Trial · NCT00701701
Immune Tolerance Induction Study
NCT00701701, Phase 4, Terminated, Genzyme, a Sanofi Company; results first posted 2020-10-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Immune Tolerance Induction Study. Trials, record NCT00701701. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00701701
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Immune Tolerance Induction Study
- NCT number
- NCT00701701
- Status
- Terminated
- Phases
- Phase 4
- Enrollment
- 4 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
- Interventions
- Myozyme® (alglucosidase alfa), Myozyme® (alglucosidase alfa)
- First posted
- 2008-06-19
- Last updated
- 2022-04-07
- Other registries' IDs
- eudract:2015-000583-34
- Results first posted
- 2020-10-12
- Limitations and caveats
- Due to low number of enrollment and exploratory nature of outcome measure, only safety data were summarized and reported.
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | From Baseline up to 18 months | 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. |
| Other | Number of Participants With Anti-Recombinant Human Acid Alpha-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibodies at Month 18 | Month 18 | |
| Other | Number of Participants With Recombinant Human Acid Alpha-glucosidase (rhGAA) Inhibitory Antibody at Month 18 | 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. |
| Other | Overall Survival (OS) | From randomization until death or study cut-off whichever comes earlier (up to 18 months) | OS was defined as the time interval from the date of first IMP administration to the date of death due to any cause. |
| Other | Number of Participants With Ventilator Use | From Baseline up to 18 months | |
| Other | Left Ventricular Mass (LVM) Z-Score and LVM Index | From Baseline up to 18 months | 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. |
| Other | Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88) Score | From Baseline up to 18 months | 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. |
| Other | Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS) Score | From Baseline up to 18 months | 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. |
| Other | Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Score | From Baseline up to 18 months | 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. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | From Baseline up to 18 months | Analysis was performed on safety set population that included participants who received at least 1 dose of alglucosidase alfa in the study. | Participants | COUNT_OF_PARTICIPANTS |
| OTHER_PRE_SPECIFIED | Number of Participants With Anti-Recombinant Human Acid Alpha-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibodies at Month 18 | Month 18 | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | ||
| OTHER_PRE_SPECIFIED | Number of Participants With Recombinant Human Acid Alpha-glucosidase (rhGAA) Inhibitory Antibody at Month 18 | Month 18 | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | ||
| OTHER_PRE_SPECIFIED | Overall Survival (OS) | From randomization until death or study cut-off whichever comes earlier (up to 18 months) | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | ||
| OTHER_PRE_SPECIFIED | Number of Participants With Ventilator Use | From Baseline up to 18 months | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | ||
| OTHER_PRE_SPECIFIED | Left Ventricular Mass (LVM) Z-Score and LVM Index | From Baseline up to 18 months | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | ||
| OTHER_PRE_SPECIFIED | Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88) Score | From Baseline up to 18 months | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | ||
| OTHER_PRE_SPECIFIED | Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS) Score | From Baseline up to 18 months | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. | ||
| OTHER_PRE_SPECIFIED | Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Score | From Baseline up to 18 months | Data were not summarized for this exploratory outcome measure due to low number of enrollment of participants. |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Regimen A: Alglucosidase Alfa and Cyclophosphamide | Any TEAE | 1 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | Any TEAE | 3 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Regimen A: Alglucosidase Alfa and Cyclophosphamide | Any Treatment-emergent SAE | 1 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | Any Treatment-emergent SAE | 3 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Regimen A: Alglucosidase Alfa and Cyclophosphamide | Any TEAE leading to withdrawal from study | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | Any TEAE leading to withdrawal from study | 1 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Regimen A: Alglucosidase Alfa and Cyclophosphamide | Any TEAE leading to death | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | Any TEAE leading to death | 1 | Participants |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 1 | |
| Overall Study | STARTED | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 3 | |
| Overall Study | COMPLETED | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 1 | |
| Overall Study | COMPLETED | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 1 | |
| Overall Study | NOT COMPLETED | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 0 | |
| Overall Study | NOT COMPLETED | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 2 |
Source ClinicalTrials.gov
Adverse events242
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Leukocytosis | Blood and lymphatic system disorders | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 0 | 1 | 0 |
| other | Leukocytosis | Blood and lymphatic system disorders | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 1 | 3 | 1 |
| other | Bradycardia | Cardiac disorders | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 1 | 1 | 5 |
| other | Bradycardia | Cardiac disorders | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 0 | 3 | 0 |
| other | Tachycardia | Cardiac disorders | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 0 | 1 | 0 |
| other | Tachycardia | Cardiac disorders | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 1 | 3 | 3 |
| other | Ventricular Hypertrophy | Cardiac disorders | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 0 | 1 | 0 |
| other | Ventricular Hypertrophy | Cardiac disorders | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 1 | 3 | 1 |
| other | Conductive Deafness | Ear and labyrinth disorders | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 0 | 1 | 0 |
| other | Conductive Deafness | Ear and labyrinth disorders | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 1 | 3 | 2 |
1 to 10 of 242Source ClinicalTrials.gov
See all 242 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 0 | 1 |
| Total, all-cause mortality | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 1 | 3 |
| Total, other adverse events | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 1 | 1 |
| Total, other adverse events | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 3 | 3 |
| Total, serious adverse events | Regimen A: Alglucosidase Alfa and Cyclophosphamide | 1 | 1 |
| Total, serious adverse events | Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate | 3 | 3 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Regimen A: Alglucosidase Alfa and Cyclophosphamide vs Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate: deaths from any cause | Trial comparisons |
| Regimen A: Alglucosidase Alfa and Cyclophosphamide vs Regimen B: Alglucosidase Alfa, Rituximab and Methotrexate: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00701701
GET https://conductscience.org/api/trials/v1/NCT00701701/events?page=2