Trial · NCT00171301
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
NCT00171301, Phase 4, Completed, Novartis Pharmaceuticals; results first posted 2011-07-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration). Trials, record NCT00171301. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00171301
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
- NCT number
- NCT00171301
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 233 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia
- Interventions
- Deferasirox
- First posted
- 2005-09-15
- Last updated
- 2011-08-31
- Results first posted
- 2011-07-29
- Limitations and caveats
An internal review revealed major Good Clinical Practice violations at 2 sites in Saudi Arabia: 602 for core + extension, 601 for 2-yr extension. Therefore data was excluded (completely for 602 + partly for 601) from analyses.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age | From Core Study Baseline, to Extension End of Study, Up to 3 Years | Success was defined as the percentage of participants with decreased liver iron content (LIC) at the end of extension study compared to core baseline (BL) LIC. Success Criteria: For participants with Baseline LIC from 1 - \<7 mg Fe/g dw, success was achieved if LIC level maintained at 1 - \<7 mg Fe/g dw. For participants with Baseline LIC ≥7 - \<10 mg Fe/g dw, success was achieved if LIC dropped to between 1 and \< 7 mg Fe/g dw. For participants with Baseline LIC ≥10 mg Fe/g dw, success was achieved if LIC dropped by at least 3 mg Fe/g dw. LIC was measured by biopsy or magnetic resonance imaging. |
| Primary | Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category | From Baseline of Core Study to End of Extension Study, up to 3 years | Liver MRI or Liver Biopsy was performed at the core study baseline (BL) and then 1 year and 2 years in the core study, baseline of the extension study and time of discontinuation from the extension visit (end of study). Liver iron content (LIC) is reported in milligram Iron per gram dry weight (mg Fe/g dw). Absolute change in LIC from core study baseline to the end of the extension study is presented for participants with the following two core study baseline LIC levels: 1-\<7 mg Fe/g dw and ≥7 mg Fe/g dw. |
| Secondary | Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study | From Baseline of Core Study to End of Extension Study, up to 3 years | Serum Levels were assessed at core study baseline (BL), 1 year, 2 years in core study, baseline of extension study and time of discontinuation from the extension visit (end of study) in monthly intervals. Serum Ferritin is reported in micrograms per Liter (µg/L). |
| Secondary | Absolute Change in Serum Ferritin Level for All Participants Measured From Core Study Baseline (BL) to End of Extension Study, by Baseline Liver Iron Content (LIC) | From Baseline of Core Study to End of Extension Study, up to 3 years | Serum Levels were assessed at core study baseline (BL) and then 1 year and 2 years in core study, baseline of extension study and time of discontinuation from the extension visit (end of study). Serum Ferritin is reported in micrograms per Liter. Absolute change in Serum Ferritin from core study baseline to the end of the extension study is presented for participants with the following two core study baseline LIC levels: 1-\<7 mg Fe/g dw and ≥7 mg Fe/g dw. |
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 | Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age | From Core Study Baseline, to Extension End of Study, Up to 3 Years | The primary analysis will be on the intent-to-treat (ITT) population. ITT population includes all participants who performed the core end of study (EOS) visit evaluation and assessments and were included in the extension study. n is the number of participants analyzed in each category. | percentage of participants | MEAN |
| PRIMARY | Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category | From Baseline of Core Study to End of Extension Study, up to 3 years | Participants from the Intent-to-Treat (ITT) population for whom LIC data was available at Core Study baseline and at the End of Extension Study. n is the number of participants analyzed in each category. | mg Fe/g dw | MEAN |
| SECONDARY | Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study | From Baseline of Core Study to End of Extension Study, up to 3 years | Participants from the Intent-to-Treat (ITT) population for whom Serum Ferritin data was available at Core Study baseline and at the End of Extension Study. | µg/L | MEAN |
