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Late-Onset Treatment Study Extension Protocol

Sponsor
Genzyme, a Sanofi Company (Industry)
Overall Status
Completed
CT.gov ID
NCT00455195
Collaborator
(none)
81
Enrollment
31
Locations
2
Arms
20.1
Duration (Months)
2.6
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this extension study is to assess the long-term safety and efficacy of alglucosidase alfa treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704 (NCT00158600).

Condition or Disease Intervention/Treatment Phase
  • Biological: alglucosidase alfa
 Phase 4

Study Design

Study Type:
Interventional
Actual Enrollment :
81 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
An Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704
Study Start Date :
Mar 1, 2007
Actual Primary Completion Date :
Oct 1, 2008
Actual Study Completion Date :
Nov 1, 2008

Arms and Interventions

Arm Intervention/Treatment
Experimental: Alglucosidase Alfa/Alglucosidase Alfa

Participants who received alglucosidase alfa during the double-blind study and, if they completed the double-blind study, continued that treatment during the extension study. Participants received an intravenous (IV) infusion of 20 mg/kg of alglucosidase alfa every other week (qow) until their participation in both the AGLU02704 (NCT00158600) and AGLU03206 studies combined equaled a minimum of 104 weeks.

Biological: alglucosidase alfa
IV infusion of 20 mg/kg; every other week (qow)
Other Names:
  • Myozyme
  • Lumizyme

    		•
    Experimental: Placebo/Alglucosidase Alfa

    Participants given placebo during the double-blind study, completed the double-blind study (study AGLU02704, NCT00158600), and qualified to continue into the extension study on alglucosidase alfa. Participants received an intravenous (IV) infusion of 20 mg/kg of alglucosidase alfa every other week (qow) for up to 52 weeks. Only the alglucosidase alfa treatment experience is included in this extension study.

    Biological: alglucosidase alfa
    IV infusion of 20 mg/kg; every other week (qow)
    Other Names:
  • Myozyme
  • Lumizyme

    		•

    Outcome Measures

    Primary Outcome Measures

    1. 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).

    2. 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).

    3. 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.

    4. 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).

    5. 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 Outcome Measures

    1. 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.

    2. 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.

    3. 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).

    4. 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).

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    8 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Patient must have completed protocol AGLU02704 (NCT00158600)

    • Patient must provide signed, informed consent prior to performing any study-related procedures

    • Patient (and patient's legal guardian if patient is under 18 years of age) must have the ability to comply with the clinical protocol

    • A female patient of childbearing potential must have a negative pregnancy test at Baseline. (note: all female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.)

    Exclusion Criteria:
    • The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, would preclude treatment with alglucosidase alfa.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Anchorage Alaska United States
    2 Phoenix Arizona United States
    3 Beverly Hills California United States
    4 San Diego California United States
    5 Washington District of Columbia United States
    6 Coral Springs Florida United States
    7 Gainesville Florida United States
    8 Sarasota Florida United States
    9 Chicago Illinois United States
    10 Kansas City Kansas United States
    11 New Orleans Louisiana United States
    12 Boston Massachusetts United States
    13 Omaha Nebraska United States
    14 New York New York United States
    15 Durham North Carolina United States
    16 Cincinnati Ohio United States
    17 Toledo Ohio United States
    18 Tulsa Oklahoma United States
    19 Portland Oregon United States
    20 Philadelphia Pennsylvania United States
    21 Pittsburgh Pennsylvania United States
    22 Greenville South Carolina United States
    23 Dallas Texas United States
    24 San Antonio Texas United States
    25 Seattle Washington United States
    26 Green Bay Wisconsin United States
    27 Westmead Australia
    28 Calgary Alberta Canada
    29 Sherbrooke Quebec Canada
    30 Paris France
    31 Rotterdam Netherlands

    Sponsors and Collaborators

    • Genzyme, a Sanofi Company

    Investigators

    • Study Director: Medical Monitor, Genzyme, a Sanofi Company

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Genzyme, a Sanofi Company
    ClinicalTrials.gov Identifier:
    NCT00455195
    Other Study ID Numbers:
    • AGLU03206
    • 2006-003644-31
    First Posted:
    Apr 3, 2007
    Last Update Posted:
    Mar 7, 2014
    Last Verified:
    Feb 1, 2014
    Additional relevant MeSH terms:
    Glycogen Storage Disease Type II
    Glycogen Storage Disease

