia, Hemosiderosis
- Interventions
- Deferasirox
- First posted
- 2006-03-16
- Last updated
- 2011-05-09
- Results first posted
- 2011-05-09
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths | Core study Baseline to the end of the study (up to 60 months) | Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect. |
| Secondary | The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study | Core study Baseline to end of extension study (up to 60 months) | Liver iron concentration was monitored at the start of the core study, the end of the core study, and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. |
| Secondary | The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study | Core study Baseline to end of extension study (up to 60 months) | Liver iron concentration was monitored at the end of the core study and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. Pediatric participants or participants with a medical contraindication to liver biopsy were allowed the use of SQUID in the extension study. |
| Secondary | The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study | Core study Baseline to end of extension study (up to 60 months) | Serum ferritin was monitored monthly and the dose of deferasirox was increased or decreased in steps of 5 to 10 mg/kg/day up to a maximum of 40 mg/kg/day if appropriate, every 3 months. If serum ferritin fell to 500 ng/mL or lower on two consecutive study visits, an interruption of treatment until serum ferritin was more than 500 ng/mL was considered. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths | Core study Baseline to the end of the study (up to 60 months) | The safety analysis set comprised all participants who received at least one dose of deferasirox during either the core or extension studies. | Participants | NUMBER |
| SECONDARY | The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study | Core study Baseline to end of extension study (up to 60 months) | The full analysis Set (FAS) comprised all participants who received at least one dose of deferasirox during either the core or extension studies. | mg Fe/g dw | MEAN |
| SECONDARY | The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study | Core study Baseline to end of extension study (up to 60 months) | The FAS comprised all participants who received at least one dose of deferasirox during either the core or extension studies. | mg Fe/g dw | MEAN |
| SECONDARY | The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study | Core study Baseline to end of extension study (up to 60 months) | The FAS comprised all participants who received at least one dose of deferasirox during either the core or extension studies. | μg/L | MEAN |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths | β-thalassemia Patients | Adverse Events | 85 | Participants | |
| The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths | Rare Anemias Patients | Adverse Events | 99 | Participants | |
| The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths | β-thalassemia Patients | Serious Adverse Events | 36 | Participants | |
| The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths | Rare Anemias Patients | Serious Adverse Events | 50 | Participants | |
| The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths | β-thalassemia Patients | Deaths | 2 | Participants | |
| The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths | Rare Anemias Patients | Deaths | 12 | Participants | |
| The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study | β-thalassemia Patients | -5.17 | Standard Deviation 11.659 | mg Fe/g dw | |
| The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study | Rare Anemias Patients | -5.10 | Standard Deviation 7.756 | mg Fe/g dw | |
| The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study | β-thalassemia Patients | 0.19 | Standard Deviation 8.577 | mg Fe/g dw | |
| The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study | Rare Anemias Patients | -2.04 | Standard Deviation 4.521 | mg Fe/g dw |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | β-thalassemia Patients | 85 | |
| Overall Study | STARTED | Rare Anemias Patients | 99 | |
| Overall Study | COMPLETED | β-thalassemia Patients | 50 | |
| Overall Study | COMPLETED | Rare Anemias Patients | 37 | |
| Overall Study | NOT COMPLETED | β-thalassemia Patients | 35 | |
| Overall Study | NOT COMPLETED | Rare Anemias Patients | 62 |
Source ClinicalTrials.gov
Adverse events524
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 | Lymphadenopathy | Blood and lymphatic system disorders | Beta-thalassemia | 5 | 85 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Rare Anemias | 1 | 99 | |
| other | Palpitations | Cardiac disorders | Beta-thalassemia | 12 | 85 | |
| other | Palpitations | Cardiac disorders | Rare Anemias | 8 | 99 | |
| other | Tachycardia | Cardiac disorders | Beta-thalassemia | 12 | 85 | |
| other | Tachycardia | Cardiac disorders | Rare Anemias | 6 | 99 | |
| other | Ear pain | Ear and labyrinth disorders | Beta-thalassemia | 10 | 85 | |
| other | Ear pain | Ear and labyrinth disorders | Rare Anemias | 6 | 99 | |
| other | Tinnitus | Ear and labyrinth disorders | Beta-thalassemia | 6 | 85 | |
| other | Tinnitus | Ear and labyrinth disorders | Rare Anemias | 0 | 99 |
1 to 10 of 524Source ClinicalTrials.gov
See all 524 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Beta-thalassemia | ||
| Total, all-cause mortality | Rare Anemias | ||
| Total, other adverse events | Beta-thalassemia | 84 | 85 |
| Total, other adverse events | Rare Anemias | 96 | 99 |
| Total, serious adverse events | Beta-thalassemia | 36 | 85 |
| Total, serious adverse events | Rare Anemias | 50 | 99 |
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Beta-thalassemia vs Rare Anemias: serious adverse events |
Source Trial comparisons
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/NCT00303329
GET https://conductscience.org/api/trials/v1/NCT00303329/events?page=2