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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-002282-61 | EudraCT Number |
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Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox dispersible tablet (DT) in patient with transfusion - dependent thalassemia or non-transfusion -dependent thalassemia as measured by preference questionnaire at Week 48
This was an open-label, multicenter, single arm, phase II study aimed at collecting data on preference for iron chelation therapy in patients with transfusion-dependent thalassemia (TDT) or non-transfusion-dependent thalassemia (NTDT) throughout a 48 week treatment period. Participants who were either chelator-naïve, or who were previously treated with iron chelators (excluding deferasirox) for at least 6 months continuously, were eligible to participate in this study.
The study was divided into 2 phases:
Core Phase:
At the discretion of the investigator, patients could switch from deferasirox DT to deferasirox FCT at any time during Period 1 of the Core phase, and vice versa, from deferasirox FCT to deferasirox DT at any time during Period 2 of the Core phase. Re-switching treatments was not allowed within each period.
Extension Phase:
Participants could continue deferasirox FCT formulation as per the judgment of the investigator, through an extension phase for a maximum of 48 weeks months from the last dose of deferasirox FCT received at the end of period 2 in the Core Phase or until one of the end of study criteria defined is met, whichever came first. Participation in the extension phase was optional.
The end of study was defined as the earliest occurrence of one of the following:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Deferasirox DT followed by deferasirox FCT | Experimental | Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deferasirox dispersable tablet (DT) | Drug | Deferasirox DT was provided as 125 mg, 250 mg, 500 mg dispersible tablets for oral use. The strengths provided in an individual country could differ and reflected the strengths available commercially in each country. For iron chelation naive participants, the starting dose on Baseline Day 1 was 20 mg/kg/day in TDT and 10 mg/kg/day in NTDT. For iron chelation (deferoxamine and/or deferiprone) pre-treated participants, the starting dose was equivalent to the dose of deferoxamine received (for participants pre-treated with deferoxamine) and based on their serum ferritin levels (for participants pre-treated with deferiprone). Participants took deferasirox DT once daily for 24 weeks (core phase). The required number of deferasirox DT tablets were to be dispersed with gentle stirring in a glass of water. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2) | Number of participants preferring deferasirox FCT or DT as measured by preference questionnaire (item 2) at Week 48. The preference questionnaire was a 3-item questionnaire. At Week 48, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Film coated tablet" (=deferasirox FCT), "Sprinkle powder on food" (=deferasirox FCT) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. The analysis was performed for participants who answered the item 2 of the preference questionnaire. | Week 48 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2) | Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 28. The preference questionnaire was a 3-item questionnaire. At Week 28, the second item of this questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Film coated tablet (taken once a day)" (=deferasirox FCT), "Sprinkle powder on food" (=deferasirox FCT), "Tablet (taken 3 times a day)" (=previous iron chelation therapy), "Injection" (=previous iron chelation therapy) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered the item 2 of the preference questionnaire. |
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Inclusion Criteria:
Prior to any screening procedures were performed, written informed consent/assent must be provided. For pediatric patients, consent was obtained from parent(s) or legal patient's representative. Investigators also obtained assent of patients according to local, regional or national guidelines.
Male and female patient aged ≥ 2 years
Deferasirox naïve patient or chelated naive patient or treated by other chelators for at least 6 months, such as:
Subject was willing to discontinue current iron chelation therapy at least 5 days prior to study day 1 and for the duration of the study
Patients with transfusion-dependent thalassemia (independent of underlying condition) with transfusional iron overload as shown by a serum ferritin level of > 1000 ng/ml at screening and if available, LIC > 3 mg Fe/g dw within 6 months prior to screening
Patients with non-transfusion-dependent thalassemia with iron overload as shown by a serum ferritin level of ≥ 800 ng/ml at screening and if available, LIC ≥ 5 mg Fe/g dw within 6 months prior to screening
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Alexandria | 21131 | Egypt | |||
| 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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Participants took part in 17 investigative sites in 7 countries
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| ID | Title | Description |
|---|---|---|
| FG000 | Deferasirox DT Followed by Deferasirox FCT | Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Core Phase - Period 1 |
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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 | Feb 12, 2019 | Sep 6, 2021 |
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| Deferasirox film coated tablet (FCT) | Drug | Deferasirox FCT was provided as 90 mg, 180 mg, 360 mg film coated tablets for oral use. The FCT starting dose on Week 25 was 14 mg/kg/day in TDT and 7 mg/kg/day in NTDT. Participants took deferasirox FCT once daily for 24 weeks during the core phase and up to 48 weeks during the extension phase. For patients with difficulties in swallowing deferasirox FCT, it was allowed to crush the film-coated tablets and administer the study drug by sprinkling the full dose on soft food (like yogurt or apple puree). |
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| Week 28 |
| Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2) | Number of participants preferring deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 4 and 24. The preference questionnaire was a 3-item questionnaire. At Week 4 and 24, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Tablet (taken 3 times a day)" (=previous iron chelation therapy), "Injection" (=previous iron chelation therapy) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered item 2 of the preference questionnaire. | Week 4 and Week 24 |
| Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 | The preference questionnaire was a 3 item questionnaire. The first item asked the patients (or parents of young patients from 2 to 9 years old) which medicine they were taking. The second item asked which of the medicines did the patient "Like best". Finally, the third item asked the patient why he/she preferred the medicine they chose in the second item. The number of participants who selected each response option for item 3 was assessed. Participants could select multiple reasons for treatment preference at each timepoint. | Week 28 and Week 48 |
| Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods | The percentage of consumed tablet counts (compliance) was calculated for each treatment period in the core phase: deferasirox DT (period 1) and deferasirox FCT (period 2). Compliance was defined as the total tablet count consumed divided by total tablet count prescribed and multiplied by 100. Total tablet count consumed was calculated as total number of tablets dispensed minus total number of tablets lost/wasted or returned. Total tablet count prescribed was calculated as the number of tablets that the patient should have taken during this period. If a patient did not return the study drug, the compliance was not calculated. | Deferasirox DT: From Baseline up to Week 24. Deferasirox FCT: From Week 25 up to Week 48 |
| Change Over Time in Aftertaste Score of Palatability Questionnaire | The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. The aftertaste item scored on a 5-point response scale with the response option: Very good = 1, Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5. This item offered an additional response option of "no aftertaste". The aftertaste score was calculated among participants who had an aftertaste. Higher aftertaste scores indicated a worse aftertaste. For participants less than (<) 10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm remained the same as for participants greater than or equal to (≥) 10 years old. Change in aftertaste score over time was assessed | Week 4, 24, 28 and 48 |
| Change Over Time in Palatability Score of Palatability Questionnaire | The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response scale. The other two items measured what happened after taking the medicine and how the perceived amount of liquid taken with the medicine was. Responses to these 3 items were combined and converted into a single palatability score using a scoring matrix: each combination of responses on each of 3 items corresponded to a predefined palatability score. E.g. if a participant responded "bad" to item 1, "vomited <30min" to item 2 and "not enough" to item 3, then the palatability score assigned was 0. This score ranged from 0 to 11; higher scores indicated better palatability. For participants <10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm were the same as for participants ≥10 years old. Change in palatability score over time was assessed | Week 4, 24, 28 and 48 |
| Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire | The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The adherence domain score consisted of 6 adherence items, measured using a 5-point response scale. The adherence score was calculated by summing these 6 items, with scores ranging from 6 to 30. Higher scores indicated worse adherence. For participants <10 years old, an observer version (ObsRO) was administered. The adherence score remained the same as for participants ≥10 years old. | Baseline (week 2 or, if missing, week 3), week 24, 28 and 48 |
| Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire | The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The preference/satisfaction domain score consisted of 2 preference/satisfaction items, measured using a 5-point response scale. The preference score was calculated by summing these 2 items, with scores ranging from 2 to 10. Higher scores indicated worse satisfaction. For participants < 10 years old, an observer version (ObsRO) was administered. Preference score remained the same as for participants ≥ 10 years old. | Baseline (week 2 or, if missing, week 3), week 24, 28 and 48 |
| Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire | The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The concerns domain score consisted of 3 items to address any concerns or worries with the medication. All 3 items were measured on a 5-point response scale. The concerns score was calculated by summing the 3 items, with scores ranging from 3 to 15. Higher scores indicated fewer concerns. For participants < 10 years old, an observer version (ObsRO) was administered. Concerns score remained the same as for participants ≥ 10 years old. | Baseline (week 2 or, if missing, week 3), week 24, 28 and 48 |
| Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire | The GI symptom score was calculated from responses to 5 questions of the GI questionnaire, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. An observer GI symptom questionnaire was administered to those patients who were < 10 years old. The questionnaire was completed by the parents of the participants. All items and the scoring algorithm remained the same as for participants ≥ 10 years old. | Baseline (week -1 or, if missing, week -2), week 24, 28 and 48 |
| Change From Baseline in Serum Ferritin Levels | Absolute change from baseline over time in serum ferritin levels | From Baseline (Day 1) up to 96 weeks |
| Cairo |
| 11562 |
| Egypt |
| Novartis Investigative Site | Zagazig | 44519 | Egypt |
| Novartis Investigative Site | Hazmiyeh | Beirut | PO BOX 213 | Lebanon |
| Novartis Investigative Site | Rabat | 10102 | Morocco |
| Novartis Investigative Site | Al Ahsa | SAU | Saudi Arabia |
| Novartis Investigative Site | Jeddah | 21159 | Saudi Arabia |
| Novartis Investigative Site | Jeddah | 21589 | Saudi Arabia |
| Novartis Investigative Site | Riyadh | 11117 | Saudi Arabia |
| Novartis Investigative Site | Bangkok Noi | Bangkok | 10700 | Thailand |
| Novartis Investigative Site | Bangkoknoi | Bangkok | 10700 | Thailand |
| Novartis Investigative Site | Bangkok | 10400 | Thailand |
| Novartis Investigative Site | Ankara | 06100 | Turkey (Türkiye) |
| Novartis Investigative Site | Antalya | 07070 | Turkey (Türkiye) |
| Novartis Investigative Site | Istanbul | 34093 | Turkey (Türkiye) |
| Novartis Investigative Site | Hanoi | Vietnam |
| Novartis Investigative Site | Ho Chi Minh City | DISTRICT 1 | Vietnam |