    Study Results

    Participant Flow

    Recruitment Details
    Pre-assignment Detail 81 of the 90 participants enrolled in the double-blind placebo controlled AGLU02704 (NCT00158600) study continued on to the open-label extension study AGLU03206.
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa Placebo/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206. Participants who received placebo during the double-blind study AGLU02704 (NCT00158600), completed the double-blind study, and started alglucosidase alfa in the extension study. Only participants' experience on alglucosidase alfa in the extension study is represented.
    Period Title: Double-blind Study AGLU02704
    STARTED 60 30
    COMPLETED 55 26
    NOT COMPLETED 5 4
    Period Title: Double-blind Study AGLU02704
    STARTED 55 26
    COMPLETED 55 26
    NOT COMPLETED 0 0

    Baseline Characteristics

    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa Placebo/Alglucosidase Alfa Total
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206. Participants who received placebo during the double-blind study AGLU02704 (NCT00158600), completed the double-blind study, and started alglucosidase alfa in the extension study. Only participants' experience on alglucosidase alfa in the extension study is represented. Total of all reporting groups
    Overall Participants 60 26 86
    Age (years) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [years]
    45.3
    (12.37)
    46.8
    (8.62)
    45.73
    (11.34)
    Sex: Female, Male (Count of Participants)
    Female
    26
    43.3%
    15
    57.7%
    41
    47.7%
    Male
    34
    56.7%
    11
    42.3%
    45
    52.3%
    Race/Ethnicity, Customized (participants) [Number]
    Hispanic
    1
    1.7%
    1
    3.8%
    2
    2.3%
    Asian
    1
    1.7%
    0
    0%
    1
    1.2%
    Black or African American
    0
    0%
    0
    0%
    0
    0%
    White
    57
    95%
    24
    92.3%
    81
    94.2%
    Unknown or Not Reported
    1
    1.7%
    1
    3.8%
    2
    2.3%

    Outcome Measures

    1. Primary Outcome
    Title Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
    Description 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).
    Time Frame Week 0 to 2.5 years

    Outcome Measure Data

    Analysis Population Description
    The safety population includes all participants randomized to alglucosidase alfa treatment in AGLU02704 (NCT00158600) who received at least one infusion of alglucosidase alfa.
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206.
    Measure Participants 60
    Participants with any AEs
    60
    100%
    Participants with Treatment-Related AEs
    37
    61.7%
    Participants with Infusion-Associated Reactions
    21
    35%
    Participants with Serious AEs
    15
    25%
    Participants with Severe AEs
    16
    26.7%
    Participants- Discontinued Due to AEs (incl death)
    3
    5%
    Participants Who Died
    1
    1.7%
    2. Secondary Outcome
    Title 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)
    Description 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.
    Time Frame Week 0

    Outcome Measure Data

    Analysis Population Description
    Intent-to-treat population
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206.
    Measure Participants 60
    Mean (Standard Deviation) [percent of predicted QMT]
    37.7
    (18.88)
    3. Primary Outcome
    Title 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)
    Description 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).
    Time Frame Week 0

    Outcome Measure Data

    Analysis Population Description
    Intent-to-treat population.
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206.
    Measure Participants 60
    Mean (Standard Deviation) [meters]
    332.2
    (126.69)
    4. Primary Outcome
    Title 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)
    Description 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.
    Time Frame Week 0, Week 104

    Outcome Measure Data

    Analysis Population Description
    Intent-to-treat population of participants who had both baseline (Week 0) and Week 104 data.
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206.
    Measure Participants 53
    Mean (Standard Deviation) [meters]
    21.3
    (78.02)
    5. Secondary Outcome
    Title 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)
    Description 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.
    Time Frame Week 0, Week 104

    Outcome Measure Data

    Analysis Population Description
    Intent-to-treat population of participants who had both baseline (Week 0) and Week 104 data.
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206.
    Measure Participants 53
    Mean (Standard Deviation) [percent of predicted QMT]
    2.1
    (11.06)
    6. Primary Outcome
    Title Baseline Values (Week 0) for Percent Predicted Forced Vital Capacity (FVC) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
    Description 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).
    Time Frame Week 0

