Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2011-005595-42 | EudraCT Number |
Not provided
Not provided
Not provided
Study was stopped due to approved label expansion of alglucosidase alfa.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Alglucosidase Alfa 4000 L material (Non-US participants) | Experimental | Alglucosidase alfa 4000 L material for 52 weeks. |
|
| Alglucosidase Alfa 160 L material (US participants) | Active Comparator | Alglucosidase alfa 160 L material for 52 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| alglucosidase alfa | Biological | Intravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Cardiac Function at Week 52 | Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. | Baseline, Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Estimated Probability of Survival | Up to Week 52 | |
| Number of Participants With Invasive Ventilator-Free Survival | Invasive ventilator-free survival was defined as the time during which the participant is alive and not invasively ventilated. Number of Participants with invasive ventilator-free survival were reported. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Little Rock | Arkansas | United States | ||||
A total of 5 participants were screened and 4 participants were treated.
The study was conducted between 21 August 2012 and 1 December 2014.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Alglucosidase Alfa 4000 L Material (Non-US Participants) | Alglucosidase alfa (4000 L material) 20 mg/kg intravenous (IV) infusion every other week (QOW) for 52 weeks. |
| FG001 | Alglucosidase Alfa 160 L Material (US Participants) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| alglucosidase alfa | Biological | IV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW. |
|
|
| Up to Week 52 |
| Change From Baseline in Motor Development Status at Week 52 | Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. 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 was 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 was expressed as a percentage of the maximum score for that dimension. Total score ranges from 0% to 100%, where higher scores indicate better motor functions. | Baseline, Week 52 |
| Oakland |
| California |
| United States |
| Gainsville | Florida | United States |
| Decatur | Georgia | United States |
| Chicago | Illinois | United States |
| Cambridge | Massachusetts | United States |
| Detroit | Michigan | United States |
| Las Vegas | Nevada | United States |
| New Brunswick | New Jersey | United States |
| New York | New York | United States |
| Durham | North Carolina | United States |
| Cincinnati | Ohio | United States |
| Fort Worth | Texas | United States |
| Seattle | Washington | United States |
| Geiben | Germany |
| Mainz | Germany |
| Taipei | Taiwan |
Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks.
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Alglucosidase Alfa 4000 L Material (Non-US Participants) | Alglucosidase alfa (4000 L material) 20 mg/kg IV infusion QOW for 52 weeks. |
| BG001 | Alglucosidase Alfa 160 L Material (US Participants) | Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| 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 Cardiac Function at Week 52 | Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. | Full analysis population defined as all participants who receive at least 1 infusion of alglucosidase alfa. For this endpoint no participants were analyzed in 'Algucosidase Alfa 4000 L material' arm at Baseline and Week 52. One participant from 'Algucosidase Alfa 160 L Material' arm was discontinued from study at Week 31 due to physician's decision | Posted | Mean | Standard Deviation | Z-score | Baseline, Week 52 |
|
|
| ||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Estimated Probability of Survival | Full analysis population. | Posted | Number | percentage of participants | Up to Week 52 |
|
| |||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Invasive Ventilator-Free Survival | Invasive ventilator-free survival was defined as the time during which the participant is alive and not invasively ventilated. Number of Participants with invasive ventilator-free survival were reported. | Full analysis population. | Posted | Number | participants | Up to Week 52 |
|
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Motor Development Status at Week 52 | Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. 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 was 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 was expressed as a percentage of the maximum score for that dimension. Total score ranges from 0% to 100%, where higher scores indicate better motor functions. | Full analysis population. For this endpoint no participants were analyzed in 'Algucosidase Alfa 4000 L material' arm at Baseline and Week 52. One participant from 'Algucosidase Alfa 160 L Material' arm was discontinued from study at Week 31 due to physician's decision. | Posted | Mean | Standard Deviation | percentage of maximum total score | Baseline, Week 52 |
|
All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (Week 52) regardless of seriousness or relationship to investigational product.
Reported adverse events are treatment-emergent adverse events that is AEs that developed/worsened during the 'on treatment period' (the period from the first infusion date to the date that the last data were collected). Analysis was carried out on full analysis population.
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 4000 L Material (Non-US Participants) | Alglucosidase alfa (4000 L material) 20 mg/kg IV infusion QOW for 52 weeks. | 0 | 1 | 1 | 1 | ||
| EG001 | Alglucosidase Alfa 160 L Material (US Participants) | Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks. | 3 | 3 | 3 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiac failure | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Lobar pneumonia | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Hypotonia | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Nodal rhythm | Cardiac disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Middle ear effusion | Ear and labyrinth disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Device occlusion | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Infusion site erythema | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Eye infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Fungal infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA 17.1 | Systematic Assessment |
| |
| Muscle contracture | Musculoskeletal and connective tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Nystagmus | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Bronchial secretion retention | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 17.1 | Systematic Assessment |
|
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 |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C509951 | GAA protein, human |
Not provided
Not provided
Not provided
| Male |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|