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| ID | Type | Description | Link |
|---|---|---|---|
| AGLU03707 | Other Identifier | Genzyme Corporation | |
| 2015-000583-34 | EudraCT Number |
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Study was conducted to fulfill a post marketing commitment (PMC 6). FDA acknowledged fulfillment of PMC.
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An exploratory, open-labeled study of participants with Pompe disease, who had previously received Myozyme® (alglucosidase alfa) treatment, to evaluate the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction (ITI) regimens in combination with Myozyme®. Eligible participants who were then receiving Myozyme® therapy were enrolled into the study, and were followed for a minimum of 18 months on-study (a 6-month ITI treatment module and a 12-month follow-up module on Myozyme® alone). Eligible participants were followed for a minimum of 18 months on treatment or, if a participant was <6 months of age at the time of enrollment, until the participant was 2 years of age. Both cross-reacting immunologic material (CRIM)-negative and CRIM-positive participants were eligible for Regimen A depending if they met the required criteria. Regimen B, however, was limited to CRIM-negative participants.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Regimen A: Alglucosidase alfa and Cyclophosphamide | Experimental | Participants exhibiting clinical decline since starting alglucosidase alfa (Myozyme®) therapy and having inhibitory antibodies and/or a sustained high recombinant human acid alpha-glucosidase (rhGAA) antibody titer (defined as at least 2 titers greater than or equal to [>=] 25,600 obtained at least 1 month apart), regardless of their CRIM status, were assigned to Regimen A. In Regimen A, participants received alglucosidase alfa (Myozyme®) Intravenous (IV) infusion of 20 milligram per kilogram (mg/kg) every other week (qow) for a minimum of 18 months or, until the participant reached the age of 2 years (if the participant was less than [<6] months of age at the time of enrollment). In addition, cyclophosphamide 250 milligram per square meter (mg/m^2) IV infusion was administered every 4 weeks (q4w) after Myozyme® infusion for 6 months. |
|
| Regimen B: Alglucosidase alfa, Rituximab and Methotrexate | Experimental | CRIM-negative participants were assigned to Regimen B if they either(1)exhibited clinical decline since starting alglucosidase alfa (Myozyme®)therapy and did not have inhibitory antibodies and/or a sustained rhGAA antibody titer(defined as at least 2 titers >=25,600 obtained at least 1 month apart),or(2) did not exhibit clinical decline since starting alglucosidase alfa(Myozyme®) therapy, regardless of their anti-rhGAA or inhibitory antibody status. Regimen B participants with CRIM-negative status received alglucosidase alfa(Myozyme®) IV infusion of 20 mg/kg qow for a minimum of 18 months or,until participant reached the age of 2 years (if participant was <6 months of age at time of enrollment). In addition,rituximab 375 mg/m^2 IV was administered weekly beginning the day after Myozyme® infusion for 4 weeks(an optional 2nd cycle could be administered at the discretion of the investigator) and biweekly methotrexate 15 mg/m^2 subcutaneous on the day after Myozyme® infusion for 6 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Myozyme® (alglucosidase alfa) | Biological | Myozyme®: IV infusion of 20 mg/kg qow; Cyclophosphamide: 250 mg/m^2 IV q4w after Myozyme infusion for 6 months. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | An Adverse event (AE) was defined as any undesirable physical, psychological, or behavioral effect experienced by participant during his/her participation in an investigational study, in conjunction with the use of the drug or biologic, whether or not product-related. TEAEs were defined as AEs that occurred or worsened during the on-treatment period (time from the start of investigational medicinal product [IMP] administration up to 18 months). Serious AE (SAE) was any AE that resulted in any of the following outcomes: death, was life-threatening, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity; congenital anomaly; or important medical events that may jeopardize the patient or participant and may require medical or surgical intervention to prevent one of the outcomes listed above; and/or new invasive ventilator use. | From Baseline up to 18 months |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Anti-Recombinant Human Acid Alpha-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibodies at Month 18 | Month 18 | |
| Number of Participants With Recombinant Human Acid Alpha-glucosidase (rhGAA) Inhibitory Antibody at Month 18 | Participants with positive anti-rhGAA IgG antibody were planned to be 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. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Louisville | Kentucky | United States | ||||
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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A total of 4 participants were included and treated in this study. Participants were assigned to either Regimen A or Regimen B.
The study was conducted in 2 countries. A total of 5 participants were screened between 14 December 2008 and 17 August 2010 (dates when first participant and last participant signed informed consent), of which one participant died before enrollment.
