Trial · NCT02435212
Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload
NCT02435212, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2024-09-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload. Trials, record NCT02435212. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02435212
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload
- NCT number
- NCT02435212
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 224 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Transfusion-dependent Anemia
- Interventions
- Deferasirox granule formulation, Deferasirox DT formulation
- First posted
- 2015-05-06
- Last updated
- 2024-09-19
- Other registries' IDs
- eudract:2013-004739-55
- Results first posted
- 2024-09-19
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Overall Compliance Using Stick Pack or Tablet Counts in Iron Chelation Therapy (ICT)-naïve Participants During the Core Phase | 24 weeks | Compliance was calculated as the ratio of total count consumed to total count prescribed of deferasirox granule stick packs or dispersible tablets, where total count consumed was derived from cumulative dispensed, returned and lost/wasted counts over 24 weeks of treatment and total count prescribed was derived from cumulative prescribed count over 24 weeks of treatment. |
| Primary | Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | From Baseline to Week 25 | The analysis included the comparison of means between the two treatment arms of change from baseline after 24 weeks of treatment in serum ferritin in pediatric ICT naïve participants with iron overload. The endpoint was assessed at Week 25 visit. |
| Secondary | Percentage of Overall Compliance Using Stick Pack or Tablet Counts in ICT-naïve Participants During the Core Phase | 48 weeks | Compliance was calculated as the ratio of total count consumed to total count prescribed of deferasirox granule stick packs or dispersible tablets over 48 weeks of treatment. |
| Secondary | Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | From Baseline to 48 weeks | The analysis included the comparison of means between the two treatment arms of change from baseline after 48 weeks of treatment in serum ferritin in pediatric ICT naïve participants with iron overload. |
| Secondary | Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in Pre-treated Participants During the Core Phase | From Baseline to Week 25 and Week 48 | The analysis included the comparison of means between the two treatment arms of change from baseline after 25 weeks and after 48 weeks of treatment in serum ferritin in pre-treated participants. The analyses were performed at Week 25 and Week 48. |
| Secondary | Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Patient Reported Outcomes (PRO) Questionnaires | At Week 2, Week 3, Week 25 and Week 48 | Participants aged between 10 years and less than 18 years at enrollment completed PRO questionnaires by themselves. The mSICT questionnaire for PRO consisted of 3 domains: adherence, satisfaction/preference, and concerns. The adherence domain had a minimum score of 6 and maximum score of 30; a lower score for adherence indicates better adherence. Satisfaction/preference domain had a minimum score of 2 and maximum score of 10; a lower score for satisfaction/preference indicates better satisfaction/preference. Concerns domain had a minimum score of 3 and maximum score of 15; a higher score for concerns indicate fewer concerns. |
| Secondary | Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Caregiver's Perspective) | At Week 2, Week 3, Week 25 and Week 48 | The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses. The mSICT questionnaire consisted of 2 domains: adherence and concerns per caregiver's perspective. The adherence domain had a minimum score of 5 and a maximum score of 25; a lower score for adherence indicates better adherence. The concerns domain had a minimum score of 1 and a maximum score of 5; a higher score for concerns indicates fewer concerns. |
| Secondary | Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Child's Perspective) | At Week 2, Week 3, Week 25 and Week 48 | The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses. The mSICT questionnaire is presented for 2 domains: adherence and concerns per child's perspective. The adherence domain had a minimum score of 6 and a maximum score of 30; a lower score for adherence indicates better adherence. The concerns domain had a minimum score of 2 and a maximum score of 10; a higher score for concerns indicates fewer concerns. |