    Outcome Measure Data

    Analysis Population Description
    Intent-to-treat population
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206.
    Measure Participants 60
    Mean (Standard Deviation) [percent of predicted FVC]
    55.4
    (14.44)
    7. Primary Outcome
    Title 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)
    Description 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.
    Time Frame Week 0, Week 104

    Outcome Measure Data

    Analysis Population Description
    Intent-to-treat population of participants who had both baseline (Week 0) and Week 104 data.
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206.
    Measure Participants 53
    Mean (Standard Deviation) [percent of predicted FVC]
    0.8
    (6.68)
    8. Secondary Outcome
    Title 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)
    Description 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).
    Time Frame Week 0

    Outcome Measure Data

    Analysis Population Description
    Intent-to-treat population of participants with valid baseline (Week 0) PCS surveys.
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206.
    Measure Participants 58
    Mean (Standard Deviation) [units on a scale]
    34.33
    (8.934)
    9. Secondary Outcome
    Title 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)
    Description 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).
    Time Frame Week 0 , Week 104

    Outcome Measure Data

    Analysis Population Description
    Intent-to-treat population of participants who had valid baseline (Week 0) and Week 104 data.
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206.
    Measure Participants 49
    Mean (Standard Deviation) [units on a scale]
    0.64
    (7.618)

