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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-004739-55 | EudraCT Number |
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This was a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox dispersible tablet (DT) formulation in children and adolescents aged ≥ 2 and < 18 years at enrolment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, to demonstrate the effect of improved compliance on iron burden.
Randomization was stratified by age groups (2 to <10 years, 10 to <18 years) and prior iron chelation therapy (Yes/ No). There were two study phases which include a 1 year core phase where participants were randomized to a 48 week treatment period to either Deferasirox DT or granules, and an optional extension phase where all participants received the granules up to 5 years. Participants who demonstrated benefit to granules or DT in the core phase, and/or expressed the wish to continue in the optional extension phase on granules, were offered this possibility until there was local access to the new formulation (granules or film-coated tablet (FCT)) or up to 5 years, whichever occurred first.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DFX DT | Active Comparator | Participants will be administered deferasirox dispersible tablets orally once daily based on body weight for 48 weeks. |
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| DFX Granule | Experimental | Participants will be administered deferasirox granules orally once daily in the form of stick packs based on body weight for 48 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deferasirox granule formulation | Drug | Deferasirox granules will be provided as stick packs containing 90 mg, 180 mg and 360 mg granules for oral use and will be administered based on body weight. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Overall Compliance Using Stick Pack or Tablet Counts in Iron Chelation Therapy (ICT)-naïve Participants During the Core Phase | 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. | 24 weeks |
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | 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. | From Baseline to Week 25 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Overall Compliance Using Stick Pack or Tablet Counts in ICT-naïve Participants During the Core Phase | 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. | 48 weeks |
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase |
| Measure | Description | Time Frame |
|---|---|---|
| Exposure-Response Relationship in Relation to Pre- and Post-Dose Deferasirox Concentrations (PK/PD Relationship) | This outcome measure explores exposure-response relationships for measures of safety and effectiveness through serum creatinine change from baseline, notable serum creatinine values, serum creatinine clearance change form baseline and notable serum creatinine clearance categories, serum ferritin change from baseline, in relationship to derived PK parameters for pre- and post-dose deferasirox concentrations. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Oakland | California | 94609-1809 | United States | ||
| Novartis Investigative Site |
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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After enrollment, participants previously treated with iron chelation therapy (ICT) underwent a 5-day chelation washout period prior to the commencement of the 48-week treatment (Core phase).
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| ID | Title | Description |
|---|---|---|
| FG000 | DFX DT | Participants received deferasirox (DFX) dispersible tablets (DT) orally once daily based on body weight for 48 weeks in the Core phase. The starting dose was 20mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and cross over to DFX granules administered orally once daily in the form of stick packs for up to 5 years. Participants entering the Optional Extension phase received the equivalent strength-adjusted DFX granules dose corresponding to the last DT dose in the Core phase taking dose adjustment guidelines into account. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Core Phase |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 24, 2021 | Jul 9, 2024 |
