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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-002529-12 | EudraCT Number |
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Due to enrolment challenge
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The purpose of this study was to evaluate the efficacy and safety of deferasirox film coated tablet (FCT) versus phlebotomy for the management of iron overload in adults with Hereditary Hemochromatosis (HH) at risk of iron-related morbidity. This evaluation provided information on the two treatment options in terms of the rate of response of proportion of patients reaching the study target SF ≤ 100 μg/L and their associated safety profiles.
In addition to exploring the safety and efficacy of deferasirox FCT in hereditary hemochromatosis (HH), this study is being conducted to fulfill an FDA post-marketing requirement [PMC 750-10 (Exjade) /PMR 2888-8 (Jadenu)] to provide additional randomized data to confirm the ocular safety profile of deferasirox through detailed ocular assessments in patients treated with deferasirox FCT for 2 years.
This was a Phase II, multicenter, open-label, randomized two-year study in adults with Hereditary Hemochromatosis (HH) confirmed by HH genotype with iron overload. Eligible subjects were identified during a 4-week screening period, then randomized in a 2:1 ratio to be treated with deferasirox FCT or phlebotomy for up to 24 months (104 weeks).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Deferasirox FCT Arm | Experimental | An initial dose of deferasirox FCT 7 mg/kg/day was used for 3 months (12 weeks), then was adjusted according to the serum ferritin (SF) level. Deferasirox was interrupted when the study-defined target SF ≤ 100 μg/L was achieved. Deferasirox was to be reinitiated when SF ≥ 300 μg/L, according to deferasirox label. |
|
| phlebotomy | Active Comparator | Phlebotomy was conducted at a frequency determined by the physician. Phlebotomy was interrupted when the study-defined target SF ≤ 100 μg/L was achieved. Phlebotomy was to be reinitiated when SF > 100 μg/L, based on standard of care practice. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deferasirox FCT | Drug | Taken orally once per day (QD) as a film coated tablet (FCT) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Patients Achieving Target SF ≤ 100 μg/L for the First Time | Proportion of participants achieving target serum ferritin (SF) ≤ 100 μg/L on or before Month 24. Participants were considered responders if they met response criteria (target SF ≤100 µg/L) on or before Month 24 (Week 104) during the treatment phase. Any participant who discontinued treatment prematurely before meeting such criterion and participants with unknown or missing SF by Month 24 were counted as non-responder. | Up to Month 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) | Number of participants with at least one ocular treatment emergent adverse event (new or worsening from baseline). | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. |
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Inclusion Criteria:
Written informed consent must be obtained prior to any screening procedures.
Patients eligible for inclusion must meet all following criteria prior to receiving study treatment:
1. Male or female ≥ 18-years-old 2. Documented genotype testing confirming homozygous for the C282Y mutation (C282Y/C282Y) 3. Transferrin saturation ≥ 45% (at either screening visit) 4. Serum ferritin (SF) ≥ 500 μg/L (at either screening visit)
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Exclusion Criteria:
Medical conditions that preclude inclusion:
Concomitant therapy that precludes enrollment:
Abnormal Laboratory Values:
Participation in an investigational study:
Pregnancy and contraception:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Leuven | 3000 | Belgium | |||
| Novartis Investigative Site |
Not provided
| Label | URL |
|---|---|
| A Plain Language Trial Summary is available on novctrd.com | View source |
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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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There was a screening period of 4 weeks to assess participants eligibility.
