Trial · NCT00235391
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
NCT00235391, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2011-05-02
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload. Trials, record NCT00235391. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00235391
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
- NCT number
- NCT00235391
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 1,683 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Interventions
- Deferasirox
- First posted
- 2005-10-10
- Last updated
- 2011-06-07
- Results first posted
- 2011-05-02
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) | 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. |
| Secondary | The Change in Serum Ferritin Values From Baseline Through Completion of the Study | Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) | 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. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) | The safety population, comprising all participants who received at least one dose of deferasirox during the study, was used in the analyses. | Participants | NUMBER |
| SECONDARY | The Change in Serum Ferritin Values From Baseline Through Completion of the Study | Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) | Safety population defined as all participants who received at least one dose of study drug. This analysis did not include participants with unknown status at baseline and/or at the end of the study. | Participants | NUMBER |
Source ClinicalTrials.gov
Results by group42
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | 2 to < 6 Years | Number of deaths | 0 | Participants | |
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | 6 to < 12 Years | Number of deaths | 0 | Participants | |
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | 12 to < 16 Years | Number of deaths | 1 | Participants | |
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | 16 to < 50 Years | Number of deaths | 4 | Participants | |
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | 50 to < 65 Years | Number of deaths | 0 | Participants | |
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | ≥ 65 Years | Number of deaths | 0 | Participants | |
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | 2 to < 6 Years | Non-fatal SAEs | 10 | Participants | |
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | 6 to < 12 Years | Non-fatal SAEs | 22 | Participants | |
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | 12 to < 16 Years | Non-fatal SAEs | 27 | Participants | |
| Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events | 16 to < 50 Years | Non-fatal SAEs | 129 | Participants |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 2 to < 6 Years | 97 | |
| Overall Study | STARTED | 6 to < 12 Years | 200 | |
| Overall Study | STARTED | 12 to < 16 Years | 172 | |
| Overall Study | STARTED | 16 to < 50 Years | 1,164 | |
| Overall Study | STARTED | 50 to < 65 Years | 43 | |
| Overall Study | STARTED | ≥ 65 Years | 7 | |
| Overall Study | Deferasirox Treatment | 2 to < 6 Years | 97 | |
| Overall Study | Deferasirox Treatment | 6 to < 12 Years | 200 | |
| Overall Study | Deferasirox Treatment | 12 to < 16 Years | 172 | |
| Overall Study | Deferasirox Treatment | 16 to < 50 Years | 1,164 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events155
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 | Rash | Skin and subcutaneous tissue disorders | All Participants | 85 | 1,683 | |
| serious | Acute chest syndrome | Blood and lymphatic system disorders | All Participants | 2 | 1,683 | |
| serious | Anaemia | Blood and lymphatic system disorders | All Participants | 2 | 1,683 | |
| serious | Anaemia haemolytic autoimmune | Blood and lymphatic system disorders | All Participants | 2 | 1,683 | |
| serious | Coombs positive haemolytic anaemia | Blood and lymphatic system disorders | All Participants | 1 | 1,683 | |
| serious | Haemolytic anaemia | Blood and lymphatic system disorders | All Participants | 1 | 1,683 | |
| serious | Hypersplenism | Blood and lymphatic system disorders | All Participants | 3 | 1,683 | |
| serious | Intravascular haemolysis | Blood and lymphatic system disorders | All Participants | 1 | 1,683 | |
| serious | Leukopenia | Blood and lymphatic system disorders | All Participants | 1 | 1,683 | |
| serious | Neutropenia | Blood and lymphatic system disorders | All Participants | 2 | 1,683 |
1 to 10 of 155Source ClinicalTrials.gov
See all 155 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | All Participants | ||
| Total, other adverse events | All Participants | 85 | 1,683 |
| Total, serious adverse events | All Participants | 194 | 1,683 |
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.
GET https://conductscience.org/api/trials/v1/NCT00235391
GET https://conductscience.org/api/trials/v1/NCT00235391/results?page=2