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| Deferasirox DT formulation | Drug | Deferasirox DT will be provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use and will be administered based on body weight. |
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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. |
| From Baseline to 48 weeks |
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in Pre-treated Participants During the Core Phase | 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. | From Baseline to Week 25 and Week 48 |
| Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Patient Reported Outcomes (PRO) Questionnaires | 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. | At Week 2, Week 3, Week 25 and Week 48 |
| Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Caregiver's Perspective) | 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. | At Week 2, Week 3, Week 25 and Week 48 |
| Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Child's Perspective) | 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. | At Week 2, Week 3, Week 25 and Week 48 |
| Change Over-time in Domain Score of Palatability Using Patient Reported Outcomes (PRO) Questionnaires | 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. | At Week 2, Week 3, Week 25 and Week 48 |
| Change Over-time in Domain Score of Palatability Using Observer Reported Outcomes (ObsRO) Questionnaire | 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. | At Week 2, Week 3, Week 25 and Week 48 |
| Change Over Time in Weekly Dose Violation Rate Using Compliance Patient Reported Outcomes (PRO) Questionnaire | The compliance questionnaire consisted of 2 items: 1. To assess if the medication was taken (yes/no) and 2. To record of the time when the medication was taken (with a not applicable option for participants who did not take their medication). Daily diary records were used to calculate the rate of dose violation in each study arm (doses missed completely or not taken before 12 PM). The dose violation rate was calculated as: [Number of dose violations / Drug exposure (days)] *100. Higher values represent more dose violations. | At Week 1, Week 13, Week 25, Week 37 and Week 48 |
| Change Over Time in Weekly Dose Violation Rate Using Compliance Observer Reported Outcomes (ObsRO) Questionnaire | The compliance questionnaire consisted of 2 items: 1. To assess if the medication was taken (yes/no) and 2. To record the time when the medication was taken (with a not applicable option for participants who did not take their medication). Daily diary records were used to calculate the rate of dose violation in each treatment arm (doses missed completely or not taken before 12 PM). 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 dose violation rate was calculated as: [Number of dose violations / Drug exposure (days)] *100. Higher values represent more dose violations. | At Week 1, Week 13, Week 25, Week 37 and Week 48 |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) During the Core Phase | An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. | From Baseline to 48 weeks |
| Pre-dose Concentrations of Deferasirox to Support the Assessment of Compliance | Pre-dose pharmacokinetic (PK) data from participants in the Pharmacokinetic Analysis Set 1 (PAS-1) were analyzed to assess variability of individual participant's compliance. A linear mixed effect power model to pre-dose samples which fulfill compliance criteria in terms of steady state (4 consecutive same doses prior to the PK sample drawn), time-windows (PK sample drawn 20 to 28 hours after previous dose) and without any vomiting episodes within the 4 hours prior to the PK sample were fitted. The model considered dose, treatment group, stratification factors and potential other factors, such as body weight as covariates. | At Weeks 1, 3, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, and 45 |
| Concentrations of Deferasirox Between 2 and 4 Hours Post-dose at Weeks 5 and 9 | Post-dose pharmacokinetic (PK) data from participants in the Pharmacokinetic Analysis Set 1 (PAS-1) were analyzed along with Pre-dose PK data. | At Week 5 and Week 9 |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) During the Entire Granule Period | An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation after participant providing written informed consent for participation in the study. In the DFX Granules arm, AEs are reported since the initial randomization to the arm in the core phase and continuing in the extension phase. In the DFX cross-over arm, AEs are reported for participants since the participant crossed-over from dispersible tablet to granules in the extension phase only. | From Baseline to 305 weeks |