    Adverse Events

    Time Frame Adverse events for the Alglucosidase Alfa/Alglucosidase Alfa group cover up to 2.5 years (Week 0 to 2.5 years), and for the Placebo/Alglucosidase Alfa group cover up to 1.0 year (Week 78 to 2.5 years).
    Adverse Event Reporting Description In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables. Events are listed independent of relationship to treatment reported.
    Arm/Group Title Alglucosidase Alfa/Alglucosidase Alfa Placebo/Alglucosidase Alfa Overall
    Arm/Group Description Participants who received alglucosidase alfa during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double blind study, continued alglucosidase alfa in the extension study. Both study experiences on alglucosidase alfa are represented within AGLU03206. Participants who received placebo during the double-blind study AGLU02704 (NCT00158600) and, if they completed the double-blind study, started alglucosidase alfa in the extension study. Only participants' experience on alglucosidase alfa in the extension study is represented. The combined alglucosidase alfa treatment experience from the two treatment groups.
    All Cause Mortality
    Alglucosidase Alfa/Alglucosidase Alfa Placebo/Alglucosidase Alfa Overall
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total / (NaN) / (NaN) / (NaN)
    Serious Adverse Events
    Alglucosidase Alfa/Alglucosidase Alfa Placebo/Alglucosidase Alfa Overall
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 15/60 (25%) 2/26 (7.7%) 17/86 (19.8%)
    Cardiac disorders
    Coronary artery disease 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Supraventricular tachycardia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Gastrointestinal disorders
    Abdominal pain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Gastric ulcer 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    General disorders
    Chest discomfort 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Non-cardiac chest pain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Immune system disorders
    Hypersensitivity 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Infections and infestations
    Gastroenteritis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Pneumonia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Injury, poisoning and procedural complications
    Fall 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Humerus fracture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Spinal compression fracture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Metabolism and nutrition disorders
    Dehydration 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Musculoskeletal and connective tissue disorders
    Intervertebral disc protrusion 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Cervix carcinoma stage II 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Nervous system disorders
    Brain stem ischaemia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Renal and urinary disorders
    Hydronephrosis 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Nephrolithiasis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Renal cyst 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Respiratory, thoracic and mediastinal disorders
    Lung disorder 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Throat tightness 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Skin and subcutaneous tissue disorders
    Angioneurotic oedema 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Vascular disorders
    Aneurysm 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Other (Not Including Serious) Adverse Events
    Alglucosidase Alfa/Alglucosidase Alfa Placebo/Alglucosidase Alfa Overall
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 60/60 (100%) 25/26 (96.2%) 85/86 (98.8%)
    Blood and lymphatic system disorders
    Anaemia 4/60 (6.7%) 0/26 (0%) 4/86 (4.7%)
    Lymphadenopathy 5/60 (8.3%) 0/26 (0%) 5/86 (5.8%)
    Macrocytosis 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Normochromic normocytic anaemia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Spontaneous haematoma 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Cardiac disorders
    Angina pectoris 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Atrial hypertrophy 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Bundle branch block left 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Bundle branch block right 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Coronary artery disease 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Palpitations 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Sinus tachycardia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tachycardia 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Tricuspid valve incompetence 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Ventricular dysfunction 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Ventricular hypertrophy 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Ear and labyrinth disorders
    Auricular swelling 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Cerumen impaction 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Ear congestion 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Ear discomfort 5/60 (8.3%) 0/26 (0%) 5/86 (5.8%)
    Ear pain 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Hypoacusis 23/60 (38.3%) 3/26 (11.5%) 26/86 (30.2%)
    Presbyacusis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tinnitus 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Vertigo 5/60 (8.3%) 0/26 (0%) 5/86 (5.8%)
    Vertigo positional 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Eye disorders
    Altered visual depth perception 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Asthenopia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Cataract 4/60 (6.7%) 1/26 (3.8%) 5/86 (5.8%)
    Conjunctivitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Diplopia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Dry eye 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Eye irritation 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Eye pain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Eye pruritus 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Eyelid ptosis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Keratoconjunctivitis sicca 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Photophobia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Pinguecula 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Retinal detachment 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Vision blurred 4/60 (6.7%) 0/26 (0%) 4/86 (4.7%)
    Visual acuity reduced 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Visual disturbance 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Vitreous floaters 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Gastrointestinal disorders
    Abdominal discomfort 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Abdominal distension 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Abdominal mass 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Abdominal pain 5/60 (8.3%) 0/26 (0%) 5/86 (5.8%)
    Abdominal pain lower 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Abdominal pain upper 6/60 (10%) 1/26 (3.8%) 7/86 (8.1%)
    Abdominal tenderness 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Anal fissure 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Anorectal disorder 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Aphthous stomatitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Bowel sounds abnormal 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Chapped lips 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Constipation 7/60 (11.7%) 0/26 (0%) 7/86 (8.1%)