| Secondary | Change Over-time in Domain Score of Palatability Using Patient Reported Outcomes (PRO) Questionnaires | At Week 2, Week 3, Week 25 and Week 48 | The palatability questionnaire was used to measure: taste, aftertaste, whether medication was taken and how the participant perceived the amount of medication taken. This questionnaire had a minimum score of 0 and maximum score of 11; a higher score means better palatability. Participants aged between 10 years and less than 18 years at enrollment completed the PRO questionnaire by themselves. |
| Secondary | Change Over-time in Domain Score of Palatability Using Observer Reported Outcomes (ObsRO) Questionnaire | At Week 2, Week 3, Week 25 and Week 48 | The palatability questionnaire was used to measure: taste, aftertaste, whether medication was taken and how the participant perceived the amount of medication taken. This questionnaire had a minimum score of 0 and maximum score of 11; a higher score means better palatability. The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Overall Compliance Using Stick Pack or Tablet Counts in Iron Chelation Therapy (ICT)-naïve Participants During the Core Phase | 24 weeks | The Full Analysis Set 1 (FAS-1) consisted of all ICT naive randomized participants during the core phase. | percentage of compliance | MEAN |
| PRIMARY | Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | From Baseline to Week 25 | The Full Analysis Set 1 (FAS-1) consisted of all ICT naive randomized participants during the core phase. | μg/L | MEAN |
| SECONDARY | Percentage of Overall Compliance Using Stick Pack or Tablet Counts in ICT-naïve Participants During the Core Phase | 48 weeks | The Full Analysis Set 1 (FAS-1) consisted of all ICT naive randomized participants during the core phase. | percentage of compliance | MEAN |
| SECONDARY | Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | From Baseline to 48 weeks | The Full Analysis Set 1 (FAS-1) consisted of all ICT naive randomized participants during the core phase. | μg/L | MEAN |
| SECONDARY | Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in Pre-treated Participants During the Core Phase | From Baseline to Week 25 and Week 48 | The Full Analysis Set 2 (FAS-2) consisted of all ICT pre-treated randomized participants during the core phase. | μg/L | MEAN |
| SECONDARY | Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Patient Reported Outcomes (PRO) Questionnaires | At Week 2, Week 3, Week 25 and Week 48 | The Full Analysis Set 3 (FAS-3) consisted of all randomized participants during the core phase. This includes only the participants aged between 10 years and less than 18 years with a valid assessment for this outcome measure. | score on a scale | MEAN |
| SECONDARY | Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Caregiver's Perspective) | At Week 2, Week 3, Week 25 and Week 48 | The Full Analysis Set 3 (FAS-3) consisted of all randomized participants during the core phase. This includes only the participants aged between 2 years and less than 10 years with a valid assessment for this outcome measure. | score on a scale | MEAN |
| SECONDARY | Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Child's Perspective) | At Week 2, Week 3, Week 25 and Week 48 | The Full Analysis Set 3 (FAS-3) consisted of all randomized participants during the core phase. This includes only the participants aged between 2 years and less than 10 years with a valid assessment for this outcome measure. | score on a scale | MEAN |
| SECONDARY | Change Over-time in Domain Score of Palatability Using Patient Reported Outcomes (PRO) Questionnaires | At Week 2, Week 3, Week 25 and Week 48 | The Full Analysis Set 3 (FAS-3) consisted of all randomized participants during the core phase. This includes only the participants aged between 10 years and less than 18 years with a valid assessment for this outcome measure. | score on a scale | MEAN |
| SECONDARY | Change Over-time in Domain Score of Palatability Using Observer Reported Outcomes (ObsRO) Questionnaire | At Week 2, Week 3, Week 25 and Week 48 | The Full Analysis Set 3 (FAS-3) consisted of all randomized participants during the core phase. This includes only the participants aged between 2 years and less than 10 years with a valid assessment for this outcome measure. | score on a scale | MEAN |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group174