| Number of Participants With Adverse Events of Special Interest (AESI) During the Entire Granule Period | An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation after participant providing written informed consent for participation in the study. In the DFX Granules arm, AEs are reported since the initial randomization to the arm in the core phase and continuing in the extension phase. In the DFX cross-over arm, AEs are reported for participants since the participant crossed-over from dispersible tablet to granules in the extension phase only. AESI included active monitoring for renal toxicity; including renal failure, hepatic toxicity; including hepatic failure, and gastrointestinal hemorrhage | From Baseline to 305 weeks |
| From Baseline to 48 weeks |
| Atlanta |
| Georgia |
| 30342 |
| United States |
| Novartis Investigative Site | Chicago | Illinois | 60611 | United States |
| Novartis Investigative Site | New York | New York | 10021 | United States |
| Weill Cornell Medical College SC - | New York | New York | 10021 | United States |
| Childrens Hospital at Montefiore | The Bronx | New York | 10467 | United States |
| Novartis Investigative Site | The Bronx | New York | 10467 | United States |
| Childrens Hospital of Philadelphia Onc. Dept | Philadelphia | Pennsylvania | 19104 4399 | United States |
| Novartis Investigative Site | Philadelphia | Pennsylvania | 19104 4399 | United States |
| Medical Uni of South Carolina Medical Uni of South Carolina | Charleston | South Carolina | 29425 | United States |
| Novartis Investigative Site | Charleston | South Carolina | 29425 | United States |
| Novartis Investigative Site | Memphis | Tennessee | 38105 | United States |
| St. Jude Children's Research Hospital Memphis St Jude | Memphis | Tennessee | 38105 | United States |
| Novartis Investigative Site | Edegem | Antwerpen | 2650 | Belgium |
| Novartis Investigative Site | Brussels | 1200 | Belgium |
| Novartis Investigative Site | Plovdiv | 4002 | Bulgaria |
| Novartis Investigative Site | Sofia | 1527 | Bulgaria |
| Novartis Investigative Site | Varna | 9010 | Bulgaria |
| Novartis Investigative Site | Alexandria | 21131 | Egypt |
| Novartis Investigative Site | Créteil | 94000 | France |
| Novartis Investigative Site | Paris | 75015 | France |
| Novartis Investigative Site | Debrecen | 4032 | Hungary |
| Novartis Investigative Site | Kolkata | West Bengal | 700017 | India |
| Novartis Investigative Site | Genova | GE | 16128 | Italy |
| Novartis Investigative Site | Palermo | PA | 90127 | Italy |
| Novartis Investigative Site | Palermo | PA | 90146 | Italy |
| Novartis Investigative Site | Naples | 80132 | Italy |
| Novartis Investigative Site | Hazmiyeh | Beirut | PO BOX 213 | Lebanon |
| Novartis Investigative Site | Ipoh | Perak | 30450 | Malaysia |
| Novartis Investigative Site | Kuching | Sarawak | 93586 | Malaysia |
| Novartis Investigative Site | Kuala Lumpur | 50589 | Malaysia |
| Novartis Investigative Site | Pulau Pinang | 10990 | Malaysia |
| Novartis Investigative Site | Muscat | 123 | Oman |
| Novartis Investigative Site | Panama City | Republica de Panama | 0801 | Panama |
| Novartis Investigative Site | Quezon | 1100 | Philippines |
| Novartis Investigative Site | Quezon City | 1100 | Philippines |
| Novartis Investigative Site | Moscow | 117198 | Russia |
| Novartis Investigative Site | Bangkok Noi | Bangkok | 10700 | Thailand |
| Novartis Investigative Site | Muang | Chiangmai | 50200 | Thailand |
| Novartis Investigative Site | Tunis | 1006 | Tunisia |
| Novartis Investigative Site | Adana | 01330 | Turkey (Türkiye) |
| Novartis Investigative Site | Ankara | 06100 | Turkey (Türkiye) |
| Novartis Investigative Site | Izmir | 35040 | Turkey (Türkiye) |
| FG001 | DFX Granule | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
| COMPLETED |
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| NOT COMPLETED |
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| Optional Extension Phase |
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Full Analysis Set (FAS) was defined according to the Intent to Treat (ITT) principle, and included all randomized participants.