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| ID | Title | Description |
|---|---|---|
| FG000 | Regimen A: Alglucosidase Alfa and Cyclophosphamide | Participants exhibiting clinical decline since starting alglucosidase alfa (Myozyme®) therapy and having inhibitory antibodies and/or a sustained high recombinant human acid alpha-glucosidase (rhGAA) antibody titer (defined as at least 2 titers greater than or equal to [>=] 25,600 obtained at least 1 month apart), regardless of their cross-reacting immunologic material (CRIM) status, were assigned to Regimen A. In Regimen A, participants received alglucosidase alfa (Myozyme®) Intravenous (IV) infusion of 20 milligram per kilogram (mg/kg) every other week (qow) for a minimum of 18 months or, until the participant reached the age of 2 years (if the participant was less than [<6] months of age at the time of enrollment). In addition, cyclophosphamide 250 milligram per square meter (mg/m^2) IV infusion was administered every 4 weeks (q4w) after Myozyme® infusion for 6 months. |
| FG001 | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | CRIM-negative participants were assigned to Regimen B if they either(1)exhibited clinical decline since starting alglucosidase alfa (Myozyme®)therapy and did not have inhibitory antibodies and/or a sustained rhGAA antibody titer(defined as at least 2 titers >=25,600 obtained at least 1 month apart),or(2) did not exhibit clinical decline since starting alglucosidase alfa(Myozyme®) therapy, regardless of their anti-rhGAA or inhibitory antibody status. Regimen B participants with CRIM-negative status received alglucosidase alfa(Myozyme®) IV infusion of 20 mg/kg qow for a minimum of 18 months or,until participant reached the age of 2 years (if participant was <6 months of age at time of enrollment). In addition,rituximab 375 mg/m^2 IV was administered weekly beginning the day after Myozyme® infusion for 4 weeks(an optional 2nd cycle could be administered at the discretion of the investigator) and biweekly methotrexate 15 mg/m^2 subcutaneous on the day after Myozyme® infusion for 6 months. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Baseline population is based on full analysis set which included participants who signed informed consent, completed all baseline assessments, and received at least 1 dose of alglucosidase alfa.
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| ID | Title | Description |
|---|---|---|
| BG000 | Regimen A: Alglucosidase Alfa and Cyclophosphamide | Participants exhibiting clinical decline since starting alglucosidase alfa (Myozyme®) therapy and having inhibitory antibodies and/or a sustained high rhGAA antibody titer (defined as at least 2 titers >=25,600 obtained at least 1 month apart), regardless of their CRIM status, were assigned to Regimen A. In Regimen A, participants received alglucosidase alfa (Myozyme®) IV infusion of 20 mg/kg qow for a minimum of 18 months or, until the participant reached the age of 2 years (if the participant was <6 months of age at the time of enrollment). In addition, cyclophosphamide 250 mg/m^2 IV infusion was administered q4w after Myozyme® infusion for 6 months. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of 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 | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | An Adverse event (AE) was defined as any undesirable physical, psychological, or behavioral effect experienced by participant during his/her participation in an investigational study, in conjunction with the use of the drug or biologic, whether or not product-related. TEAEs were defined as AEs that occurred or worsened during the on-treatment period (time from the start of investigational medicinal product [IMP] administration up to 18 months). Serious AE (SAE) was any AE that resulted in any of the following outcomes: death, was life-threatening, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity; congenital anomaly; or important medical events that may jeopardize the patient or participant and may require medical or surgical intervention to prevent one of the outcomes listed above; and/or new invasive ventilator use. | Analysis was performed on safety set population that included participants who received at least 1 dose of alglucosidase alfa in the study. | Posted | Count of Participants | Participants | From Baseline up to 18 months |
All Adverse Events (AEs) were collected from first IMP administration up to 18 months
Reported AEs and deaths are treatment-emergent adverse events i.e. AEs that developed/worsened and deaths that occurred during the 'on treatment period' (time from first IMP administration until 18 months). Analysis was performed on safety population.