    Crohn's disease 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Diarrhoea 20/60 (33.3%) 2/26 (7.7%) 22/86 (25.6%)
    Diverticulum intestinal 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Dry mouth 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Dyspepsia 7/60 (11.7%) 0/26 (0%) 7/86 (8.1%)
    Epigastric discomfort 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Flatulence 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Food poisoning 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Gastrooesophageal reflux disease 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Gingivitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Haematochezia 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Haemorrhoids 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Inguinal hernia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Lip swelling 2/60 (3.3%) 1/26 (3.8%) 3/86 (3.5%)
    Mouth ulceration 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Nausea 15/60 (25%) 2/26 (7.7%) 17/86 (19.8%)
    Oesophageal pain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Oral pruritus 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Paraesthesia oral 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Rectal haemorrhage 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Retching 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Stomach discomfort 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Swollen tongue 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Toothache 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Vomiting 14/60 (23.3%) 2/26 (7.7%) 16/86 (18.6%)
    General disorders
    Adverse drug reaction 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Application site vesicles 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Asthenia 4/60 (6.7%) 0/26 (0%) 4/86 (4.7%)
    Axillary pain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Catheter related complication 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Catheter site pain 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Chest discomfort 7/60 (11.7%) 0/26 (0%) 7/86 (8.1%)
    Chest pain 6/60 (10%) 2/26 (7.7%) 8/86 (9.3%)
    Chills 4/60 (6.7%) 1/26 (3.8%) 5/86 (5.8%)
    Disease progression 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Fatigue 13/60 (21.7%) 4/26 (15.4%) 17/86 (19.8%)
    Feeling abnormal 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Feeling cold 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Feeling hot 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Gait disturbance 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Influenza like illness 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Infusion site bruising 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Infusion site pain 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Infusion site paraesthesia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Infusion site reaction 7/60 (11.7%) 0/26 (0%) 7/86 (8.1%)
    Injection site pain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Injection site phlebitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Local swelling 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Malaise 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Nodule 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Oedema peripheral 13/60 (21.7%) 1/26 (3.8%) 14/86 (16.3%)
    Pain 6/60 (10%) 0/26 (0%) 6/86 (7%)
    Pitting oedema 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Puncture site haemorrhage 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Pyrexia 12/60 (20%) 2/26 (7.7%) 14/86 (16.3%)
    Thirst 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Immune system disorders
    Hypersensitivity 2/60 (3.3%) 1/26 (3.8%) 3/86 (3.5%)
    Seasonal allergy 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Infections and infestations
    Acute tonsillitis 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Bronchiolitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Bronchitis 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Bronchitis acute 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Cellulitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Cystitis 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Dermatophytosis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Ear infection 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Eye infection 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Folliculitis 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Fungal infection 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Fungal skin infection 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Gastroenteritis 8/60 (13.3%) 0/26 (0%) 8/86 (9.3%)
    Gastroenteritis viral 4/60 (6.7%) 0/26 (0%) 4/86 (4.7%)
    Gingival infection 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Herpes simplex 4/60 (6.7%) 1/26 (3.8%) 5/86 (5.8%)
    Herpes virus infection 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Herpes zoster 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Influenza 8/60 (13.3%) 3/26 (11.5%) 11/86 (12.8%)
    Kidney infection 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Laryngitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Localised infection 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Lower respiratory tract infection 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Mucosal infection 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Nasopharyngitis 29/60 (48.3%) 8/26 (30.8%) 37/86 (43%)
    Otitis media 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Pharyngitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Pharyngitis streptococcal 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Pneumonia 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Postoperative wound infection 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Rash pustular 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Respiratory tract infection 3/60 (5%) 1/26 (3.8%) 4/86 (4.7%)
    Rhinitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Sinusitis 9/60 (15%) 3/26 (11.5%) 12/86 (14%)
    Tinea pedis 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Tonsillitis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tooth abscess 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tooth infection 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Upper respiratory tract infection 20/60 (33.3%) 6/26 (23.1%) 26/86 (30.2%)
    Urinary tract infection 5/60 (8.3%) 2/26 (7.7%) 7/86 (8.1%)
    Urinary tract infection bacterial 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Vaginal infection 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Viral infection 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Wound infection 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Injury, poisoning and procedural complications
    Animal bite 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Animal scratch 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Arthropod bite 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Arthropod sting 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Back injury 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Concussion 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Contusion 9/60 (15%) 1/26 (3.8%) 10/86 (11.6%)
    Excoriation 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Fall 39/60 (65%) 13/26 (50%) 52/86 (60.5%)
    Femur fracture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Foot fracture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Head injury 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Humerus fracture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Injury 4/60 (6.7%) 0/26 (0%) 4/86 (4.7%)