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| ID | Title | Description |
|---|---|---|
| BG000 | DFX DT | Participants received deferasirox (DFX) dispersible tablets (DT) orally once daily based on body weight for 48 weeks in the Core phase. The starting dose was 20mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and cross over to DFX granules administered orally once daily in the form of stick packs for up to 5 years. Participants entering the Optional Extension phase received the equivalent strength-adjusted DFX granules dose corresponding to the last DT dose in the Core phase taking dose adjustment guidelines into account. |
| BG001 | DFX Granule | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex/Gender, Customized | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Percentage of Overall Compliance Using Stick Pack or Tablet Counts in Iron Chelation Therapy (ICT)-naïve Participants During the Core Phase | 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. | The Full Analysis Set 1 (FAS-1) consisted of all ICT naive randomized participants during the core phase. | Posted | Mean | 95% Confidence Interval | percentage of compliance | 24 weeks |
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| Primary | Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | 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. | The Full Analysis Set 1 (FAS-1) consisted of all ICT naive randomized participants during the core phase. | Posted | Mean | 95% Confidence Interval | μg/L | From Baseline to Week 25 |
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| Secondary | Percentage of Overall Compliance Using Stick Pack or Tablet Counts in ICT-naïve Participants During the Core Phase | 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. | The Full Analysis Set 1 (FAS-1) consisted of all ICT naive randomized participants during the core phase. | Posted | Mean | 95% Confidence Interval | percentage of compliance | 48 weeks |
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| Secondary | Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | 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. | The Full Analysis Set 1 (FAS-1) consisted of all ICT naive randomized participants during the core phase. | Posted | Mean | 95% Confidence Interval | μg/L | From Baseline to 48 weeks |
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| Secondary | Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in Pre-treated Participants During the Core Phase | 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. | The Full Analysis Set 2 (FAS-2) consisted of all ICT pre-treated randomized participants during the core phase. | Posted | Mean | 95% Confidence Interval | μg/L | From Baseline to Week 25 and Week 48 |
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| Secondary | Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Patient Reported Outcomes (PRO) Questionnaires | 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. | 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. | Posted | Mean | Standard Deviation | score on a scale | At Week 2, Week 3, Week 25 and Week 48 |
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| Secondary | Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Caregiver's Perspective) | 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. | 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. | Posted | Mean | Standard Deviation | score on a scale | At Week 2, Week 3, Week 25 and Week 48 |
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| Secondary | Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Child's Perspective) | 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. | 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. | Posted | Mean | Standard Deviation | score on a scale | At Week 2, Week 3, Week 25 and Week 48 |
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| Secondary | Change Over-time in Domain Score of Palatability Using Patient Reported Outcomes (PRO) Questionnaires | 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. | 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. | Posted | Mean | Standard Deviation | score on a scale | At Week 2, Week 3, Week 25 and Week 48 |
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| Secondary | Change Over-time in Domain Score of Palatability Using Observer Reported Outcomes (ObsRO) Questionnaire | 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. | 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. | Posted | Mean | Standard Deviation | score on a scale | At Week 2, Week 3, Week 25 and Week 48 |
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| Secondary | Change Over Time in Weekly Dose Violation Rate Using Compliance Patient Reported Outcomes (PRO) Questionnaire | The compliance questionnaire consisted of 2 items: 1. To assess if the medication was taken (yes/no) and 2. To record of the time when the medication was taken (with a not applicable option for participants who did not take their medication). Daily diary records were used to calculate the rate of dose violation in each study arm (doses missed completely or not taken before 12 PM). The dose violation rate was calculated as: [Number of dose violations / Drug exposure (days)] *100. Higher values represent more dose violations. | 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. | Posted | Mean | Standard Deviation | percentage of days with dose violations | At Week 1, Week 13, Week 25, Week 37 and Week 48 |
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| Secondary | Change Over Time in Weekly Dose Violation Rate Using Compliance Observer Reported Outcomes (ObsRO) Questionnaire | The compliance questionnaire consisted of 2 items: 1. To assess if the medication was taken (yes/no) and 2. To record the time when the medication was taken (with a not applicable option for participants who did not take their medication). Daily diary records were used to calculate the rate of dose violation in each treatment arm (doses missed completely or not taken before 12 PM). 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 dose violation rate was calculated as: [Number of dose violations / Drug exposure (days)] *100. Higher values represent more dose violations. | 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. | Posted | Mean | Standard Deviation | percentage of days with dose violations | At Week 1, Week 13, Week 25, Week 37 and Week 48 |