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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 | Regimen A: Alglucosidase Alfa and Cyclophosphamide | Participants exhibiting clinical decline since starting alglucosidase alfa (Myozyme®) therapy and having inhibitory antibodies and/or a sustained high rhGAA antibody titer (defined as at least 2 titers >=25,600 obtained at least 1 month apart), regardless of their CRIM status, were assigned to Regimen A. In Regimen A, participants received alglucosidase alfa (Myozyme®) IV infusion of 20 mg/kg qow for a minimum of 18 months or, until the participant reached the age of 2 years (if the participant was <6 months of age at the time of enrollment). In addition, cyclophosphamide 250 mg/m^2 IV infusion was administered q4w after Myozyme® infusion for 6 months. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Leukocytosis | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
Due to low number of enrollment and exploratory nature of outcome measure, only safety data were summarized and reported.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi | 800-633-1610 | 1# | Contact-US@sanofi.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 1, 2009 | Aug 27, 2020 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 23, 2012 | Aug 27, 2020 | SAP_001.pdf |
| 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 |
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| ID | Term |
|---|---|
| C509951 | GAA protein, human |
Not provided
Not provided
Not provided
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Not provided
|
| Myozyme® (alglucosidase alfa) | Biological | Myozyme®: IV infusion of 20 mg/kg qow; Rituximab: 375 mg/m^2 IV weekly beginning the day after Myozyme infusion for 4 weeks (an optional additional 2nd cycle could be administered at the discretion of the investigator); Methotrexate: 15 mg/m^2 subcutaneous qow on the day after Myozyme infusion for 6 months. |
|
|
| Month 18 |
| Overall Survival (OS) | OS was defined as the time interval from the date of first IMP administration to the date of death due to any cause. | From randomization until death or study cut-off whichever comes earlier (up to 18 months) |
| Number of Participants With Ventilator Use | From Baseline up to 18 months |
| Left Ventricular Mass (LVM) Z-Score and LVM Index | LVM Z-score and LVM index were assessed by echocardiograms (ECHOs). LVM Z-Score is an indicator of degree of standard deviations from the mean in a normal distribution. The normal range for LVM Z-Score is -2 to 2. Values <-2 or >2 indicate abnormal LVM Z-Score. Values less than 0 (negative values) indicate a smaller LVM than mean and values higher than 0 indicate a larger LVM than the mean. 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 square meter (g/m^2) were considered as normal and LVM index values >=65 g/m^2 were considered as abnormal. | From Baseline up to 18 months |
| Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88) Score | 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; and walking, running and jumping. Each item is scored on a 4-point Likert scale (0=cannot do; 1=initiates [<10 percent (%) 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. | From Baseline up to 18 months |
| Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS) Score | 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 are scored as "observed" or "not observed". Items in observed range create a motor window. When scoring, subscale scores are calculated by giving child credit (1 point) for observed items within motor window in addition to being given credit (1 point) for all of the less mature items before motor window. AIMS total score is calculated by summing scores for 58 items and ranges from 0-58, with lower score indicating less mature motor development and higher score indicating more mature motor development. | From Baseline up to 18 months |
| Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Score | Pompe PEDI is a disease specific version of PEDI which assesses functional capabilities and performance in children with Pompe disease from 2 months through adolescence.It consists of all items of original PEDI (197 functional skill items in 3 domains: self-care; mobility; and social function) and additional items in functional skills, mobility, and self-care domains to reflect clinically relevant functional skills. Each domain consists of 2 subdomains: functional skill performance and caregiver assistance scale. Norm-based scoring was developed for these additional items, and scoring algorithms for PEDI have been adjusted to reflect additional normative data collected for Pompe PEDI. Total score range for each domain (mean of subdomains) and subdomains ranges from 0-100, higher score indicates higher capability. | From Baseline up to 18 months |
| Durham |
| North Carolina |
| United States |
| Salt Lake City | Utah | United States |
| Norfolk | Virginia | United States |
| Haifa | Israel |
| BG001 | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | CRIM-negative participants were assigned to Regimen B if they either(1)exhibited clinical decline since starting alglucosidase alfa (Myozyme®)therapy and did not have inhibitory antibodies and/or a sustained rhGAA antibody titer(defined as at least 2 titers >=25,600 obtained at least 1 month apart),or(2) did not exhibit clinical decline since starting alglucosidase alfa(Myozyme®) therapy, regardless of their anti-rhGAA or inhibitory antibody status. Regimen B participants with CRIM-negative status received alglucosidase alfa(Myozyme®) IV infusion of 20 mg/kg qow for a minimum of 18 months or,until participant reached the age of 2 years (if participant was <6 months of age at time of enrollment). In addition,rituximab 375 mg/m^2 IV was administered weekly beginning the day after Myozyme® infusion for 4 weeks(an optional 2nd cycle could be administered at the discretion of the investigator) and biweekly methotrexate 15 mg/m^2 subcutaneous on the day after Myozyme® infusion for 6 months. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| 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 |
|
| ID |
|---|
| Title |
|---|
| Description |
|---|
