Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from another therapeutic agent for Gaucher disease.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Gaucher Disease Participants Treated With VPRIV | Participants with Gaucher disease will be enrolled in this survey, who are in VPRIV treatment-naïve therapy or have been switched from another therapeutic agent for Gaucher disease. |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. | Baseline up to end of the study (8 years) |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration | Number of participants of efficacy categories in assessment of change from baseline in hemoglobin concentration during the study was reported. The efficacy categories were classified as Good: increased by at least 1.5g/dL, Moderate response: increase of at least 0.5 g/dL and less than 1.5 g/dL, and Ineffective: increase less than 0.5 g/dL. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
-
Not provided
Not provided
Not provided
Not provided
Participants with confirmed Gaucher disease (types 1, 2, or 3) irrespective of any age or gender who are either naïve to treatment or participants that have been treated with another therapeutic agent for Gaucher disease.
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Konan | Kōnan | Aichi-ken | 483-8704 | Japan | ||
| Nagoya |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34863216 | Derived | Sagara R, Ishigaki M, Otsuka M, Murayama K, Ida H, Fernandez J. Long-term safety and effectiveness of velaglucerase alfa in Gaucher disease: 6-year interim analysis of a post-marketing surveillance in Japan. Orphanet J Rare Dis. 2021 Dec 4;16(1):502. doi: 10.1186/s13023-021-02119-2. |
| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
Not provided
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Not provided
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Participants with Gaucher disease who received Recombinant Velaglucerase Alfa were enrolled. Participants received VPRIV intravenous infusion as part of a routine medical care.
Participants took part in the survey at 45 investigative sites in Japan, from 2 Sep 2014 to 14 May 2024.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Recombinant Velaglucerase Alfa Intravenous Infusion | Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Recombinant Velaglucerase Alfa Intravenous Infusion | Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. | Safety Analysis Set, The safety analysis set was defined as all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. | Posted | Count of Participants | Participants | Baseline up to end of the study (8 years) |
|
Baseline up to end of the study (8 years)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Recombinant Velaglucerase Alfa Intravenous Infusion | Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchitis | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchitis | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 15, 2023 | Nov 11, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 8, 2024 | Nov 11, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
Not provided
Not provided
Not provided
Not provided
Not provided
| Baseline, Every 12 weeks up to 8 years |
| Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts | Number of participants of efficacy categories in assessment of change from baseline in platelet counts during the study was reported. The efficacy categories were classified as Good: increase of at least 30 × 10^9/L, Moderate response: increase of at least 15 × 10^9/L and less than 30 × 10^9/L, and Ineffective: less than 15 × 10^9/L. | Baseline, Every 12 weeks up to 8 years |
| Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes | Number of participants of efficacy categories in assessment of change from baseline in liver volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction. | Baseline, Every 24 weeks up to 8 years |
| Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes | Number of participants of efficacy categories in assessment of change from baseline in spleen volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction. | Baseline, Every 24 weeks up to 8 years |
| Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection | T-scores were the number of standard deviations (SDs) above or below the average for a young adult at peak BMD. A T-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD T-scores and femur neck BMD T-scores related to bone density at baseline and the last data collection. | Baseline, at the time of last data collection (up to 8 years) |
| Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection | Z-scores express the BMD as the number of SDs above or below the average BMD of a healthy participant of the same age and gender. A Z-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD Z-scores and femur neck BMD Z-scores related to bone density at baseline and the last data collection. | Baseline, at the time of last data collection (up to 8 years) |
| Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection | The reported data in this outcome measure was lumbar spine BMD and femur neck BMD related to bone density at baseline and the last data collection. | Baseline, at the time of last data collection (up to 8 years) |
| Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results | Number of participants with positive anti-velaglucerase alfa antibody test results was reported. | Baseline up to end of the study (8 years) |
| Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. Adverse drug reaction refers to AE related to administered drug. | Baseline up to end of the study (8 years) |
| Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Adverse drug reaction refers to AE related to administered drug. Infusion reactions were defined as reactions occurring up to 24 hours after the start of the infusion. | Baseline up to end of the study (8 years) |
| Nagoya |
| Aichi-ken |
| 453-0801 |
| Japan |
| Kitakyushu | Kitakyushu | Fukuoka | 802-0803 | Japan |
| Kurume | Kurume | Fukuoka | 830-0011 | Japan |
| Fukuyama | Fukuyama | Hiroshima | 721-0927 | Japan |
| Higashihiroshima | Higashihiroshima | Hiroshima | 739-0041 | Japan |
| Obihiro | Obihiro | Hokkaido | 080-0016 | Japan |
| Sagamihara | Sagamihara | Kanagawa | 252-0375 | Japan |
| Sendai | Sendai | Miyagi | 980-8574 | Japan |
| Suita | Suita | Osaka | 565-0871 | Japan |
| Tondabayashi | Tondabayashi | Osaka | 584-0000 | Japan |
| Kawagoe | Kawagoe | Saitama | 350-8550 | Japan |
| Tokorozawa | Tokorozawa | Saitama | 359-8513 | Japan |
| Moriyama | Moriyama | Shiga | 524-0022 | Japan |
| Otsu | Ōtsu | Shiga | 520-2192 | Japan |
| Matsue | Matsue | Shimane | 690-0864 | Japan |
| Hamamatsu | Hamamatsu | Shizuoka | 431-3125 | Japan |
| Hamamatsu | Hamamatsu | Shizuoka | 434-8511 | Japan |
| Iwata | Iwata | Shizuoka | 438-0002 | Japan |
| Minato | Minato | Tokyo | 105-8471 | Japan |
| Sumida-ku | Sumida-ku | Tokyo | 130-0005 | Japan |
| Yonago | Yonago | Tottori | 683-8504 | Japan |
| Chiba | Chiba | 266-0007 | Japan |
| Gifu | Gifu | 500-8212 | Japan |
| Hiroshima | Hiroshima | 730-0046 | Japan |
| Hiroshima | Hiroshima | 734-0037 | Japan |
| Kagoshima | Kagoshima | 892-0853 | Japan |
| Kumamoto | Kumamoto | 860-8556 | Japan |
| Kyoto | Kyoto | 606-8507 | Japan |
| Okayama | Okayama | 701-1192 | Japan |
| Osaka | Osaka | 534-0021 | Japan |
| Osaka | Osaka | 545-8586 | Japan |
| Osaka | Osaka | 553-0003 | Japan |
| Saitama | Saitama | 339-8551 | Japan |
| Shizuoka | Shizuoka | 420-8688 | Japan |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
|
Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care. |
|
|
| Secondary | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration | Number of participants of efficacy categories in assessment of change from baseline in hemoglobin concentration during the study was reported. The efficacy categories were classified as Good: increased by at least 1.5g/dL, Moderate response: increase of at least 0.5 g/dL and less than 1.5 g/dL, and Ineffective: increase less than 0.5 g/dL. | Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study. | Posted | Count of Participants | Participants | Baseline, Every 12 weeks up to 8 years |
|
|
|
| Secondary | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts | Number of participants of efficacy categories in assessment of change from baseline in platelet counts during the study was reported. The efficacy categories were classified as Good: increase of at least 30 × 10^9/L, Moderate response: increase of at least 15 × 10^9/L and less than 30 × 10^9/L, and Ineffective: less than 15 × 10^9/L. | Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study. | Posted | Count of Participants | Participants | Baseline, Every 12 weeks up to 8 years |
|
|
|
| Secondary | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes | Number of participants of efficacy categories in assessment of change from baseline in liver volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction. | Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study. | Posted | Count of Participants | Participants | Baseline, Every 24 weeks up to 8 years |
|
|
|
| Secondary | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes | Number of participants of efficacy categories in assessment of change from baseline in spleen volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction. | Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study. | Posted | Count of Participants | Participants | Baseline, Every 24 weeks up to 8 years |
|
|
|
| Secondary | Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection | T-scores were the number of standard deviations (SDs) above or below the average for a young adult at peak BMD. A T-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD T-scores and femur neck BMD T-scores related to bone density at baseline and the last data collection. | Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study. | Posted | Mean | Standard Deviation | T-score | Baseline, at the time of last data collection (up to 8 years) |
|
|
|
| Secondary | Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection | Z-scores express the BMD as the number of SDs above or below the average BMD of a healthy participant of the same age and gender. A Z-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD Z-scores and femur neck BMD Z-scores related to bone density at baseline and the last data collection. | Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study. | Posted | Mean | Standard Deviation | Z-score | Baseline, at the time of last data collection (up to 8 years) |
|
|
|
| Secondary | Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection | The reported data in this outcome measure was lumbar spine BMD and femur neck BMD related to bone density at baseline and the last data collection. | Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study. | Posted | Mean | Standard Deviation | gram per square centimeter | Baseline, at the time of last data collection (up to 8 years) |
|