    Injury corneal 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Joint sprain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Ligament injury 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Limb crushing injury 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Limb injury 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Mouth injury 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Muscle strain 6/60 (10%) 2/26 (7.7%) 8/86 (9.3%)
    Neck injury 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Pelvic fracture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Post-traumatic pain 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Procedural pain 9/60 (15%) 1/26 (3.8%) 10/86 (11.6%)
    Radial nerve injury 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Rib fracture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Road traffic accident 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Scratch 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Skin laceration 5/60 (8.3%) 0/26 (0%) 5/86 (5.8%)
    Soft tissue injury 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tendon injury 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tendon rupture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Thermal burn 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Tooth fracture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tooth injury 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Traumatic ulcer 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Vaccination complication 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Wound 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Investigations
    Alanine aminotransferase increased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Aspartate aminotransferase increased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Blood creatine increased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Blood folate decreased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Blood glucose increased 1/60 (1.7%) 2/26 (7.7%) 3/86 (3.5%)
    Blood pressure increased 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Blood uric acid increased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Blood urine present 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Body temperature increased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Carbon dioxide increased 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Crystal urine present 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Electrocardiogram QT corrected interval prolonged 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Electrocardiogram ST segment depression 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Electrocardiogram T wave amplitude decreased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Electrocardiogram abnormal 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Fungus urine test positive 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Glucose urine present 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Haematocrit decreased 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Neutrophil count increased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Oxygen saturation decreased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Protein urine present 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Specific gravity urine increased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tandem gait test abnormal 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Urine analysis abnormal 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Urine ketone body present 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Weight decreased 2/60 (3.3%) 1/26 (3.8%) 3/86 (3.5%)
    White blood cell count increased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    White blood cells urine positive 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Metabolism and nutrition disorders
    Anorexia 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Decreased appetite 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Gout 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Hyperglycaemia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Hypernatraemia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Hypertriglyceridaemia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Hypokalaemia 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Musculoskeletal and connective tissue disorders
    Arthralgia 25/60 (41.7%) 4/26 (15.4%) 29/86 (33.7%)
    Arthritis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Back pain 19/60 (31.7%) 6/26 (23.1%) 25/86 (29.1%)
    Bone pain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Bursitis 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Buttock pain 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Costochondritis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Flank pain 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Groin pain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Joint contracture 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Joint crepitation 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Muscle atrophy 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Muscle contracture 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Muscle spasms 17/60 (28.3%) 5/26 (19.2%) 22/86 (25.6%)
    Muscle tightness 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Muscle twitching 6/60 (10%) 0/26 (0%) 6/86 (7%)
    Muscular weakness 11/60 (18.3%) 2/26 (7.7%) 13/86 (15.1%)
    Musculoskeletal chest pain 6/60 (10%) 1/26 (3.8%) 7/86 (8.1%)
    Musculoskeletal discomfort 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Musculoskeletal disorder 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Musculoskeletal pain 10/60 (16.7%) 4/26 (15.4%) 14/86 (16.3%)
    Musculoskeletal stiffness 8/60 (13.3%) 0/26 (0%) 8/86 (9.3%)
    Myalgia 16/60 (26.7%) 3/26 (11.5%) 19/86 (22.1%)
    Neck pain 9/60 (15%) 1/26 (3.8%) 10/86 (11.6%)
    Nose deformity 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Osteoarthritis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Osteopenia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Osteoporosis 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Pain in extremity 23/60 (38.3%) 2/26 (7.7%) 25/86 (29.1%)
    Senile ankylosing vertebral hyperostosis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Sensation of heaviness 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Temporomandibular joint syndrome 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tendonitis 2/60 (3.3%) 1/26 (3.8%) 3/86 (3.5%)
    Tenosynovitis stenosans 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Basal cell carcinoma 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Skin papilloma 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Nervous system disorders
    Areflexia 5/60 (8.3%) 2/26 (7.7%) 7/86 (8.1%)
    Balance disorder 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Burning sensation 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Carpal tunnel syndrome 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Dizziness 16/60 (26.7%) 3/26 (11.5%) 19/86 (22.1%)
    Dizziness postural 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Facial palsy 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Headache 31/60 (51.7%) 6/26 (23.1%) 37/86 (43%)
    Hyperreflexia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Hypoaesthesia 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Hyporeflexia 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Intercostal neuralgia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Lethargy 2/60 (3.3%) 1/26 (3.8%) 3/86 (3.5%)