| OG000 | Regimen A: Alglucosidase Alfa and Cyclophosphamide | Participants exhibiting clinical decline since starting alglucosidase alfa (Myozyme®) therapy and having inhibitory antibodies and/or a sustained high rhGAA antibody titer (defined as at least 2 titers >=25,600 obtained at least 1 month apart), regardless of their CRIM status, were assigned to Regimen A. In Regimen A, participants received alglucosidase alfa (Myozyme®) IV infusion of 20 mg/kg qow for a minimum of 18 months or, until the participant reached the age of 2 years (if the participant was <6 months of age at the time of enrollment). In addition, cyclophosphamide 250 mg/m^2 IV infusion was administered q4w after Myozyme® infusion for 6 months. |
| OG001 | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | CRIM-negative participants were assigned to Regimen B if they either(1)exhibited clinical decline since starting alglucosidase alfa (Myozyme®)therapy and did not have inhibitory antibodies and/or a sustained rhGAA antibody titer(defined as at least 2 titers >=25,600 obtained at least 1 month apart),or(2) did not exhibit clinical decline since starting alglucosidase alfa(Myozyme®) therapy, regardless of their anti-rhGAA or inhibitory antibody status. Regimen B participants with CRIM-negative status received alglucosidase alfa(Myozyme®) IV infusion of 20 mg/kg qow for a minimum of 18 months or,until participant reached the age of 2 years (if participant was <6 months of age at time of enrollment). In addition,rituximab 375 mg/m^2 IV was administered weekly beginning the day after Myozyme® infusion for 4 weeks(an optional 2nd cycle could be administered at the discretion of the investigator) and biweekly methotrexate 15 mg/m^2 subcutaneous on the day after Myozyme® infusion for 6 months. |
|
|
| Other Pre-specified | Number of Participants With Anti-Recombinant Human Acid Alpha-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibodies at Month 18 | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | Posted | Month 18 |
|
|
| Other Pre-specified | Number of Participants With Recombinant Human Acid Alpha-glucosidase (rhGAA) Inhibitory Antibody at Month 18 | Participants with positive anti-rhGAA IgG antibody were planned to be 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. | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | Posted | Month 18 |
|
|
| Other Pre-specified | Overall Survival (OS) | OS was defined as the time interval from the date of first IMP administration to the date of death due to any cause. | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | Posted | From randomization until death or study cut-off whichever comes earlier (up to 18 months) |
|
|
| Other Pre-specified | Number of Participants With Ventilator Use | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | Posted | From Baseline up to 18 months |
|
|
| Other Pre-specified | Left Ventricular Mass (LVM) Z-Score and LVM Index | LVM Z-score and LVM index were assessed by echocardiograms (ECHOs). LVM Z-Score is an indicator of degree of standard deviations from the mean in a normal distribution. The normal range for LVM Z-Score is -2 to 2. Values <-2 or >2 indicate abnormal LVM Z-Score. Values less than 0 (negative values) indicate a smaller LVM than mean and values higher than 0 indicate a larger LVM than the mean. 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 square meter (g/m^2) were considered as normal and LVM index values >=65 g/m^2 were considered as abnormal. | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | Posted | From Baseline up to 18 months |
|
|
| Other Pre-specified | Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88) Score | 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; and walking, running and jumping. Each item is scored on a 4-point Likert scale (0=cannot do; 1=initiates [<10 percent (%) 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. | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | Posted | From Baseline up to 18 months |
|
|
| Other Pre-specified | Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS) Score | 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 are scored as "observed" or "not observed". Items in observed range create a motor window. When scoring, subscale scores are calculated by giving child credit (1 point) for observed items within motor window in addition to being given credit (1 point) for all of the less mature items before motor window. AIMS total score is calculated by summing scores for 58 items and ranges from 0-58, with lower score indicating less mature motor development and higher score indicating more mature motor development. | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | Posted | From Baseline up to 18 months |
|
|
| Other Pre-specified | Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Score | Pompe PEDI is a disease specific version of PEDI which assesses functional capabilities and performance in children with Pompe disease from 2 months through adolescence.It consists of all items of original PEDI (197 functional skill items in 3 domains: self-care; mobility; and social function) and additional items in functional skills, mobility, and self-care domains to reflect clinically relevant functional skills. Each domain consists of 2 subdomains: functional skill performance and caregiver assistance scale. Norm-based scoring was developed for these additional items, and scoring algorithms for PEDI have been adjusted to reflect additional normative data collected for Pompe PEDI. Total score range for each domain (mean of subdomains) and subdomains ranges from 0-100, higher score indicates higher capability. | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | Posted | From Baseline up to 18 months |
|
|
| 0 |
| 1 |
| 1 |
| 1 |
| 1 |
| 1 |
| EG001 | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | CRIM-negative participants were assigned to Regimen B if they either(1)exhibited clinical decline since starting alglucosidase alfa (Myozyme®)therapy and did not have inhibitory antibodies and/or a sustained rhGAA antibody titer(defined as at least 2 titers >=25,600 obtained at least 1 month apart),or(2) did not exhibit clinical decline since starting alglucosidase alfa(Myozyme®) therapy, regardless of their anti-rhGAA or inhibitory antibody status. Regimen B participants with CRIM-negative status received alglucosidase alfa(Myozyme®) IV infusion of 20 mg/kg qow for a minimum of 18 months or,until participant reached the age of 2 years (if participant was <6 months of age at time of enrollment). In addition,rituximab 375 mg/m^2 IV was administered weekly beginning the day after Myozyme® infusion for 4 weeks(an optional 2nd cycle could be administered at the discretion of the investigator) and biweekly methotrexate 15 mg/m^2 subcutaneous on the day after Myozyme® infusion for 6 months. | 1 | 3 | 3 | 3 | 3 | 3 |