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| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) During the Core Phase | An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. | The Safety Set consisted of all participants who received at least 1 dose of study drug during the core phase. Three participants did not receive the study drug and hence were excluded from the safety set. | Posted | Count of Participants | Participants | From Baseline to 48 weeks |
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| Secondary | Pre-dose Concentrations of Deferasirox to Support the Assessment of Compliance | Pre-dose pharmacokinetic (PK) data from participants in the Pharmacokinetic Analysis Set 1 (PAS-1) were analyzed to assess variability of individual participant's compliance. A linear mixed effect power model to pre-dose samples which fulfill compliance criteria in terms of steady state (4 consecutive same doses prior to the PK sample drawn), time-windows (PK sample drawn 20 to 28 hours after previous dose) and without any vomiting episodes within the 4 hours prior to the PK sample were fitted. The model considered dose, treatment group, stratification factors and potential other factors, such as body weight as covariates. | Pharmacokinetic Analysis Set 1 (PAS-1) consisted of all participants who had at least one evaluable pre- or 3 hours post-dose PK concentration of deferasirox. The analysis included participants only with evaluable values. | Posted | Geometric Mean | Geometric Coefficient of Variation | μmol/L | At Weeks 1, 3, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, and 45 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Concentrations of Deferasirox Between 2 and 4 Hours Post-dose at Weeks 5 and 9 | Post-dose pharmacokinetic (PK) data from participants in the Pharmacokinetic Analysis Set 1 (PAS-1) were analyzed along with Pre-dose PK data. | Pharmacokinetic Analysis Set 1 (PAS-1) consisted of all participants who had at least one evaluable pre- or 3 hours post-dose PK concentration of deferasirox. The analysis included participants only with evaluable values. | Posted | Geometric Mean | Geometric Coefficient of Variation | μmol/L | At Week 5 and Week 9 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) During the Entire Granule Period | An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation after participant providing written informed consent for participation in the study. In the DFX Granules arm, AEs are reported since the initial randomization to the arm in the core phase and continuing in the extension phase. In the DFX cross-over arm, AEs are reported for participants since the participant crossed-over from dispersible tablet to granules in the extension phase only. | The Safety Set consisted of all participants who received at least 1 dose of granule formulation during the core or extension phase. The participants in the extension phase received granules regardless of which arm they were initially randomized. | Posted | Count of Participants | Participants | From Baseline to 305 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Adverse Events of Special Interest (AESI) During the Entire Granule Period | An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation after participant providing written informed consent for participation in the study. In the DFX Granules arm, AEs are reported since the initial randomization to the arm in the core phase and continuing in the extension phase. In the DFX cross-over arm, AEs are reported for participants since the participant crossed-over from dispersible tablet to granules in the extension phase only. AESI included active monitoring for renal toxicity; including renal failure, hepatic toxicity; including hepatic failure, and gastrointestinal hemorrhage | The Safety Set consisted of all participants who received at least 1 dose of granule formulation during the core or extension phase. The participants in the extension phase received granules regardless of which arm they were initially randomized. | Posted | Count of Participants | Participants | From Baseline to 305 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Exposure-Response Relationship in Relation to Pre- and Post-Dose Deferasirox Concentrations (PK/PD Relationship) | This outcome measure explores exposure-response relationships for measures of safety and effectiveness through serum creatinine change from baseline, notable serum creatinine values, serum creatinine clearance change form baseline and notable serum creatinine clearance categories, serum ferritin change from baseline, in relationship to derived PK parameters for pre- and post-dose deferasirox concentrations. | Not Posted | From Baseline to 48 weeks | Participants |
From initial randomization up to 48 weeks (Core phase) and Up to 5 years from entering the extension phase (Optional Extension phase)
The Safety Set consisted of all participants who received at least 1 dose of granule formulation during the core or extension phase. The participants in extension phase received granules regardless of which arm they were initially randomized. Three participants did not receive the study drug and hence were excluded from the safety set.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | DFX DT- Core Phase | Participants received deferasirox (DFX) dispersible tablets (DT) orally once daily based on body weight for 48 weeks in the Core phase. The starting dose was 20mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. | 0 | 111 | 23 | 111 | 104 | 111 |
| EG001 | DFX DT Cross-over Granule - Optional Extension Phase | After the Core phase, participants could enter the Optional Extension phase and cross over to DFX granules administered orally once daily in the form of stick packs for up to 5 years. Participants entering the Optional Extension phase received the equivalent strength-adjusted DFX granules dose corresponding to the last DT dose in the Core phase taking dose adjustment guidelines into account. | 1 | 69 | 22 | 69 | 63 | 69 |
