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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-003069-14 |
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This study was designed to assess the evolution of renal function and to collect efficacy, safety, and tolerability data of everolimus in co-exposure with reduced CNI in paediatric liver transplant recipients.
Study is completed (was active and ongoing but no longer recruiting since December 2014). The study Data Monitoring Committee meeting communicated to Novartis the following safety findings in the study population: high rate of premature discontinuation of study medication, high rate of post-transplant lymphoproliferative disease and high rate of related serious infections leading to hospitalization. In light of the safety findings, Novartis followed the DMC recommendation to discontinue the study medication in this age group and to stop enrolling new patients in this study (regardless of age).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Everolimus based regimen | Experimental | Conversion at Baseline from an immunosuppressive regimen which contains either cyclosporine (CsA) or tacrolimus (TAC) with or without mycophenolic acid (MPA), with or without corticosteroids in a regimen which contains everolimus combined reduced dose of either cyclosporine (CsA) or tacrolimus (TAC). The dosing schedule was twice daily, 12 hours apart. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Introduction of everolimus with reduced cyclosporine or tacrolimus dose, the earliest 1 month and the latest 6 months after liver transplantation. | Drug | Immunosuppression after liver transplantation. Pediatric transplant recipients received a starting dose of 0.8 mg/m^2/dose in combination wit Cyclosporine A or 2.0 mg/m^2/dose in combination with tacrolimus, twice-daily. Thereafter, doses were adjusted to achieve everolimus C-0h blood trough level between 3 to 8 ng/ml. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Estimated Glomerular Filtration Rate - Month 12 | Evolution of renal function assessed by estimated Glomerular Filtration Rate (eGFR) calculated by the Chronic Kidney Disease in Children (CKiD) Schwartz formula (Schwartz 2009), expressed in mean change in eGFR of CKiD between start of study (baseline assessment) and Month 12. | Baseline, Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Kaplan-Meier Estimates for Failure Rates of Efficacy Endpoints | The proportion of patients with composite efficacy failure (treated biopsy proven acute rejection[tBPAR], graft loss [GL] , death [D]) before/at Month 12 and Month 24, estimated with Kaplan-Meier (KM) methods and the proportion of patients who experienced any of the components of composite efficacy failure (tBPAR, GL, D) before/at Month 12 and Month 24, separately for each component. AR: acute rejection; BPAR: biopsy proven acute rejection. Rate = Kaplan-Meier estimate for failure in %; CI = confidence interval for failure rate. |
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Key Inclusion Criteria:
Signed informed consent from both parents or legal guardian(s) prior to patient participation in the study.
Paediatric liver transplant recipients aged greater than or equal to 1 month and younger than 18 years of age.
Paediatric recipients at the earliest 1 month and latest 6 month after liver transplantation.
Key Exclusion Criteria:
Patients with hepato-biliary malignancies and/or patients transplanted due to fulminant hepatitis /acute liver failure.
Presence of thrombosis of any major hepatic arteries, major/reconstructed hepatic veins, portal vein or inferior vena cava at any time prior to the start of study drug.
Patients with serum creatinine value >2 times age-related ULN at Baseline or who received renal replacement therapy within one week prior to the start of study drug and patients with a confirmed spot urine protein/creatinine ratio indicating a urinary protein excretion >500 mg/m2/24 hrs, at Baseline.
Patients with clinically significant systemic infection and/or in a critical care setting requiring life support measures such as mechanical ventilation, dialysis, or vasopressor agents.
Patients with a known hypersensitivity to the drugs used on study or their class, or to any of the excipients.
Pregnant or nursing (lactating) female patients, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive βHCG laboratory test (>9 mIU/mL) at Baseline.
Female patients of child-bearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they agree for abstinence from sexual activity.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Los Angeles | California | 90027 | United States | ||
| Novartis Investigative Site |
Overall 62 patients were screened prior to the data monitoring committee (DMC) recommendation to terminate enrolment.
Six patients were screen failures. The other 56 patients were included and treated.