| Atrial Thrombosis | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
|
| Cardiac Hypertrophy | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
|
| Cyanosis | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
|
| Hypertrophic Cardiomyopathy | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| General Physical Health Deterioration | General disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Pneumonia Streptococcal | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Pneumonia Viral | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Blood Pressure Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Oxygen Saturation Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Staphylococcus Test Positive | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
|
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Hypercapnia | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Increased Upper Airway Secretion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Respiratory Distress | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Respiratory Failure | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Cold Sweat | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pallor | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
|
| Bradycardia | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
|
| Ventricular Hypertrophy | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
|
| Conductive Deafness | Ear and labyrinth disorders | MedDRA 15.1 | Systematic Assessment |
|
| Deafness Bilateral | Ear and labyrinth disorders | MedDRA 15.1 | Systematic Assessment |
|
| Mixed Deafness | Ear and labyrinth disorders | MedDRA 15.1 | Systematic Assessment |
|
| Conjunctivitis | Eye disorders | MedDRA 15.1 | Systematic Assessment |
|
| Abdominal Distension | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Faecaloma | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Faeces Hard | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Retching | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Catheter Site Phlebitis | 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 |
|
| Pain | General disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
|
| Thrombosis In Device | General disorders | MedDRA 15.1 | Systematic Assessment |
|
| Bacterial Tracheitis | 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 |
|
| Pharyngitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Staphylococcal Bacteraemia | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Tracheitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Urinary Tract Infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
|
| Arthropod Bite | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
|
| Excoriation | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
|
| Feeding Tube Complication | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
|
| Thermal Burn | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
|
| Tracheal Haemorrhage | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
|
| Alanine Aminotransferase Increased | 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 Immunoglobulin M Increased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Blood Magnesium Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Blood Potassium Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Body Temperature Increased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Heart Rate Increased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Lymphocyte Count Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Lymphocyte Percentage Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Muscle Enzyme Increased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Neutrophil Count Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Neutrophil Percentage Increased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Oxygen Saturation Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Urine Output Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| White Blood Cell Count Decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| White Blood Cells Urine Positive | Investigations | MedDRA 15.1 | Systematic Assessment |
|
| Joint Contracture | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Clonus | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
|
| Hypokinesia | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
|
| Agitation | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
|
| Restlessness | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
|
| Hydronephrosis | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
|
| Oliguria | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
|
| Urinary Retention | Renal and urinary 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 |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Increased Bronchial Secretion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Increased Upper Airway Secretion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pleural Effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pneumonia Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pulmonary Oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Respiratory Distress | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Decubitus Ulcer | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Dermatitis Diaper | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Papule | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Red Man Syndrome | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Skin Discolouration | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Skin Exfoliation | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Skin Irritation | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Skin Swelling | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
|
| Phlebitis | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
|
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
| 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 |