Trial · NCT00455195
Late-Onset Treatment Study Extension Protocol
NCT00455195, Phase 4, Completed, Genzyme, a Sanofi Company; results first posted 2012-01-05
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ConductScience Library. Late-Onset Treatment Study Extension Protocol. Trials, record NCT00455195. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00455195
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Late-Onset Treatment Study Extension Protocol
- NCT number
- NCT00455195
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 81 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)
- Interventions
- alglucosidase alfa
- First posted
- 2007-04-03
- Last updated
- 2014-03-07
- Other registries' IDs
- eudract:2006-003644-31
- Results first posted
- 2012-01-05
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 to 2.5 years | The numbers of participants who experienced Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment with alglucosidase alfa. Participants with long-term exposure to alglucosidase alfa (those from the Alglucosidase Alfa/Alglucosidase Alfa treatment group) are included. Time frames are stated from the start of the double-blind study AGLU02704 (NCT00158600). |
| Primary | Baseline Values (Week 0) of Functional Endurance as Measured by Six-Minute Walk Test (6MWT) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 | Six-Minute Walk Test (6MWT) measures the distance walked (in meters) in 6 minutes. A longer distance indicates greater endurance. Time frames are stated from the start of the double-blind study AGLU02704 (NCT00158600). |
| Primary | Change From Baseline (Week 0) in the Six-Minute Walk Test (6MWT) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0, Week 104 | Six-Minute Walk Test (6MWT) measures the distance walked (in meters) in 6 minutes. A longer distance indicates greater endurance. Time frames are stated from the start of the double-blind study AGLU02704 (NCT00158600). Change is calculated as the value minus the baseline value. |
| Primary | Baseline Values (Week 0) for Percent Predicted Forced Vital Capacity (FVC) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 | Forced vital capacity (FVC) is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%. Time frames are stated from the start of the double-blind study AGLU02704 (NCT00158600). |
| Primary | Change From Baseline (Week 0) in the Percent Predicted Forced Vital Capacity (FVC) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0, Week 104 | Forced vital capacity (FVC) is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%. Time frames are stated from the start of the double-blind study AGLU02704 (NCT00158600). Change is calculated as the value minus the baseline value. |
| Secondary | Baseline Values (Week 0) for Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 | Quantitative muscle testing (QMT) is a standardized system to measure muscle force production during maximal voluntary isometric contraction. QMT data were collected directly from sensors into laptop computers. Predicted normal values for QMT are based on a formula using sex, age and body mass index of a person, and are an estimate of healthy muscle force. Percent of predicted QMT = (observed value)/(predicted value) \* 100%. The QMT Leg Score is the average of the bilateral means for percent predicted knee flexors and extensors. A value of 100% indicates 'normal' muscle strength. |
| Secondary | Change From Baseline (Week 0) for Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0, Week 104 | Quantitative muscle testing (QMT) is a standardized system to measure muscle force production during maximal voluntary isometric contraction. QMT data were collected directly from sensors into laptop computers. Predicted normal values for QMT are based on a formula using sex, age and body mass index of a person, and are an estimate of healthy muscle force. Percent of predicted QMT = (observed value)/(predicted value) \* 100%. The QMT Leg Score is the average of the bilateral means for percent predicted knee flexors and extensors. A value of 100% indicates 'normal' muscle strength. |
| Secondary | Baseline Values (Week 0) for Quality of Life Related to Physical Component Score (PCS) as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 | The Medical Outcomes Study Short Form (MOS SF)-36 questionnaire consists of 36 items grouped into 8 domains designed to assess generic health-related quality of life in healthy and ill adult populations. Physical Component Score reports the four domains of physical functioning, role-physical, bodily pain, and general health and are standardized as Z-scores (scale of 0-100). Higher scores are associated with better quality of life. Time frames are stated from the start of the double-blind study AGLU02704 (NCT00158600). |
| Secondary | Change From Baseline in Quality of Life Related to Physical Component Score (PCS) as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 , Week 104 | The Medical Outcomes Study Short Form (MOS SF)-36 questionnaire consists of 36 items grouped into 8 domains designed to assess generic health-related quality of life in healthy and ill adult populations. Physical Component Score reports the four domains of physical functioning, role-physical, bodily pain, and general health and is standardized as Z-scores (scale of 0-100). Higher scores are associated with better quality of life. Change is calculated as the value minus the baseline value. Time frames are stated from the start of the double-blind study AGLU02704 (NCT00158600). |
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 | Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 to 2.5 years | The safety population includes all participants randomized to alglucosidase alfa treatment in AGLU02704 (NCT00158600) who received at least one infusion of alglucosidase alfa. | participants | NUMBER |
| SECONDARY | Baseline Values (Week 0) for Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 | Intent-to-treat population | percent of predicted QMT | MEAN |