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| ID | Title | Description |
|---|---|---|
| FG000 | Everolimus Based Regimen | Conversion at baseline from an immunosuppressive regimen which contains either cyclosporine (CsA) or tacrolimus (TAC) with or without mycophenolic acid (MPA), with or without corticosteroids to a regimen which contains everolimus combined with reduced dose of either cyclosporine (CsA) or tacrolimus (TAC). The dosing schedule was twice daily, 12 hours apart. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Baseline to Month 12 |
|
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|
| At 12-month and 24-month after start of study drug |
| Change From Baseline in Estimated Glomerular Filtration Rate - Month 24 | Evolution of renal function assessed by estimated Glomerular Filtration Rate (eGFR) calculated by the Chronic Kidney Disease in Children (CKiD) Schwartz formula (Schwartz 2009), expressed in mean change in eGFR of CKiD between start of study (baseline assessment) and Month 24. | Baseline, Month 24 |
| Growth Development - Height at Baseline and Month 12 | Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (<=5, >5-25, >25-50, >50-75, >75-95 and >95% percentile). | Baseline, Month 12 |
| Growth Development - Weight at Baseline and Month 12 | Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (<=5, >5-25, >25-50, >50-75, >75-95 and >95% percentile). | Baseline, Month 12 |
| Growth Development - Weight at Baseline and Month 24 | Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (<=5, >5-25, >25-50, >50-75, >75-95 and >95% percentile). | Baseline, Month 24 |
| Growth Development - Height at Baseline and Month 24 | Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (<=5, >5-25, >25-50, >50-75, >75-95 and >95% percentile). | Baseline, Month 24 |
| New Haven |
| Connecticut |
| 06520 |
| United States |
| Novartis Investigative Site | Chicago | Illinois | 60637 | United States |
| Novartis Investigative Site | St Louis | Missouri | 63110 | United States |
| Novartis Investigative Site | New York | New York | 110032 | United States |
| Novartis Investigative Site | Charleston | South Carolina | 29425 | United States |
| Novartis Investigative Site | Houston | Texas | 77030-2400 | United States |
| Novartis Investigative Site | Salt Lake City | Utah | 84132 | United States |
| Novartis Investigative Site | Madison | Wisconsin | 53792 | United States |
| Novartis Investigative Site | Parkville | Victoria | 3052 | Australia |
| Novartis Investigative Site | Brussels | 1200 | Belgium |
| Novartis Investigative Site | Edmonton | Alberta | T6G 2B7 | Canada |
| Novartis Investigative Site | København Ø | DK-2100 | Denmark |
| Novartis Investigative Site | Bron | 69677 | France |
| Novartis Investigative Site | Hanover | Germany | 30625 | Germany |
| Novartis Investigative Site | Bonn | 53105 | Germany |
| Novartis Investigative Site | Essen | 45147 | Germany |
| Novartis Investigative Site | Hamburg | 20246 | Germany |
| Novartis Investigative Site | Regensburg | 93053 | Germany |
| Novartis Investigative Site | Tübingen | 72076 | Germany |
| Novartis Investigative Site | Budapest | 1082 | Hungary |
| Novartis Investigative Site | Budapest | 1083 | Hungary |
| Novartis Investigative Site | Bergamo | BG | 24128 | Italy |
| Novartis Investigative Site | Roma | ITA | 00165 | Italy |
| Novartis Investigative Site | Padova | PD | 35128 | Italy |
| Novartis Investigative Site | Torino | TO | 10126 | Italy |
| Novartis Investigative Site | Barcelona | Catalonia | 08035 | Spain |
| Novartis Investigative Site | Madrid | Madrid | 28046 | Spain |
| Novartis Investigative Site | Stockholm | SE-141 86 | Sweden |
| Novartis Investigative Site | West Midlands | Birmingham | B4 6NH | United Kingdom |
| Novartis Investigative Site | Leeds | LS1 3EX | United Kingdom |
| Novartis Investigative Site | London | SE5 9RS | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Baseline to 24 Month |
|
|
Full analysis set (FAS): consisted of all patients enrolled into the study, including patients who did not receive any everolimus, but were followed beyond baseline.