| SECONDARY | Absolute Change in Serum Ferritin Level for All Participants Measured From Core Study Baseline (BL) to End of Extension Study, by Baseline Liver Iron Content (LIC) | From Baseline of Core Study to End of Extension Study, up to 3 years | Participants from the Intent-to-Treat (ITT) population for whom Serum Ferritin data was available at Core Study baseline and at the End of Extension Study. n is the number of participants analyzed in each category. | µg/L | MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age | Deferasirox (Between 2 <16 Years) | Core Baseline LIC 1-<7 mg Fe/g dw (n=20, 2) | 75.0 | 95% Confidence Interval 56.0 to 94.0 | percentage of participants |
| Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age | Deferasirox (16 Years or Older) | Core Baseline LIC 1-<7 mg Fe/g dw (n=20, 2) | 100 | 95% Confidence Interval 15.8 to 100 | percentage of participants |
| Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age | Deferasirox (Between 2 <16 Years) | Core Baseline LIC 7-<10 mg Fe/g dw (n=18, 8) | 72.2 | 95% Confidence Interval 51.5 to 92.9 | percentage of participants |
| Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age | Deferasirox (16 Years or Older) | Core Baseline LIC 7-<10 mg Fe/g dw (n=18, 8) | 50.0 | 95% Confidence Interval 15.7 to 84.3 | percentage of participants |
| Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age | Deferasirox (Between 2 <16 Years) | Core Baseline LIC ≥10 mg Fe/g dw (n=124, 61) | 76.6 | 95% Confidence Interval 69.2 to 84.1 | percentage of participants |
| Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age | Deferasirox (16 Years or Older) | Core Baseline LIC ≥10 mg Fe/g dw (n=124, 61) | 73.8 | 95% Confidence Interval 62.7 to 84.8 | percentage of participants |
| Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category | Deferasirox (Between 2 <16 Years) | Core Baseline LIC 1-<7 mg Fe/g dw (n=12, 0) | -1.32 | Standard Deviation 3.125 | mg Fe/g dw |
| Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category | Deferasirox (16 Years or Older) | Core Baseline LIC 1-<7 mg Fe/g dw (n=12, 0) | NA | Standard Deviation NA | mg Fe/g dw |
| Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category | Deferasirox (Between 2 <16 Years) | Core Baseline LIC ≥7 mg Fe/g dw (n=107, 41) | -9.03 | Standard Deviation 9.260 | mg Fe/g dw |
| Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category | Deferasirox (16 Years or Older) | Core Baseline LIC ≥7 mg Fe/g dw (n=107, 41) | -8.39 | Standard Deviation 11.312 | mg Fe/g dw |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Deferasirox (Between 2 <16 Years ) | 162 | |
| Overall Study | STARTED | Deferasirox (16 Years or Older) | 71 | |
| Overall Study | Received Deferasirox | Deferasirox (Between 2 <16 Years ) | 162 | |
| Overall Study | Received Deferasirox | Deferasirox (16 Years or Older) | 69 | |
| Overall Study | COMPLETED | Deferasirox (Between 2 <16 Years ) | 154 | |
| Overall Study | COMPLETED | Deferasirox (16 Years or Older) | 62 | |
| Overall Study | NOT COMPLETED | Deferasirox (Between 2 <16 Years ) | 8 | |
| Overall Study | NOT COMPLETED | Deferasirox (16 Years or Older) | 9 |
Source ClinicalTrials.gov
Adverse events94
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 | Nausea | Gastrointestinal disorders | Deferasirox (Between 2 <16 Years) | 1 | 162 | |
| other | Nausea | Gastrointestinal disorders | Deferasirox (16 Years or Older) | 5 | 69 | |
| other | Vomiting | Gastrointestinal disorders | Deferasirox (Between 2 <16 Years) | 17 | 162 | |
| other | Vomiting | Gastrointestinal disorders | Deferasirox (16 Years or Older) | 5 | 69 | |
| other | Pain | General disorders | Deferasirox (Between 2 <16 Years) | 0 | 162 | |
| other | Pain | General disorders | Deferasirox (16 Years or Older) | 5 | 69 | |
| other | Pyrexia | General disorders | Deferasirox (Between 2 <16 Years) | 3 | 162 | |
| other | Pyrexia | General disorders | Deferasirox (16 Years or Older) | 6 | 69 | |
| other | Gastroenteritis | Infections and infestations | Deferasirox (Between 2 <16 Years) | 1 | 162 | |
| other | Gastroenteritis | Infections and infestations | Deferasirox (16 Years or Older) | 4 | 69 |
1 to 10 of 94Source ClinicalTrials.gov
See all 94 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Deferasirox (Between 2 <16 Years) | ||
| Total, all-cause mortality | Deferasirox (16 Years or Older) | ||
| Total, other adverse events | Deferasirox (Between 2 <16 Years) | 54 | 162 |
| Total, other adverse events | Deferasirox (16 Years or Older) | 33 | 69 |
| Total, serious adverse events | Deferasirox (Between 2 <16 Years) | 5 | 162 |
| Total, serious adverse events | Deferasirox (16 Years or Older) | 12 | 69 |
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 |
|---|
| Deferasirox (Between 2 <16 Years) vs Deferasirox (16 Years or Older): 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/NCT00171301
GET https://conductscience.org/api/trials/v1/NCT00171301/events?page=2