Participants took part in 11 investigative sites in 7 countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | Deferasirox FCT 7mg/kg | Deferasirox film-coated tablet 7mg/kg, oral dose daily (starting dose for the first 12 weeks) |
| FG001 | Phlebotomy | Phlebotomy - standard of care |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Aug 30, 2018 | Apr 10, 2024 |
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| Phlebotomy | Procedure | according to investigator's decision |
|
| Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) by Preferred Term | Number of participants with at least one ocular treatment emergent adverse event (new or worsening from baseline). Preferred terms are based on Medical Dictionary of Regulatory Activities (MedDRA) version 26.0. | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. |
| Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Number of participants with treatment emergent AEs (any AE regardless of seriousness), AEs leading to study treatment discontinuation, and SAEs. | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. |
| Number of Adverse Events in Participants Who Had Study Treatment Interrupted Due to SF ≤ 100 μg/L and Re-initiated Study Treatment When ≥ 300 μg/L | Number of participants with Adverse Events who interrupt deferasirox FCT at least once due to SF level ≤ 100 μg/L and re-initiate therapy at SF level ≥ 300 μg/L. There were no participants that re-initiated therapy when reached 300 ug/L. | Up to 24 months |
| Categorical Analysis of logMAR Score Changes From Baseline to Best Post-baseline Changes in One Eye With More Extreme Change | Visual acuity was measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A letter score was calculated based on the number of letters that could correctly be identified from specified distances. For low luminance and standard acuity measures, visual acuity was described on a logMAR scale for all measures. For including acuity obtained with the ETDRS letter score, the values were converted to a logMAR scale, using the following formula: logMAR = 1.7-0.02*ETDRS score. With this conversion, a difference from baseline of 0.1 logMAR = 5-letter difference in visual acuity, 0.2 logMAR = 10-letter difference, 0.3 logMAR = 15-letter difference, 0.4 logMAR = 20-letter difference, 0.5 logMAR = 25-letter difference and 0.6 logMAR = 30-letter difference. Decrease in logMAR score category from baseline indicates improvement in visual acuity. The best change from baseline amongst all post baseline visit is presented. | Baseline, Weeks 24, 52, 76 and 104. |
| Categorical Analysis of logMAR Score Changes From Baseline to Worst Post-baseline Changes in One Eye With More Extreme Change | Visual acuity was measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A letter score was calculated based on the number of letters that could correctly be identified from specified distances. For low luminance and standard acuity measures, visual acuity was described on a logMAR scale for all measures. For including acuity obtained with the ETDRS letter score, the values were converted to a logMAR scale, using the following formula: logMAR = 1.7-0.02*ETDRS score. With this conversion, a difference from baseline of 0.1 logMAR = 5-letter difference in visual acuity, 0.2 logMAR = 10-letter difference, 0.3 logMAR = 15-letter difference, 0.4 logMAR = 20-letter difference, 0.5 logMAR = 25-letter difference and 0.6 logMAR = 30-letter difference. Increase in logMAR score from baseline indicates worsening in visual acuity. The worst change from baseline amongst all post baseline visit is presented. | Baseline, Weeks 24, 52, 76 and 104. |
| Categorical Analysis of Worst Post-baseline Values of Intraocular Pressure in One Eye With More Extreme Change | Intraocular pressure was measured by tonometry. Intraocular pressure values >5 to ≤21 mmHg were considered normal. The worst post-baseline value corresponds to the worst outcome amongst all post baseline visit | Weeks 24, 52, 76 and 104 |
| Categorical Analysis of Changes in Intraocular Pressure From Baseline to Best/Worst Post-baseline Changes in One Eye With More Extreme Change | Intraocular pressure was measured by tonometry. A decrease in intraocular pressure from baseline indicated improvement. | Baseline, up to Week 104 |
| Number of Participants With Slit Lamp Results for Any Evaluation and Worst Eye | Slit lamp examination was used to evaluate lids, cornea, conjunctiva, iris, anterior chamber, aqueous flare, aqueous inflammatory cells and lens. Any post-baseline abnormalities (not present at baseline) in slit lamp examination were assesses by the investigator and classified as insignificant or clinically significant. Number of participants with slit lamp results (normal, insignificant, significant, missing) for any evaluation and worst eye are reported. | Baseline, up to Week 104 |
| Number of Participants With an Increase From Baseline of ≥1 and ≥2 in LOCS III Grades | Lens Opacities Classification System III (LOCS III) grading scales include lens opacities defined as nuclear opalescence (NO), nuclear color (NC), cortical (C) cataract and posterior subcapsular (P) cataract with several degrees of extend, i.e. severity. The LOCS III scale for nuclear opalescence and for nuclear color ranges from 0 to 6. The LOCS III scale for cortical cataract and posterior subcapsular cataract opacity ranges from 0 to 5. For all scales, higher values indicate higher opacity, opalescence, or color (range: NO0/NC0/C0/P0 to NO6/NC6/C5/P5). Number of participants with an increase from baseline of ≥1 and increase of ≥2 in LOCS III grades is reported. | Baseline, up to Week 104 |
| Number of Participants With Fundus Oculi Results for Any Evaluation and Worst Eye | Fundus oculi examination was used to evaluate peripheral retina, macula, optic nerve, and vitreous hemorrhage. Any post-baseline abnormalities (not present at baseline) in fundus oculi examination were assessed by the investigator and classified as insignificant or clinically significant. Number of participants with fundus oculi results (normal, insignificant, significant, missing) for any evaluation and worst eye are reported. | Baseline, up to Week 104 |