| EG002 | DFX Granules - Core Phase | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. | 0 | 110 | 27 | 110 | 97 | 110 |
| EG003 | DFX Granules - Optional Extension Phase | After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. | 0 | 77 | 20 | 77 | 63 | 77 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypersplenism | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Lymphadenitis | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Food poisoning | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Malpositioned teeth | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Stress ulcer | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Sudden death | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cholangitis acute | Hepatobiliary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Drug-induced liver injury | Hepatobiliary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hepatitis acute | Hepatobiliary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Jaundice cholestatic | Hepatobiliary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Amoebiasis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Ascariasis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Atypical pneumonia | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Bacterial infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Bronchiolitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Bronchitis viral | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Dengue fever | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Dengue haemorrhagic fever | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Gastroenteritis rotavirus | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Gastroenteritis salmonella | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Parotid abscess | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Periorbital cellulitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Pharyngotonsillitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Pneumonia respiratory syncytial viral | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Scarlet fever | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Shigella infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Sinobronchitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Subcutaneous abscess | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Systemic viral infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Craniofacial fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Febrile nonhaemolytic transfusion reaction | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Lip injury | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Overdose | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Urine protein/creatinine ratio increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Diabetic ketoacidosis | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Electrolyte imbalance | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Lactase deficiency | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Tension headache | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Fanconi syndrome acquired | Renal and urinary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Bronchial hyperreactivity | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Obstructive sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Drug reaction with eosinophilia and systemic symptoms | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Circulatory collapse | Vascular disorders | MedDRA (26.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Systemic viral infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Bilirubin conjugated increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Urine protein/creatinine ratio increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | +1 862 778 8300 | novartis.email@novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 21, 2024 | Jul 9, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| D019190 | Iron Overload |
| D010349 | Patient Compliance |
| D010549 | Personal Satisfaction |
| ID | Term |
|---|---|
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D010342 | Patient Acceptance of Health Care |
| D000074822 | Treatment Adherence and Compliance |
| D015438 | Health Behavior |
| D001519 | Behavior |
Not provided
Not provided
| ID | Term |
|---|---|
| D000077588 | Deferasirox |
| ID | Term |
|---|---|
| D001565 | Benzoates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D014230 | Triazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
Not provided
Not provided
| Withdrawal by parent/guardian |
|
| Lack of Efficacy |
|
| Withdrawal by Subject |
|
| Protocol Violation |
|
| Recovery |
|
| Death |
|
| Technical problems |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
|
|
|
|
|
|
| OG001 | DFX Granule | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|
| OG001 | DFX Granule | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|
| OG001 | DFX Granule | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|
Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|
| OG001 | DFX Granule | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|
| OG001 | DFX Granule | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|
| OG001 | DFX Granule | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|
| Units | Counts |
|---|
| Participants |
|
|
| OG001 | DFX Granule | Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|
|
|
Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|
| OG001 |
| DFX Granules - Core and Optional Extension Phase |
Participants received deferasirox (DFX) granules orally once daily based on body weight in the form of stick packs for 48 weeks in the Core phase. The starting dose was 14 mg/kg/day which was then adjusted based on tolerability, safety and efficacy considerations. After the Core phase, participants could enter the Optional Extension phase and continued receiving DFX granules at the same dose as was given at the end of the Core phase taking dose adjustment guidelines into account for up to 5 years. |
|
|