Trial · NCT00390858
A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
NCT00390858, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2011-08-15
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload. Trials, record NCT00390858. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00390858
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
- NCT number
- NCT00390858
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 40 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
- Interventions
- Deferasirox
- First posted
- 2006-10-20
- Last updated
- 2017-03-20
- Results first posted
- 2011-08-15
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Participants With Adverse Events by Primary System Organ Class (SOC) | 4 year extension + core 1 year | Safety parameters were measured by the number and type of adverse events (AEs). An adverse event is any untoward medical occurence in a patient administered a medicinal product that does not necessarily have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign ( for example, an abnormal laboratory finding), symptom or disease temporally associated with the use of the medicinal product, whether or not this is associated with the use of this medicinal product. |
| Primary | Change in Liver Iron Concentration (LIC) | Baseline of Core Study to End of Extension Study, up to 5 years. | Change in Liver Iron Concentration \[LIC\] measured by means of SQUID (Superconducting Quantum Interference Device). LIC is expressed in milligrams of iron per gram of liver dry weight (mg Fe/g dw) |
| Secondary | Total Body Iron Elimination (TBIE) Rate (mg/kg/Day) | Baseline of Core Study to End of Extension Study, up to 5 years | Total Iron Body Elimination (TBIE) Rate \[mg/kg/Day\] was calculated for each patient based on SQUID ( Superconducting Quantum Interference Device) results. |
| Secondary | Relative Change in Serum Ferritin Level | Baseline of Core Study to Extension 18 months, up to 2.5 years. | Serum levels were drawn at the baseline of the Core Study up to 18 months of the Extension Study. Levels were analyzed for serum ferritin measured in micrograms per Liter. Relative change (%) in serum ferritin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in ferritin level from Baseline/Baseline level) x 100. |
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 | Participants With Adverse Events by Primary System Organ Class (SOC) | 4 year extension + core 1 year | The safety set comprising of all the 40 patients who received at least one dose of deferasirox during the core or extension study was used in all analyses. | participants | NUMBER |
| PRIMARY | Change in Liver Iron Concentration (LIC) | Baseline of Core Study to End of Extension Study, up to 5 years. | The safety set was used for all analyses. This comprised of all 40 patients who received at least one dose of deferasirox during the core or extension study. | mg Fe/g dw | MEAN |
| SECONDARY | Total Body Iron Elimination (TBIE) Rate (mg/kg/Day) | Baseline of Core Study to End of Extension Study, up to 5 years | The safety set was used for all analyses. This comprised of all 40 patients who received at least one dose of deferasirox during the core or extension study. | mg/kg/Day | MEAN |
| SECONDARY | Relative Change in Serum Ferritin Level | Baseline of Core Study to Extension 18 months, up to 2.5 years. | The safety set was used for all analyses. This comprised of all 40 patients who received at least one dose of deferasirox during the core or extension study. | percent change | MEAN |
Source ClinicalTrials.gov
Results by group60
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Participants With Adverse Events by Primary System Organ Class (SOC) | Children (<12 Years) | Patients with at least one Adverse Event (AE) | 20 | participants | |
| Participants With Adverse Events by Primary System Organ Class (SOC) | Adolescents ( ≧12 Years) | Patients with at least one Adverse Event (AE) | 20 | participants | |
| Participants With Adverse Events by Primary System Organ Class (SOC) | Children (<12 Years) | General disorders & administration site conditions | 19 | participants | |
| Participants With Adverse Events by Primary System Organ Class (SOC) | Adolescents ( ≧12 Years) | General disorders & administration site conditions | 20 | participants | |
| Participants With Adverse Events by Primary System Organ Class (SOC) | Children (<12 Years) | Respiratory, thoracic & mediastinal disorders | 19 | participants | |
| Participants With Adverse Events by Primary System Organ Class (SOC) | Adolescents ( ≧12 Years) | Respiratory, thoracic & mediastinal disorders | 17 | participants | |
| Participants With Adverse Events by Primary System Organ Class (SOC) | Children (<12 Years) | Gastrointestinal disorders | 18 | participants | |
| Participants With Adverse Events by Primary System Organ Class (SOC) | Adolescents ( ≧12 Years) | Gastrointestinal disorders | 17 | participants | |
| Participants With Adverse Events by Primary System Organ Class (SOC) | Children (<12 Years) | Infections & infestations | 18 | participants | |
| Participants With Adverse Events by Primary System Organ Class (SOC) | Adolescents ( ≧12 Years) | Infections & infestations | 17 | participants |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Children ( < 12 Years) | 20 | |
| Overall Study | STARTED | Adolescents ( ≧12 Years) | 20 | |
| Overall Study | Completed Core & Entered Extension Study | Children ( < 12 Years) | 19 | |
| Overall Study | Completed Core & Entered Extension Study | Adolescents ( ≧12 Years) | 20 | |
| Overall Study | COMPLETED | Children ( < 12 Years) | 11 | |
| Overall Study | COMPLETED | Adolescents ( ≧12 Years) | 13 | |
| Overall Study | NOT COMPLETED | Children ( < 12 Years) | 9 | |
| Overall Study | NOT COMPLETED | Adolescents ( ≧12 Years) | 7 |
Source ClinicalTrials.gov
Adverse events430
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 | Children (<12 Years) | 0 | 20 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Adolescents ( ≧12 Years) | 4 | 20 | |
| other | Splenomegaly | Blood and lymphatic system disorders | Children (<12 Years) | 0 | 20 | |
| other | Splenomegaly | Blood and lymphatic system disorders | Adolescents ( ≧12 Years) | 1 | 20 | |
| other | Cardiomyopathy | Cardiac disorders | Children (<12 Years) | 0 | 20 | |
| other | Cardiomyopathy | Cardiac disorders | Adolescents ( ≧12 Years) | 1 | 20 | |
| other | Extrasystoles | Cardiac disorders | Children (<12 Years) | 0 | 20 | |
| other | Extrasystoles | Cardiac disorders | Adolescents ( ≧12 Years) | 1 | 20 | |
| other | Palpitations | Cardiac disorders | Children (<12 Years) | 0 | 20 | |
| other | Palpitations | Cardiac disorders | Adolescents ( ≧12 Years) | 2 | 20 |
1 to 10 of 430Source ClinicalTrials.gov
See all 430 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Children (<12 Years) | ||
| Total, all-cause mortality | Adolescents ( ≧12 Years) | ||
| Total, other adverse events | Children (<12 Years) | 20 | 20 |
| Total, other adverse events | Adolescents ( ≧12 Years) | 20 | 20 |
| Total, serious adverse events | Children (<12 Years) | 4 | 20 |
| Total, serious adverse events | Adolescents ( ≧12 Years) | 8 | 20 |
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 |
|---|
| Children (<12 Years) vs Adolescents ( ≧12 Years): 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/NCT00390858
GET https://conductscience.org/api/trials/v1/NCT00390858/results?page=2