    Loss of consciousness 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Migraine 4/60 (6.7%) 0/26 (0%) 4/86 (4.7%)
    Nerve compression 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Neuralgia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Paraesthesia 7/60 (11.7%) 0/26 (0%) 7/86 (8.1%)
    Sciatica 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Sensory disturbance 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Sinus headache 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Somnolence 4/60 (6.7%) 0/26 (0%) 4/86 (4.7%)
    Syncope vasovagal 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Tremor 4/60 (6.7%) 1/26 (3.8%) 5/86 (5.8%)
    Psychiatric disorders
    Anger 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Anxiety 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Depression 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Insomnia 9/60 (15%) 0/26 (0%) 9/86 (10.5%)
    Post-traumatic stress disorder 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Stress 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Renal and urinary disorders
    Dysuria 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Haematuria 3/60 (5%) 1/26 (3.8%) 4/86 (4.7%)
    Leukocyturia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Nephrolithiasis 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Pollakiuria 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Proteinuria 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Pyuria 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Renal cyst 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Urge incontinence 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Urinary incontinence 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Urine flow decreased 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Urine odour abnormal 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Reproductive system and breast disorders
    Breast pain 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Breast swelling 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Breast tenderness 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Dysmenorrhoea 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Fibrocystic breast disease 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Menorrhagia 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Menstrual discomfort 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Menstruation irregular 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Pelvic pain 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Vaginal haemorrhage 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Respiratory, thoracic and mediastinal disorders
    Bronchospasm 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Choking 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Cough 8/60 (13.3%) 5/26 (19.2%) 13/86 (15.1%)
    Dyspnoea 9/60 (15%) 1/26 (3.8%) 10/86 (11.6%)
    Dyspnoea exertional 5/60 (8.3%) 0/26 (0%) 5/86 (5.8%)
    Epistaxis 4/60 (6.7%) 0/26 (0%) 4/86 (4.7%)
    Increased bronchial secretion 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Lung disorder 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Lung infiltration 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Nasal congestion 8/60 (13.3%) 0/26 (0%) 8/86 (9.3%)
    Pharyngolaryngeal pain 14/60 (23.3%) 3/26 (11.5%) 17/86 (19.8%)
    Postnasal drip 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Respiratory distress 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Respiratory failure 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Respiratory tract congestion 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Rhinorrhoea 3/60 (5%) 1/26 (3.8%) 4/86 (4.7%)
    Sinus congestion 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Sleep apnoea syndrome 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Sneezing 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Throat irritation 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Throat tightness 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Upper respiratory tract congestion 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Wheezing 1/60 (1.7%) 1/26 (3.8%) 2/86 (2.3%)
    Skin and subcutaneous tissue disorders
    Actinic keratosis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Cold sweat 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Decubitus ulcer 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Dermatitis contact 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Dry skin 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Ecchymosis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Erythema 3/60 (5%) 0/26 (0%) 3/86 (3.5%)
    Heat rash 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Hyperhidrosis 6/60 (10%) 1/26 (3.8%) 7/86 (8.1%)
    Ingrowing nail 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Night sweats 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Photosensitivity reaction 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Pruritus 8/60 (13.3%) 3/26 (11.5%) 11/86 (12.8%)
    Psoriasis 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Rash 9/60 (15%) 1/26 (3.8%) 10/86 (11.6%)
    Rash macular 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Rash maculo-papular 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Rash papular 5/60 (8.3%) 0/26 (0%) 5/86 (5.8%)
    Rash pruritic 4/60 (6.7%) 0/26 (0%) 4/86 (4.7%)
    Rosacea 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Skin hyperpigmentation 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Skin inflammation 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Skin lesion 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Skin nodule 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Skin odour abnormal 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Subcutaneous nodule 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Urticaria 7/60 (11.7%) 1/26 (3.8%) 8/86 (9.3%)
    Urticaria contact 0/60 (0%) 1/26 (3.8%) 1/86 (1.2%)
    Vascular disorders
    Aneurysm 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Cardiovascular insufficiency 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Diastolic hypotension 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Flushing 3/60 (5%) 1/26 (3.8%) 4/86 (4.7%)
    Haematoma 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Hot flush 2/60 (3.3%) 0/26 (0%) 2/86 (2.3%)
    Hypertension 5/60 (8.3%) 0/26 (0%) 5/86 (5.8%)
    Hypotension 2/60 (3.3%) 1/26 (3.8%) 3/86 (3.5%)
    Raynaud's phenomenon 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)
    Vasoconstriction 1/60 (1.7%) 0/26 (0%) 1/86 (1.2%)

    Limitations/Caveats

    [Not Specified]

    More Information

    Certain Agreements

    Principal Investigators are NOT employed by the organization sponsoring the study.

    In multi-site studies, PI can publish after Genzyme publishes or 18 months after study completion. PI gives Genzyme a draft 60 days before publication. Genzyme can ask that confidential information be removed, and can defer publication another 60 days upon notifying PI that it will file a patent application on inventions contained in the draft.

    Results Point of Contact

    Name/Title Genzyme Medical Information
    Organization Genzyme Corporation
    Phone 800-745-4447
    Email
    Responsible Party:
    Genzyme, a Sanofi Company
    ClinicalTrials.gov Identifier:
    NCT00455195
    Other Study ID Numbers:
    • AGLU03206
    • 2006-003644-31
    First Posted:
    Apr 3, 2007
    Last Update Posted:
    Mar 7, 2014
    Last Verified:
    Feb 1, 2014