| Time to Response (TTR) | Time to response (TTR) is defined as the time from the date of randomization to the date of the first time the SF achieved a value ≤ 100 μg/L during the treatment phase. Participants who did not achieve SF ≤ 100 μg/L were censored as follows: at the last serum ferritin assessment date on or before month 24 (week 104), at the day of randomization if a subject does not have any post-baseline serum ferritin value or at the death date. TTR was analyzed using the Kaplan-Meier method. | Up to Month 24 |
| Limoges |
| 87042 |
| France |
| Novartis Investigative Site | Rennes | 35043 | France |
| Novartis Investigative Site | Sibiu | 550245 | Romania |
| Novartis Investigative Site | Moscow | 125167 | Russia |
| Novartis Investigative Site | Bratislava | 831 01 | Slovakia |
| Novartis Investigative Site | Bratislava | 85107 | Slovakia |
| Novartis Investigative Site | Manresa | Espana | 08241 | Spain |
| Novartis Investigative Site | Barakaldo | Vizcaya | 48903 | Spain |
| Novartis Investigative Site | Las Palmas de Gran Canaria | 35010 | Spain |
| Novartis Investigative Site | Lugano | 6900 | Switzerland |
| COMPLETED |
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| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Deferasirox FCT 7mg/kg | Deferasirox film-coated tablet 7mg/kg, oral dose daily (starting dose for the first 12 weeks) |
| BG001 | Phlebotomy | Phlebotomy - standard of care |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| 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 | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Proportion of Patients Achieving Target SF ≤ 100 μg/L for the First Time | Proportion of participants achieving target serum ferritin (SF) ≤ 100 μg/L on or before Month 24. Participants were considered responders if they met response criteria (target SF ≤100 µg/L) on or before Month 24 (Week 104) during the treatment phase. Any participant who discontinued treatment prematurely before meeting such criterion and participants with unknown or missing SF by Month 24 were counted as non-responder. | The Full Analysis Set (FAS) comprised all subjects to whom study treatment had been assigned by randomization. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to Month 24 |
|
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) | Number of participants with at least one ocular treatment emergent adverse event (new or worsening from baseline). | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) by Preferred Term | Number of participants with at least one ocular treatment emergent adverse event (new or worsening from baseline). Preferred terms are based on Medical Dictionary of Regulatory Activities (MedDRA) version 26.0. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Number of participants with treatment emergent AEs (any AE regardless of seriousness), AEs leading to study treatment discontinuation, and SAEs. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Adverse Events in Participants Who Had Study Treatment Interrupted Due to SF ≤ 100 μg/L and Re-initiated Study Treatment When ≥ 300 μg/L | Number of participants with Adverse Events who interrupt deferasirox FCT at least once due to SF level ≤ 100 μg/L and re-initiate therapy at SF level ≥ 300 μg/L. There were no participants that re-initiated therapy when reached 300 ug/L. | Participants in the Safety Set who had study treatment interrupted due to SF ≤ 100 μg/L and re-initiated study treatment when ≥ 300 μg/L. There were no patients who re-initiated therapy when reached SF 300 μg/L. | Posted | Up to 24 months |
|
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Categorical Analysis of logMAR Score Changes From Baseline to Best Post-baseline Changes in One Eye With More Extreme Change | Visual acuity was measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A letter score was calculated based on the number of letters that could correctly be identified from specified distances. For low luminance and standard acuity measures, visual acuity was described on a logMAR scale for all measures. For including acuity obtained with the ETDRS letter score, the values were converted to a logMAR scale, using the following formula: logMAR = 1.7-0.02*ETDRS score. With this conversion, a difference from baseline of 0.1 logMAR = 5-letter difference in visual acuity, 0.2 logMAR = 10-letter difference, 0.3 logMAR = 15-letter difference, 0.4 logMAR = 20-letter difference, 0.5 logMAR = 25-letter difference and 0.6 logMAR = 30-letter difference. Decrease in logMAR score category from baseline indicates improvement in visual acuity. The best change from baseline amongst all post baseline visit is presented. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Baseline, Weeks 24, 52, 76 and 104. |
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Categorical Analysis of logMAR Score Changes From Baseline to Worst Post-baseline Changes in One Eye With More Extreme Change | Visual acuity was measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A letter score was calculated based on the number of letters that could correctly be identified from specified distances. For low luminance and standard acuity measures, visual acuity was described on a logMAR scale for all measures. For including acuity obtained with the ETDRS letter score, the values were converted to a logMAR scale, using the following formula: logMAR = 1.7-0.02*ETDRS score. With this conversion, a difference from baseline of 0.1 logMAR = 5-letter difference in visual acuity, 0.2 logMAR = 10-letter difference, 0.3 logMAR = 15-letter difference, 0.4 logMAR = 20-letter difference, 0.5 logMAR = 25-letter difference and 0.6 logMAR = 30-letter difference. Increase in logMAR score from baseline indicates worsening in visual acuity. The worst change from baseline amongst all post baseline visit is presented. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Baseline, Weeks 24, 52, 76 and 104. |