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| ID | Title | Description |
|---|---|---|
| BG000 | Everolimus Based Regimen | Conversion at baseline from an immunosuppressive regimen which contains either cyclosporine (CsA) or tacrolimus (TAC) with or without mycophenolic acid (MPA), with or without corticosteroids to a regimen which contains everolimus combined with reduced dose of either cyclosporine (CsA) or tacrolimus (TAC). The dosing schedule was twice daily, 12 hours apart. |
| 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 |
|
| 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 | Change From Baseline in Estimated Glomerular Filtration Rate - Month 12 | Evolution of renal function assessed by estimated Glomerular Filtration Rate (eGFR) calculated by the Chronic Kidney Disease in Children (CKiD) Schwartz formula (Schwartz 2009), expressed in mean change in eGFR of CKiD between start of study (baseline assessment) and Month 12. | Full Analysis Set | Posted | Mean | Standard Deviation | mL/min/1.73m^2 | Baseline, Month 12 |
|
|
| |||||||||||||||||||||||||
| Secondary | Kaplan-Meier Estimates for Failure Rates of Efficacy Endpoints | The proportion of patients with composite efficacy failure (treated biopsy proven acute rejection[tBPAR], graft loss [GL] , death [D]) before/at Month 12 and Month 24, estimated with Kaplan-Meier (KM) methods and the proportion of patients who experienced any of the components of composite efficacy failure (tBPAR, GL, D) before/at Month 12 and Month 24, separately for each component. AR: acute rejection; BPAR: biopsy proven acute rejection. Rate = Kaplan-Meier estimate for failure in %; CI = confidence interval for failure rate. | Full Analysis Set | Posted | Number | 80% Confidence Interval | Percentages | At 12-month and 24-month after start of study drug |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Estimated Glomerular Filtration Rate - Month 24 | Evolution of renal function assessed by estimated Glomerular Filtration Rate (eGFR) calculated by the Chronic Kidney Disease in Children (CKiD) Schwartz formula (Schwartz 2009), expressed in mean change in eGFR of CKiD between start of study (baseline assessment) and Month 24. | Full analysis set | Posted | Mean | Standard Deviation | mL/min/1.73m2 | Baseline, Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Growth Development - Height at Baseline and Month 12 | Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (<=5, >5-25, >25-50, >50-75, >75-95 and >95% percentile). | Safety Analysis Set | Posted | Number | Percentages | Baseline, Month 12 |
| ||||||||||||||||||||||||||||
| Secondary | Growth Development - Weight at Baseline and Month 12 | Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (<=5, >5-25, >25-50, >50-75, >75-95 and >95% percentile). | Safety Analysis Set | Posted | Number | Percentages | Baseline, Month 12 |
| ||||||||||||||||||||||||||||
| Secondary | Growth Development - Weight at Baseline and Month 24 | Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (<=5, >5-25, >25-50, >50-75, >75-95 and >95% percentile). | Safety Analysis Set | Posted | Number | Percentages | Baseline, Month 24 |
| ||||||||||||||||||||||||||||
| Secondary | Growth Development - Height at Baseline and Month 24 | Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (<=5, >5-25, >25-50, >50-75, >75-95 and >95% percentile). | Safety Analysis Set | Posted | Number | Percentages | Baseline, Month 24 |
|
Day 1 to month 24.
The incidence of AEs (including infections), by primary preferred terms sorted by system organ class were collected on Day 1 and last day with study medication plus 7 days.
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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 | All Patients | All patients | 43 | 56 | 51 | 56 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | 19.0 | Systematic Assessment |
| |
| Haemolytic uraemic syndrome | Blood and lymphatic system disorders | 19.0 | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | 19.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | 19.0 | Systematic Assessment |
| |
| Splenic lesion | Blood and lymphatic system disorders | 19.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | 19.0 | Systematic Assessment |
| |
| Abdominal adhesions | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Aphthous ulcer | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Enterocolitis haemorrhagic | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Internal hernia | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Intestinal haemorrhage | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Oesophagitis | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Chest pain | General disorders | 19.0 | Systematic Assessment |
| |
| General physical health deterioration | General disorders | 19.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | 19.0 | Systematic Assessment |
| |
| Bile duct stenosis | Hepatobiliary disorders | 19.0 | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | 19.0 | Systematic Assessment |
| |
| Hepatic function abnormal | Hepatobiliary disorders | 19.0 | Systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | 19.0 | Systematic Assessment |
| |
| Hepatic vein occlusion | Hepatobiliary disorders | 19.0 | Systematic Assessment |
| |
| Hepatocellular injury | Hepatobiliary disorders | 19.0 | Systematic Assessment |
| |
| Portal fibrosis | Hepatobiliary disorders | 19.0 | Systematic Assessment |
| |
| Food allergy | Immune system disorders | 19.0 | Systematic Assessment |
| |
| Transplant rejection | Immune system disorders | 19.0 | Systematic Assessment |
| |
| Adenoviral upper respiratory infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Alpha haemolytic streptococcal infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Bacterial pyelonephritis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Catheter site infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Cholangitis infective | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Cytomegalovirus viraemia | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Diarrhoea infectious | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Enteritis infectious | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Enterovirus infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Epstein-Barr viraemia | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Epstein-Barr virus infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Escherichia infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Gastroenteritis adenovirus | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Gastroenteritis rotavirus | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Gastroenteritis sapovirus | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Mastoiditis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Oral viral infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Paronychia | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Pneumonia respiratory syncytial viral | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Puncture site infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Rotavirus infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Scarlet fever | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Varicella zoster virus infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Wound infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Biliary anastomosis complication | Injury, poisoning and procedural complications | 19.0 | Systematic Assessment |
