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| ID | Type | Description | Link |
|---|---|---|---|
| MSC12862 | Other Identifier | Sanofi |
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The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset Pompe disease.
The objectives were to assess the efficacy of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa, as assessed by anti-recombinant human acid alpha-glucosidase (anti-rhGAA) antibody titers, and antibodies that inhibit the activity and/or uptake of alglucosidase alfa; to evaluate the clinical benefit as measured by overall survival, ventilator-free survival, left ventricular mass index (LVMI), gross motor function and development, disability index and the incidence of adverse events (AEs), serious adverse events (SAEs), and clinical laboratory abnormalities.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Alglucosidase Alfa | Experimental | Alglucosidase alfa (Myozyme®) 20 milligrams per kilogram (mg/kg) intravenous (IV) infusion every other week (qow) (or optionally 20 mg/kg IV infusion every week [qw]) beginning from Day 0 to a minimum of 18 months or if the patient was less than (<) 6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 milligrams per square meter (mg/m^2) (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Alglucosidase Alfa | Biological | Administered as IV infusion. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibody at End of Study | Serum samples from patients were analyzed for the presence of anti-rhGAA IgG antibodies. End of study (EOS) refers to the last post baseline observation during study period (up to Week 79). | Baseline, End of Study (up to Week 79 or early termination) |
| Number of Patients With Recombinant Human Acid Alfa-glucosidase (rhGAA) Inhibitory Antibody at End of Study | Patients with positive anti-rhGAA IgG antibody were assessed for the presence of inhibitory antibodies (inhibition of enzyme activity and inhibition of enzyme uptake). Enzyme-linked immunosorbent assay (ELISA) was used to measure inhibition of rhGAA enzymatic activity in vitro and a cell-based assay was used to measure the inhibition of the uptake of rhGAA in normal fibroblast cells by flow cytometry. | End of study (up to Week 79) |
| Number of Patients Who Survived at End of Study | Baseline up to End of study (Week 79) | |
| Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index at End of Study | LVM Z-score and LVM index were assessed by ECHO. LVM Z-Score provides an indicator of degree of standard deviations from the mean in a normal distribution. Negative values indicate a smaller LVM than mean and values higher than 0 indicate a larger LVM than the mean. The normal range for LVM Z-Score is -2 to 2. Values <-2 or >2 indicate abnormal LVM Z-Score. LVM index is an index value derived by normalizing LVM by body surface area. LVM index provides evidence of cardiomyopathy. LVM index values <65 gram per meter^2 (g/m^2) were considered as normal and LVM index values >=65 g/m^2 were considered as abnormal. End of study refers to the last post baseline observation during study period (up to Week 79). | End of study (up to Week 79 or early termination) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme Coorporation | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kosair Children's Hospital | Louisville | Kentucky | 40202 | United States | ||
| Duke University Medical Center |
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The study was conducted at 2 centers in the United States of America between October 01, 2009 and March 27, 2013.
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| ID | Title | Description |
|---|---|---|
| FG000 | Alglucosidase Alfa | Alglucosidase alfa (Myozyme®) 20 milligrams per kilogram (mg/kg) intravenous (IV) infusion every other week (qow) (or optionally 20 mg/kg IV infusion every week [qw]) beginning from Day 0 to a minimum of 18 months or if the patient was less than (<) 6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 milligrams per square meter (mg/m^2) (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Methotrexate | Drug | Administered subcutaneously. |
|
| Rituximab | Drug | Administered as IV infusion. |
|
| Number of Patients With Ventilator Use at End of Study |
Number of patients who had ventilator support at end of study was reported. End of study refers to the last post baseline observation during study period (up to Week 79). |
| End of study (up to Week 79 or early termination) |
| Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88) at End of Study | GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item is scored on a 4-point Likert scale (0 = cannot do; 1 = initiates [<10% of the task]; 2 = partially completes [10% to <100% of the task]; 3 = task completion). The score for each dimension is expressed as a percentage of the maximum score for that dimension. Total score is obtained by adding the percentage scores for each dimension and dividing the sum by the total number of dimensions. Total score ranges from 0% to 100%, where higher scores indicate better motor functions. A total score of <7.5% demonstrates gross motor disability. End of study refers to the last post baseline observation during study period (up to Week 79). | End of study (up to Week 79 or early termination) |
| Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS) at End of Study | AIMS is a 58-item reliable and valid measure of motor development for infants at risk for motor delay. It assesses infant movement in 4 positions (subscales): prone (reciprocal crawling); supine (moving hands to feet); sitting (sitting with arm support); and standing (pulls to stand). For each subscale, items were scored as "observed" or "not observed". Item in the observed range create a motor window. When scoring, subscale scores are calculated by giving the child credit (1 point) for observed items within the motor window in addition to being given credit (1 point) for all of the less mature items before motor window. AIMS total score was calculated by summing the scores for 58 items and ranged from 0 to 58, with higher score indicating more mature motor development. Score was then compared with age-equivalent peers from normative sample and equivalence level age (in months) is reported. End of study refers to the last post baseline observation during study period (up to Week 79). | End of study (up to Week 79 or early termination) |
| Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at End of Study | The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. It consists of all items of the original PEDI (197 functional skill items in 3 domains: self-care; mobility; and social function) and additional items in the functional skills, mobility, and self-care domains to reflect clinically relevant functional skills for children with Pompe disease. Each domain consisted of 2 subdomains: functional skill performance and caregiver assistance scale. Norm-based scoring was developed for these additional items, and scoring algorithms for the PEDI have been adjusted to reflect additional normative data collected for the Pompe PEDI. Total score range for each domain (mean of subdomains) and subdomain ranges from 0 to 100, where higher score indicates high capability. End of study refers to the last post baseline observation during study period (up to Week 79). | End of study (up to Week 79 or early termination) |
| Durham |
| North Carolina |
| 27710 |
| United States |
| Full Analysis Set (FAS) |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety population included all patients who received at least one dose of alglucosidase alfa.
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| ID | Title | Description |
|---|---|---|
| BG000 | Alglucosidase Alfa | Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week [qw]) beginning from Day 0 to a minimum of 18 months or if the patient was <6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Number | participants |
| |||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
| |||||||||||||||||||||||
| Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G | As all patients were treatment naïve, it was expected that no patient would be Anti-rhGAA immunoglobulin G (IgG) antibody positive at baseline. | Number | participants |
| ||||||||||||||||||||||
| Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index | LVM Z-Score and LVM Index were assessed by echocardiography (ECHO). LVM Z-Score: an indicator of degree of standard deviations from mean in a normal distribution. Negative values indicate a smaller LVM than mean and values higher than 0 indicate a larger LVM than mean. Normal range is -2 to 2; values <-2 or >2 indicate abnormal score. LVM index: an index value derived by normalizing LVM by body surface area. LVM index provides evidence of cardiomyopathy. LVM index values <65 gram per square meter (g/m^2) were considered as normal and LVM index values >=65 g/m^2 were considered as abnormal. | Number | participants |
| ||||||||||||||||||||||
| Number of Patients With Ventilator Use | Number | participants |
| |||||||||||||||||||||||
| Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88) | GMFM-88:an 88-item measure to detect gross motor function; consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item is scored on a 4-point Likert scale(0=cannot do; 1=initiates [<10% of the task]; 2=partially completes [10% to <100% of the task]; 3=task completion). Score for each dimension is expressed as a percentage of maximum score for that dimension. Total score=sum of percentage scores for each dimension divided by number of dimensions. Total score range: 0% to 100%, where higher scores indicate better motor functions. | Median | Full Range | percentage of maximum total score |
| |||||||||||||||||||||
| Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS) | AIMS:58-item in 4 subscales: prone; supine; sitting; and standing. Each item is scored as observed/not observed. Item in observed range create a motor window. Subscale scores are calculated by giving child 1 point for observed items within motor window in addition to being given 1 point for all less mature items before motor window. AIMS total score=sum of scores for 58 items, range: 0-58,higher score=more mature motor development. Score was then compared with age-equivalent peers from normative sample and equivalence level age (in months) is reported. Here, number of patients analyzed = 3. | Median | Full Range | months |
| |||||||||||||||||||||
| Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) | It consists of all items of original PEDI (197 functional skill items in 3 domains:self-care;mobility;social function) and additional items in functional skills,mobility,self-care domains to reflect functional skills for children with Pompe. Each domain consists of 2 subdomains: functional skill performance, caregiver assistance scale. Norm-based scoring was developed for additional items, and scoring for PEDI have been adjusted to reflect additional normative data collected for Pompe PEDI. Total score range for each domain (mean of subdomains) and subdomain=0-100;higher score=high capability. | Median | Full Range | units on a scale |
|
| 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 Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibody at End of Study | Serum samples from patients were analyzed for the presence of anti-rhGAA IgG antibodies. End of study (EOS) refers to the last post baseline observation during study period (up to Week 79). | FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. | Posted | Number | participants | Baseline, End of Study (up to Week 79 or early termination) |
|
|
| |||||||||||||||||||||||||||||||||
| Primary | Number of Patients With Recombinant Human Acid Alfa-glucosidase (rhGAA) Inhibitory Antibody at End of Study | Patients with positive anti-rhGAA IgG antibody were assessed for the presence of inhibitory antibodies (inhibition of enzyme activity and inhibition of enzyme uptake). Enzyme-linked immunosorbent assay (ELISA) was used to measure inhibition of rhGAA enzymatic activity in vitro and a cell-based assay was used to measure the inhibition of the uptake of rhGAA in normal fibroblast cells by flow cytometry. | FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. Here, number of patients analyzed = patients with positive anti-rhGAA IgG antibody. | Posted | Number | participants | End of study (up to Week 79) |
| |||||||||||||||||||||||||||||||||||
| Primary | Number of Patients Who Survived at End of Study | FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. | Posted | Number | participants | Baseline up to End of study (Week 79) |
|
| |||||||||||||||||||||||||||||||||||
| Primary | Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index at End of Study | LVM Z-score and LVM index were assessed by ECHO. LVM Z-Score provides an indicator of degree of standard deviations from the mean in a normal distribution. Negative values indicate a smaller LVM than mean and values higher than 0 indicate a larger LVM than the mean. The normal range for LVM Z-Score is -2 to 2. Values <-2 or >2 indicate abnormal LVM Z-Score. LVM index is an index value derived by normalizing LVM by body surface area. LVM index provides evidence of cardiomyopathy. LVM index values <65 gram per meter^2 (g/m^2) were considered as normal and LVM index values >=65 g/m^2 were considered as abnormal. End of study refers to the last post baseline observation during study period (up to Week 79). | FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. | Posted | Number | participants | End of study (up to Week 79 or early termination) |