|
|
| Secondary | Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results | Number of participants with positive anti-velaglucerase alfa antibody test results was reported. | Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study. | Posted | Count of Participants | Participants | Baseline up to end of the study (8 years) |
|
|
|
| Secondary | Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. Adverse drug reaction refers to AE related to administered drug. | Safety Analysis Set, The safety analysis set was defined as all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. | Posted | Count of Participants | Participants | Baseline up to end of the study (8 years) |
|
|
|
| Secondary | Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Adverse drug reaction refers to AE related to administered drug. Infusion reactions were defined as reactions occurring up to 24 hours after the start of the infusion. | Safety Analysis Set, The safety analysis set was defined as all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. | Posted | Count of Participants | Participants | Baseline up to end of the study (8 years) |
|
|
|
| 14 |
| 60 |
| 27 |
| 60 |
| 31 |
| 60 |
| Cellulitis | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Peritonitis | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Pneumonia aspiration | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Pyelonephritis | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Sepsis | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Staphylococcal sepsis | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Device related sepsis | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Acute myeloid leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA/J v26.0 | Systematic Assessment |
|
| Myelodysplastic syndrome | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA/J v26.0 | Systematic Assessment |
|
| Autoimmune neutropenia | Blood and lymphatic system disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Eating disorder | Psychiatric disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Tension | Psychiatric disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Bipolar disorder | Psychiatric disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Myoclonus | Nervous system disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Seizure | Nervous system disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Myoclonic epilepsy | Nervous system disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Arrhythmia | Cardiac disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Cardiac arrest | Cardiac disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Cardiac failure congestive | Cardiac disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Cardio-respiratory arrest | Cardiac disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Ventricular fibrillation | Cardiac disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Asphyxia | Respiratory, thoracic and mediastinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Central sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Ascites | Gastrointestinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Colitis ischaemic | Gastrointestinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Duodenal ulcer | Gastrointestinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Gastroenteritis eosinophilic | Gastrointestinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Bile duct stone | Hepatobiliary disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Cholelithiasis | Hepatobiliary disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Renal failure | Renal and urinary disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Vesicoureteric reflux | Renal and urinary disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Renal impairment | Renal and urinary disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Acute kidney injury | Renal and urinary disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Death | General disorders | MedDRA/J v26.0 | Systematic Assessment | The reasons of events are not determined because assessment findings were insufficient to specify the reason. |
|
| Pyrexia | General disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| General physical health deterioration | General disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Multiple organ dysfunction syndrome | General disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Head injury | Injury, poisoning and procedural complications | MedDRA/J v26.0 | Systematic Assessment |
|
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA/J v26.0 | Systematic Assessment |
|
| Foreign body in throat | Injury, poisoning and procedural complications | MedDRA/J v26.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA/J v26.0 | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Zinc deficiency | Metabolism and nutrition disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Myoclonus | Nervous system disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA/J v26.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA/J v26.0 | Systematic Assessment |
|
Not provided
Not provided
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
| Femur Neck BMD T-scores: Baseline |
|
| Femur Neck BMD T-scores: At the last data collection |
|
|
| Femur Neck BMD Z-scores: Baseline |
|
|
| Femur Neck BMD Z-scores: At the last data collection |
|
|
|
| Femur Neck BMD: Baseline |
|
|
| Femur Neck BMD: At the last data collection |
|
|