| COMPLETED |
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| NOT COMPLETED |
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| Core Phase- Period 2 |
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| Extension Phase |
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| ID | Title | Description |
|---|---|---|
| BG000 | Deferasirox DT Followed by Deferasirox FCT | Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT |
| 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: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | 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 | Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2) | Number of participants preferring deferasirox FCT or DT as measured by preference questionnaire (item 2) at Week 48. The preference questionnaire was a 3-item questionnaire. At Week 48, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Film coated tablet" (=deferasirox FCT), "Sprinkle powder on food" (=deferasirox FCT) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. The analysis was performed for participants who answered the item 2 of the preference questionnaire. | Full Analysis Set (FAS): Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Only participants with available data for this outcome measure were included in this analysis. | Posted | Count of Participants | Participants | Week 48 |
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| Secondary | Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2) | Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 28. The preference questionnaire was a 3-item questionnaire. At Week 28, the second item of this questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Film coated tablet (taken once a day)" (=deferasirox FCT), "Sprinkle powder on food" (=deferasirox FCT), "Tablet (taken 3 times a day)" (=previous iron chelation therapy), "Injection" (=previous iron chelation therapy) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered the item 2 of the preference questionnaire. | Participants in the FAS [to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT)] and who received iron chelation therapy prior to enrolling in the study. Only participants with available data for this outcome measure were included in this analysis. | Posted | Count of Participants | Participants | Week 28 |
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| Secondary | Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2) | Number of participants preferring deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 4 and 24. The preference questionnaire was a 3-item questionnaire. At Week 4 and 24, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Tablet (taken 3 times a day)" (=previous iron chelation therapy), "Injection" (=previous iron chelation therapy) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered item 2 of the preference questionnaire. | Participants in the FAS [to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT)] and who received iron chelation therapy prior to enrolling in the study. Number analyzed signified number of participants with available data for this outcome measure at specified timepoints. | Posted | Count of Participants | Participants | Week 4 and Week 24 |
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| Secondary | Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 | The preference questionnaire was a 3 item questionnaire. The first item asked the patients (or parents of young patients from 2 to 9 years old) which medicine they were taking. The second item asked which of the medicines did the patient "Like best". Finally, the third item asked the patient why he/she preferred the medicine they chose in the second item. The number of participants who selected each response option for item 3 was assessed. Participants could select multiple reasons for treatment preference at each timepoint. | FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). | Posted | Count of Participants | Participants | Week 28 and Week 48 |
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| Secondary | Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods | The percentage of consumed tablet counts (compliance) was calculated for each treatment period in the core phase: deferasirox DT (period 1) and deferasirox FCT (period 2). Compliance was defined as the total tablet count consumed divided by total tablet count prescribed and multiplied by 100. Total tablet count consumed was calculated as total number of tablets dispensed minus total number of tablets lost/wasted or returned. Total tablet count prescribed was calculated as the number of tablets that the patient should have taken during this period. If a patient did not return the study drug, the compliance was not calculated. | FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure for each treatment period | Posted | Mean | Standard Deviation | Percentage of tablet counts | Deferasirox DT: From Baseline up to Week 24. Deferasirox FCT: From Week 25 up to Week 48 |
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| Secondary | Change Over Time in Aftertaste Score of Palatability Questionnaire | The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. The aftertaste item scored on a 5-point response scale with the response option: Very good = 1, Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5. This item offered an additional response option of "no aftertaste". The aftertaste score was calculated among participants who had an aftertaste. Higher aftertaste scores indicated a worse aftertaste. For participants less than (<) 10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm remained the same as for participants greater than or equal to (≥) 10 years old. Change in aftertaste score over time was assessed | FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints. | Posted | Mean | Standard Deviation | Score on a Scale | Week 4, 24, 28 and 48 |
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| Secondary | Change Over Time in Palatability Score of Palatability Questionnaire | The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response scale. The other two items measured what happened after taking the medicine and how the perceived amount of liquid taken with the medicine was. Responses to these 3 items were combined and converted into a single palatability score using a scoring matrix: each combination of responses on each of 3 items corresponded to a predefined palatability score. E.g. if a participant responded "bad" to item 1, "vomited <30min" to item 2 and "not enough" to item 3, then the palatability score assigned was 0. This score ranged from 0 to 11; higher scores indicated better palatability. For participants <10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm were the same as for participants ≥10 years old. Change in palatability score over time was assessed | FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints. | Posted | Mean | Standard Deviation | Score on a Scale | Week 4, 24, 28 and 48 |