| |||||||||||||||||||||||||||||||||||
| Primary | Number of Patients With Ventilator Use at End of Study | Number of patients who had ventilator support at end of study was reported. End of study refers to the last post baseline observation during study period (up to Week 79). | FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. | Posted | Number | participants | End of study (up to Week 79 or early termination) |
|
| ||||||||||||||||||||||||||||||||||
| Primary | Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88) at End of Study | GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item is scored on a 4-point Likert scale (0 = cannot do; 1 = initiates [<10% of the task]; 2 = partially completes [10% to <100% of the task]; 3 = task completion). The score for each dimension is expressed as a percentage of the maximum score for that dimension. Total score is obtained by adding the percentage scores for each dimension and dividing the sum by the total number of dimensions. Total score ranges from 0% to 100%, where higher scores indicate better motor functions. A total score of <7.5% demonstrates gross motor disability. End of study refers to the last post baseline observation during study period (up to Week 79). | FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. Here, numbers of patients analyzed = patients with end of study GMFM-88 assessment. | Posted | Median | Full Range | percentage of maximum total score | End of study (up to Week 79 or early termination) |
| ||||||||||||||||||||||||||||||||||
| Primary | Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS) at End of Study | AIMS is a 58-item reliable and valid measure of motor development for infants at risk for motor delay. It assesses infant movement in 4 positions (subscales): prone (reciprocal crawling); supine (moving hands to feet); sitting (sitting with arm support); and standing (pulls to stand). For each subscale, items were scored as "observed" or "not observed". Item in the observed range create a motor window. When scoring, subscale scores are calculated by giving the child credit (1 point) for observed items within the motor window in addition to being given credit (1 point) for all of the less mature items before motor window. AIMS total score was calculated by summing the scores for 58 items and ranged from 0 to 58, with higher score indicating more mature motor development. Score was then compared with age-equivalent peers from normative sample and equivalence level age (in months) is reported. End of study refers to the last post baseline observation during study period (up to Week 79). | FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. Here, numbers of patients analyzed = patients with end of study AIMS assessment. | Posted | Median | Full Range | months | End of study (up to Week 79 or early termination) |
| ||||||||||||||||||||||||||||||||||
| Primary | Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at End of Study | The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. It consists of all items of the original PEDI (197 functional skill items in 3 domains: self-care; mobility; and social function) and additional items in the functional skills, mobility, and self-care domains to reflect clinically relevant functional skills for children with Pompe disease. Each domain consisted of 2 subdomains: functional skill performance and caregiver assistance scale. Norm-based scoring was developed for these additional items, and scoring algorithms for the PEDI have been adjusted to reflect additional normative data collected for the Pompe PEDI. Total score range for each domain (mean of subdomains) and subdomain ranges from 0 to 100, where higher score indicates high capability. End of study refers to the last post baseline observation during study period (up to Week 79). | FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. Here, number of patient analyzed = number of patients with end of study Pompe PEDI assessment and n = number of patients with end of study assessment of specified category. | Posted | Median | Full Range | units on a scale | End of study (up to Week 79 or early termination) |
|
First dose of study drug up to end of study (up to Week 79)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to 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 | Alglucosidase Alfa | Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week [qw]) beginning from Day 0 to a minimum of 18 months or if the patient was <6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information. | 3 | 4 | 4 | 4 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Arrhythmia | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Cardiopulmonary failure | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Tympanic membrane disorder | Ear and labyrinth disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Disease progression | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Serratia sepsis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Tracheitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Feeding tube complication | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Torus fracture | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Pulse absent | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Fluid imbalance | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Muscle contracture | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Apnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Laryngeal stenosis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Vena cava thrombosis | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Cyanosis | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Extrasystoles | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Tympanic membrane disorder | Ear and labyrinth disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Conjunctivitis | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Eyelid ptosis | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Periorbital oedema | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Anal sphincter atony | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Anorectal disorder | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Duodenogastric reflux | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Retching | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Teething | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Catheter site discharge | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Catheter site erosion | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Catheter site erythema | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Catheter site pruritus | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Catheter site rash | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Catheter site related reaction | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Device dislocation | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Device occlusion | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Face oedema | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Intentional medical device removal by patient | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Medical device complication | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Body tinea | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Candidiasis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Catheter site cellulitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Impetigo | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Serratia infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Tracheitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Urinary tract infection enterococcal | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Burns second degree | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Lip injury | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Medication error | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Overdose | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Procedural site reaction | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Torus fracture | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Antibiotic resistant Staphylococcus test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Band neutrophil percentage increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood chloride decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase MB increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood culture positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood immunoglobulin G increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood iron decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood potassium increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood sodium decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Body temperature increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Clostridium test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Eosinophil percentage increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Heart rate decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Heart rate irregular | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Lymphocyte percentage decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Moraxella test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Neutrophil percentage increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Occult blood positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Protein total decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory rate decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory rate increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Reticulocyte percentage increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Specific gravity urine increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Urinary hexose tetrasaccharide increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Urine output decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Muscle contracture | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Fibroma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.1 | Systematic Assessment |
| |
| Clonus | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Bronchial secretion retention | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Diaphragm muscle weakness | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Diaphragmatic disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Increased bronchial secretion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Laryngeal stenosis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Excessive granulation tissue | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hair colour changes | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Macule | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Palmar-plantar erythrodysaesthesia syndrome | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Papule | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Red man syndrome | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Skin hypopigmentation | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Swelling face | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Gastrointestinal tube removal | Surgical and medical procedures | MedDRA 15.1 | Systematic Assessment |
| |
| Post procedural drainage | Surgical and medical procedures | MedDRA 15.1 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pallor | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Vena cava thrombosis | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
|
Due to the small number of patients assessed in this study the results must be interpreted with caution.
If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi | Contact-us@sanofi.com |
| ID | Term |
|---|---|
| D006009 | Glycogen Storage Disease Type II |
| D006356 | Heartburn |
| ID | Term |
|---|---|
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| 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 |
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D012817 | Signs and Symptoms, Digestive |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C509951 | GAA protein, human |
| D008727 | Methotrexate |
| D000069283 | Rituximab |
| ID | Term |
|---|---|
| D000630 | Aminopterin |
| D011622 | Pterins |
| D011621 | Pteridines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
| D058846 | Antibodies, Monoclonal, Murine-Derived |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
Not provided
Not provided
| Race: White, Black |
|
| Ethnicity: Hispanic |
|
| Ethnicity: Non Hispanic |
|
| Title | Measurements |
|---|---|
|
| LVM index: Normal |
|
| LVM index: Abnormal |
|
|
| Functional Skills: Social Function Score (n=1) |
|
| Caregiver Assistance: Mobility Score (n=2) |
|
| Caregiver Assistance: Self-Care Score (n=2) |
|
| Caregiver Assistance: Social Function Score (n=2) |
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|
|
|
|
|
|
|