| |
| Complications of transplanted liver | Injury, poisoning and procedural complications | 19.0 | Systematic Assessment |
| |
| Fibula fracture | Injury, poisoning and procedural complications | 19.0 | Systematic Assessment |
| |
| Incisional hernia | Injury, poisoning and procedural complications | 19.0 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | 19.0 | Systematic Assessment |
| |
| Transplant failure | Injury, poisoning and procedural complications | 19.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | 19.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | 19.0 | Systematic Assessment |
| |
| Body temperature increased | Investigations | 19.0 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | 19.0 | Systematic Assessment |
| |
| Drug level increased | Investigations | 19.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | 19.0 | Systematic Assessment |
| |
| Liver function test increased | Investigations | 19.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | 19.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | 19.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | 19.0 | Systematic Assessment |
| |
| Hypovolaemia | Metabolism and nutrition disorders | 19.0 | Systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | 19.0 | Systematic Assessment |
| |
| Post transplant lymphoproliferative disorder | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 19.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | 19.0 | Systematic Assessment |
| |
| Food aversion | Psychiatric disorders | 19.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | 19.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | 19.0 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | 19.0 | Systematic Assessment |
| |
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | 19.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | 19.0 | Systematic Assessment |
| |
| Lung infiltration | Respiratory, thoracic and mediastinal disorders | 19.0 | Systematic Assessment |
| |
| Organising pneumonia | Respiratory, thoracic and mediastinal disorders | 19.0 | Systematic Assessment |
| |
| Stridor | Respiratory, thoracic and mediastinal disorders | 19.0 | Systematic Assessment |
| |
| Angioedema | Skin and subcutaneous tissue disorders | 19.0 | Systematic Assessment |
| |
| Dermal cyst | Skin and subcutaneous tissue disorders | 19.0 | Systematic Assessment |
| |
| Swelling face | Skin and subcutaneous tissue disorders | 19.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | 19.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | 19.0 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | 19.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | 19.0 | Systematic Assessment |
| |
| Fatigue | General disorders | 19.0 | Systematic Assessment |
| |
| Pain | General disorders | 19.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | 19.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Cytomegalovirus viraemia | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Diarrhoea infectious | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Epstein-Barr viraemia | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Epstein-Barr virus infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Eye infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | 19.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | 19.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | 19.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | 19.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | 19.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | 19.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | 19.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 19.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | 19.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | 19.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 19.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | 19.0 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | 19.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | 19.0 | Systematic Assessment |
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| Hypertension | Vascular disorders | 19.0 | Systematic Assessment |
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Based on DMC recommendation, enrolment was terminated and patients below 7 years were converted form study medication to local standard of care but patients were followed up until Month 24.
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 |
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| OG004 | >75% - 95% Percentile | Growth percentile category |
| OG005 | >95% Percentile | Growth percentile category |
| OG006 | Total | The classified change from baseline in growth percentiles cross-tabulated against baseline categories of growth percentiles. |
|
|
| OG004 | >75% - 95% Percentile | Growth percentile category |
| OG005 | >95% Percentile | Growth percentile category |
| OG006 | Total | The classified change from baseline in growth percentiles cross-tabulated against baseline categories of growth percentiles. |
|
|
| OG004 | >75% - 95% Percentile | Growth percentile category |
| OG005 | >95% Percentile | Growth percentile category |
| OG006 | Total | The classified change from baseline in growth percentiles cross-tabulated against baseline categories of growth percentiles. |
|
|
| OG004 | >75% - 95% Percentile | Growth percentile category |
| OG005 | >95% Percentile | Growth percentile category |
| OG006 | Total | The classified change from baseline in growth percentiles cross-tabulated against baseline categories of growth percentiles. |
|
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