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| Secondary | Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire | The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The adherence domain score consisted of 6 adherence items, measured using a 5-point response scale. The adherence score was calculated by summing these 6 items, with scores ranging from 6 to 30. Higher scores indicated worse adherence. For participants <10 years old, an observer version (ObsRO) was administered. The adherence score remained the same as for participants ≥10 years old. | FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints | Posted | Mean | Standard Deviation | Score on a scale | Baseline (week 2 or, if missing, week 3), week 24, 28 and 48 |
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| Secondary | Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire | The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The preference/satisfaction domain score consisted of 2 preference/satisfaction items, measured using a 5-point response scale. The preference score was calculated by summing these 2 items, with scores ranging from 2 to 10. Higher scores indicated worse satisfaction. For participants < 10 years old, an observer version (ObsRO) was administered. Preference score remained the same as for participants ≥ 10 years old. | FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints | Posted | Mean | Standard Deviation | Score on a scale | Baseline (week 2 or, if missing, week 3), week 24, 28 and 48 |
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| Secondary | Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire | The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The concerns domain score consisted of 3 items to address any concerns or worries with the medication. All 3 items were measured on a 5-point response scale. The concerns score was calculated by summing the 3 items, with scores ranging from 3 to 15. Higher scores indicated fewer concerns. For participants < 10 years old, an observer version (ObsRO) was administered. Concerns score remained the same as for participants ≥ 10 years old. | FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints | Posted | Mean | Standard Deviation | Score on a scale | Baseline (week 2 or, if missing, week 3), week 24, 28 and 48 |
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| Secondary | Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire | The GI symptom score was calculated from responses to 5 questions of the GI questionnaire, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. An observer GI symptom questionnaire was administered to those patients who were < 10 years old. The questionnaire was completed by the parents of the participants. All items and the scoring algorithm remained the same as for participants ≥ 10 years old. | FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints | Posted | Mean | Standard Deviation | Score on a scale | Baseline (week -1 or, if missing, week -2), week 24, 28 and 48 |
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| Secondary | Change From Baseline in Serum Ferritin Levels | Absolute change from baseline over time in serum ferritin levels | FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints | Posted | Mean | Standard Deviation | microgram/liter (ug/L) | From Baseline (Day 1) up to 96 weeks |
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Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase).
Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Deferasirox DT (Core Phase) | Participants who were treated with deferasirox DT once daily in the core phase | 0 | 148 | 13 | 148 | 82 | 148 |
| EG001 | Deferasirox FCT (Core Phase) | Participants who were treated with deferasirox FCT once daily in the core phase | 0 | 140 | 6 | 140 | 59 | 140 |
| EG002 | Deferasirox FCT (Extension Phase) | Participants who were treated with deferasirox FCT once daily in the extension phase | 0 | 116 | 16 | 116 | 49 | 116 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemolysis | Blood and lymphatic system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Cardiac failure chronic | Cardiac disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Optic nerve cupping | Eye disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Cholangitis acute | Hepatobiliary disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Jaundice cholestatic | Hepatobiliary disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Diarrhoea infectious | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Ecthyma | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Abdominal injury | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Gastrointestinal injury | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Splenic rupture | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Ulna fracture | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (23.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (23.1) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (23.1) | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA (23.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Cholangiocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (23.1) | Systematic Assessment |
| |
| Polyneuropathy | Nervous system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (23.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Serum ferritin increased | Investigations | MedDRA (23.1) | Systematic Assessment |
| |
| Urine protein/creatinine ratio increased | Investigations | MedDRA (23.1) | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA (23.1) | 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 (ie, data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study director | Novartis Pharmaceuticals | 862-778-8300 | novartis.email@novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 8, 2021 | Sep 6, 2021 | SAP_001.pdf |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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
| Lost to Follow-up |
|
| Deferasirox FCT locally reimbursed |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
|---|---|
| Participants |
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