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Categorical Analysis of Worst Post-baseline Values of Intraocular Pressure in One Eye With More Extreme Change | Intraocular pressure was measured by tonometry. Intraocular pressure values >5 to ≤21 mmHg were considered normal. The worst post-baseline value corresponds to the worst outcome amongst all post baseline visit | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Weeks 24, 52, 76 and 104 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Categorical Analysis of Changes in Intraocular Pressure From Baseline to Best/Worst Post-baseline Changes in One Eye With More Extreme Change | Intraocular pressure was measured by tonometry. A decrease in intraocular pressure from baseline indicated improvement. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Baseline, up to Week 104 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Slit Lamp Results for Any Evaluation and Worst Eye | Slit lamp examination was used to evaluate lids, cornea, conjunctiva, iris, anterior chamber, aqueous flare, aqueous inflammatory cells and lens. Any post-baseline abnormalities (not present at baseline) in slit lamp examination were assesses by the investigator and classified as insignificant or clinically significant. Number of participants with slit lamp results (normal, insignificant, significant, missing) for any evaluation and worst eye are reported. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Baseline, up to Week 104 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With an Increase From Baseline of ≥1 and ≥2 in LOCS III Grades | Lens Opacities Classification System III (LOCS III) grading scales include lens opacities defined as nuclear opalescence (NO), nuclear color (NC), cortical (C) cataract and posterior subcapsular (P) cataract with several degrees of extend, i.e. severity. The LOCS III scale for nuclear opalescence and for nuclear color ranges from 0 to 6. The LOCS III scale for cortical cataract and posterior subcapsular cataract opacity ranges from 0 to 5. For all scales, higher values indicate higher opacity, opalescence, or color (range: NO0/NC0/C0/P0 to NO6/NC6/C5/P5). Number of participants with an increase from baseline of ≥1 and increase of ≥2 in LOCS III grades is reported. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Baseline, up to Week 104 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Fundus Oculi Results for Any Evaluation and Worst Eye | Fundus oculi examination was used to evaluate peripheral retina, macula, optic nerve, and vitreous hemorrhage. Any post-baseline abnormalities (not present at baseline) in fundus oculi examination were assessed by the investigator and classified as insignificant or clinically significant. Number of participants with fundus oculi results (normal, insignificant, significant, missing) for any evaluation and worst eye are reported. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Posted | Count of Participants | Participants | Baseline, up to Week 104 |
|
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| Secondary | Time to Response (TTR) | Time to response (TTR) is defined as the time from the date of randomization to the date of the first time the SF achieved a value ≤ 100 μg/L during the treatment phase. Participants who did not achieve SF ≤ 100 μg/L were censored as follows: at the last serum ferritin assessment date on or before month 24 (week 104), at the day of randomization if a subject does not have any post-baseline serum ferritin value or at the death date. TTR was analyzed using the Kaplan-Meier method. | The Full Analysis Set (FAS) comprised all subjects to whom study treatment has been assigned by randomization. | Posted | Median | 95% Confidence Interval | months | Up to Month 24 |
|
|
Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks.
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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 FCT 7mg/kg | Deferasirox film-coated tablet 7mg/kg, oral dose daily (starting dose for the first 12 weeks) | 1 | 30 | 7 | 30 | 23 | 30 |
| EG001 | Phlebotomy | Phlebotomy - standard of care | 0 | 15 | 0 | 15 | 12 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Sudden death | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Erysipelas | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Bladder neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.0) | Systematic Assessment |
| |
| Thoracic outlet syndrome | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA (26.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Sinus bradycardia | Cardiac disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Deafness unilateral | Ear and labyrinth disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Middle ear inflammation | Ear and labyrinth disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Phobic postural vertigo | Ear and labyrinth disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Cataract nuclear | Eye disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Glaucoma | Eye disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Gastrointestinal pain | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Vaccination site pain | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Bone abscess | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Escherichia urinary tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Suspected COVID-19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood cholesterol increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Blood uric acid increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Glycosylated haemoglobin increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Vitamin D decreased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Folate deficiency | Metabolism and nutrition disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Vitamin B12 deficiency | Metabolism and nutrition disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Taste disorder | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Catarrh | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (26.0) | Systematic Assessment |
|
The results should be interpreted with caution due to low number of participants enrolled in both arms.
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 | May 22, 2023 | Apr 10, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D006432 | Hemochromatosis |
| D019190 | Iron Overload |
| ID | Term |
|---|---|
| D008664 | Metal Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000077588 | Deferasirox |
| D018962 | Phlebotomy |
| 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 |
| D001800 | Blood Specimen Collection |
| D013048 | Specimen Handling |
| D019411 | Clinical Laboratory Techniques |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
| D011677 | Punctures |
| D013812 | Therapeutics |
| D013514 | Surgical Procedures, Operative |
| D008919 | Investigative Techniques |
Not provided
Not provided
| Male |
|
|
|
|
|
|
|
|
|
|
|
| Participants |
|
|
|
|
| Significant |
|
| Missing |
|
| Significant |
|
| Missing |
|