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Overall Compliance Using Stick Pack or Tablet Counts in Iron Chelation Therapy (ICT)-naïve Participants During the Core Phase | DFX DT | 89.45 | 95% Confidence Interval 85.29 to 93.61 | percentage of compliance | |
| Percentage of Overall Compliance Using Stick Pack or Tablet Counts in Iron Chelation Therapy (ICT)-naïve Participants During the Core Phase | DFX Granule | 91.78 | 95% Confidence Interval 87.81 to 95.75 | percentage of compliance | |
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | DFX DT | Baseline | 2063.7 | 95% Confidence Interval 1807.17 to 2320.15 | μg/L |
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | DFX Granule | Baseline | 1955.5 | 95% Confidence Interval 1712.57 to 2198.49 | μg/L |
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | DFX DT | Week 25 | 2216.3 | 95% Confidence Interval 1929.59 to 2502.93 | μg/L |
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | DFX Granule | Week 25 | 2228.4 | 95% Confidence Interval 2011.69 to 2445.18 | μg/L |
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | DFX DT | Change from Baseline to Week 25 | 250.5 | 95% Confidence Interval -84.63 to 585.58 | μg/L |
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | DFX Granule | Change from Baseline to Week 25 | 340.0 | 95% Confidence Interval 115.48 to 564.59 | μg/L |
| Percentage of Overall Compliance Using Stick Pack or Tablet Counts in ICT-naïve Participants During the Core Phase | DFX DT | 91.57 | 95% Confidence Interval 87.65 to 95.49 | percentage of compliance | |
| Percentage of Overall Compliance Using Stick Pack or Tablet Counts in ICT-naïve Participants During the Core Phase | DFX Granule | 94.80 | 95% Confidence Interval 91.48 to 98.13 | percentage of compliance |
1 to 10 of 174Source ClinicalTrials.gov
See all 174 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Core Phase | STARTED | DFX DT | 112 | |
| Core Phase | STARTED | DFX Granule | 112 | |
| Core Phase | COMPLETED | DFX DT | 87 | |
| Core Phase | COMPLETED | DFX Granule | 99 | |
| Core Phase | NOT COMPLETED | DFX DT | 25 | |
| Core Phase | NOT COMPLETED | DFX Granule | 13 | |
| Optional Extension Phase | STARTED | DFX DT | 69 | |
| Optional Extension Phase | STARTED | DFX Granule | 77 | |
| Optional Extension Phase | COMPLETED | DFX DT | 42 | |
| Optional Extension Phase | COMPLETED | DFX Granule | 46 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events516
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 | Abdominal pain | Gastrointestinal disorders | DFX DT- Core Phase | 4 | 111 | |
| other | Abdominal pain | Gastrointestinal disorders | DFX DT Cross-over Granule - Optional Extension Phase | 4 | 69 | |
| other | Abdominal pain | Gastrointestinal disorders | DFX Granules - Core Phase | 12 | 110 | |
| other | Abdominal pain | Gastrointestinal disorders | DFX Granules - Optional Extension Phase | 0 | 77 | |
| other | Abdominal pain upper | Gastrointestinal disorders | DFX DT- Core Phase | 4 | 111 | |
| other | Abdominal pain upper | Gastrointestinal disorders | DFX DT Cross-over Granule - Optional Extension Phase | 3 | 69 | |
| other | Abdominal pain upper | Gastrointestinal disorders | DFX Granules - Core Phase | 4 | 110 | |
| other | Abdominal pain upper | Gastrointestinal disorders | DFX Granules - Optional Extension Phase | 4 | 77 | |
| other | Constipation | Gastrointestinal disorders | DFX DT- Core Phase | 5 | 111 | |
| other | Constipation | Gastrointestinal disorders | DFX DT Cross-over Granule - Optional Extension Phase | 4 | 69 |
1 to 10 of 516Source ClinicalTrials.gov
See all 516 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DFX DT- Core Phase | 0 | 111 |
| Total, all-cause mortality | DFX DT Cross-over Granule - Optional Extension Phase | 1 | 69 |
| Total, all-cause mortality | DFX Granules - Core Phase | 0 | 110 |
| Total, all-cause mortality | DFX Granules - Optional Extension Phase | 0 | 77 |
| Total, other adverse events | DFX DT- Core Phase | 104 | 111 |
| Total, other adverse events | DFX DT Cross-over Granule - Optional Extension Phase | 63 | 69 |
| Total, other adverse events | DFX Granules - Core Phase | 97 | 110 |
| Total, other adverse events | DFX Granules - Optional Extension Phase | 63 | 77 |
| Total, serious adverse events | DFX DT- Core Phase | 23 | 111 |
| Total, serious adverse events | DFX DT Cross-over Granule - Optional Extension Phase | 22 | 69 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
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/NCT02435212
GET https://conductscience.org/api/trials/v1/NCT02435212/results?page=2