| PRIMARY | Baseline Values (Week 0) of Functional Endurance as Measured by Six-Minute Walk Test (6MWT) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 | Intent-to-treat population. | meters | MEAN |
| PRIMARY | Change From Baseline (Week 0) in the Six-Minute Walk Test (6MWT) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0, Week 104 | Intent-to-treat population of participants who had both baseline (Week 0) and Week 104 data. | meters | MEAN |
| SECONDARY | Change From Baseline (Week 0) for Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0, Week 104 | Intent-to-treat population of participants who had both baseline (Week 0) and Week 104 data. | percent of predicted QMT | MEAN |
| PRIMARY | Baseline Values (Week 0) for Percent Predicted Forced Vital Capacity (FVC) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 | Intent-to-treat population | percent of predicted FVC | MEAN |
| PRIMARY | Change From Baseline (Week 0) in the Percent Predicted Forced Vital Capacity (FVC) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0, Week 104 | Intent-to-treat population of participants who had both baseline (Week 0) and Week 104 data. | percent of predicted FVC | MEAN |
| SECONDARY | Baseline Values (Week 0) for Quality of Life Related to Physical Component Score (PCS) as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 | Intent-to-treat population of participants with valid baseline (Week 0) PCS surveys. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Quality of Life Related to Physical Component Score (PCS) as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Week 0 , Week 104 | Intent-to-treat population of participants who had valid baseline (Week 0) and Week 104 data. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group15
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | Participants with any AEs | 60 | participants | |
| Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | Participants with Treatment-Related AEs | 37 | participants | |
| Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | Participants with Infusion-Associated Reactions | 21 | participants | |
| Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | Participants with Serious AEs | 15 | participants | |
| Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | Participants with Severe AEs | 16 | participants | |
| Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | Participants- Discontinued Due to AEs (incl death) | 3 | participants | |
| Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | Participants Who Died | 1 | participants | |
Baseline Values (Week 0) for Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | 37.7 | Standard Deviation 18.88 | percent of predicted QMT | |
| Baseline Values (Week 0) of Functional Endurance as Measured by Six-Minute Walk Test (6MWT) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | 332.2 | Standard Deviation 126.69 | meters | |
| Change From Baseline (Week 0) in the Six-Minute Walk Test (6MWT) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) | Alglucosidase Alfa/Alglucosidase Alfa | 21.3 | Standard Deviation 78.02 | meters |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-blind Study AGLU02704 | STARTED | Alglucosidase Alfa/Alglucosidase Alfa | 60 | |
| Double-blind Study AGLU02704 | STARTED | Placebo/Alglucosidase Alfa | 30 | |
| Double-blind Study AGLU02704 | COMPLETED | Alglucosidase Alfa/Alglucosidase Alfa | 55 | |
| Double-blind Study AGLU02704 | COMPLETED | Placebo/Alglucosidase Alfa | 26 | |
| Double-blind Study AGLU02704 | NOT COMPLETED | Alglucosidase Alfa/Alglucosidase Alfa | 5 | |
| Double-blind Study AGLU02704 | NOT COMPLETED | Placebo/Alglucosidase Alfa | 4 | |
| Extension Study AGLU03206 | STARTED | Alglucosidase Alfa/Alglucosidase Alfa | 55 | |
| Extension Study AGLU03206 | STARTED | Placebo/Alglucosidase Alfa | 26 | |
| Extension Study AGLU03206 | COMPLETED | Alglucosidase Alfa/Alglucosidase Alfa | 55 | |
| Extension Study AGLU03206 | COMPLETED | Placebo/Alglucosidase Alfa | 26 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events1,215
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 | Anaemia | Blood and lymphatic system disorders | Alglucosidase Alfa/Alglucosidase Alfa | 4 | 60 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo/Alglucosidase Alfa | 0 | 26 | |
| other | Anaemia | Blood and lymphatic system disorders | Overall | 4 | 86 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Alglucosidase Alfa/Alglucosidase Alfa | 5 | 60 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Placebo/Alglucosidase Alfa | 0 | 26 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Overall | 5 | 86 | |
| other | Macrocytosis | Blood and lymphatic system disorders | Alglucosidase Alfa/Alglucosidase Alfa | 0 | 60 | |
| other | Macrocytosis | Blood and lymphatic system disorders | Placebo/Alglucosidase Alfa | 1 | 26 | |
| other | Macrocytosis | Blood and lymphatic system disorders | Overall | 1 | 86 | |
| other | Normochromic normocytic anaemia | Blood and lymphatic system disorders | Alglucosidase Alfa/Alglucosidase Alfa | 1 | 60 |
1 to 10 of 1,215Source ClinicalTrials.gov
See all 1,215 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Alglucosidase Alfa/Alglucosidase Alfa | ||
| Total, all-cause mortality | Placebo/Alglucosidase Alfa | ||
| Total, all-cause mortality | Overall | ||
| Total, other adverse events | Alglucosidase Alfa/Alglucosidase Alfa | 60 | 60 |
| Total, other adverse events | Placebo/Alglucosidase Alfa | 25 | 26 |
| Total, other adverse events | Overall | 85 | 86 |
| Total, serious adverse events | Alglucosidase Alfa/Alglucosidase Alfa | 15 | 60 |
| Total, serious adverse events | Placebo/Alglucosidase Alfa | 2 | 26 |
| Total, serious adverse events | Overall | 17 | 86 |
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT00455195
GET https://conductscience.org/api/trials/v1/NCT00455195/events?page=2