Pompe Disease (Late-Onset)Phase 4NCT00455195
Late-Onset Treatment Study Extension Protocol
A Phase 4 study testing alglucosidase alfa in people with Pompe Disease (Late-Onset) and Glycogen Storage Disease Type II (GSD-II), run by Genzyme, a Sanofi Company at 31 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II
- What is being tested?
- alglucosidase alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 81 participants
- Where
- 31 sitesin 5 countries
- Study dates
- Mar 2007to Nov 2008
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.)
Who cannot join
- 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.
Trial locations and contacts
31 sites in 5 countries.
31 sites in 5 countries
United States26 sites
Study site
Anchorage, Alaska
Study site
Beverly Hills, California
Study site
Boston, Massachusetts
Study site
Chicago, Illinois
Study site
Cincinnati, Ohio
Study site
Coral Springs, Florida
Study site
Dallas, Texas
Study site
Durham, North Carolina
Study site
Gainesville, Florida
Study site
Green Bay, Wisconsin
Study site
Greenville, South Carolina
Study site
Kansas City, Kansas
Study site
New Orleans, Louisiana
Study site
New York, New York
Study site
Omaha, Nebraska
Study site
Philadelphia, Pennsylvania
Study site
Phoenix, Arizona
Study site
Pittsburgh, Pennsylvania
Study site
Portland, Oregon
Study site
San Antonio, Texas
Study site
San Diego, California
Study site
Sarasota, Florida
Study site
Seattle, Washington
Study site
Toledo, Ohio
Study site
Tulsa, Oklahoma
Study site
Washington D.C., District of Columbia
About this study
Official title: An Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704
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).
- Conditions
- Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)
- Being tested
- alglucosidase alfa
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
alglucosidase alfaBiological
Also called Myozyme, Lumizyme
IV infusion of 20 mg/kg; every other week (qow)
| Study group | Role | What they receive |
|---|---|---|
| Alglucosidase Alfa/Alglucosidase Alfa | Experimental | 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. Receives: Biological: alglucosidase alfa |
| Placebo/Alglucosidase Alfa | Experimental | 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. Receives: Biological: alglucosidase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
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).
Measured: Week 0 to 2.5 years
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)
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).
Measured: Week 0
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)
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.
Measured: Week 0, Week 104
Baseline Values (Week 0) for Percent Predicted Forced Vital Capacity (FVC) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
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).
Measured: Week 0
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)
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.
Measured: Week 0, Week 104
Secondary outcomes (4)
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)
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.
Measured: Week 0
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)
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.
Measured: Week 0, Week 104
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)
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).
Measured: Week 0
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)
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).
Measured: Week 0 , Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2007 (estimated)
- Study startMar 2007
- Primary completionOct 2008
- Study completionNov 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pompe Disease (Late-Onset)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 81 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00455195
- Sponsor's protocol ID
- AGLU03206
- Other IDs
- 2006-003644-31
- Status last verified
- Feb 2014
- First submitted
- 30 Mar 2007
- Last updated
- 7 Mar 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704
- ClinicalTrials.gov ID
- NCT00455195
- Acronym / sponsor ID
- AGLU03206
- Conditions
- Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 81 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Mar 2007
- Primary completion
- Oct 2008 (actual)
- Study completion
- Nov 2008 (actual)
- First posted
- 3 Apr 2007 (estimated)
- Results
- Posted 5 Jan 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Alglucosidase Alfa/Alglucosidase Alfa | Experimental | Biological: alglucosidase alfaParticipants 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. |
| Placebo/Alglucosidase Alfa | Experimental | Biological: alglucosidase alfaParticipants 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. |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.)
Key exclusion criteria (1 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| alglucosidase alfaAlso: Myozyme, LumizymeIV infusion of 20 mg/kg; every other week (qow) | Biological | Alglucosidase Alfa/Alglucosidase Alfa, Placebo/Alglucosidase Alfa |
Primary outcome
Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
Time frame: 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).
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)
Time frame: 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).
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)
Time frame: 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.
Baseline Values (Week 0) for Percent Predicted Forced Vital Capacity (FVC) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
Time frame: 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).
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)
Time frame: 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.
4 secondary outcomes
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)
Time frame: Week 0
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)
Time frame: Week 0, Week 104
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)
Time frame: Week 0
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)
Time frame: Week 0 , Week 104
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme, a Sanofi Company
31 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing alglucosidase alfa in people with Pompe Disease (Late-Onset) and Glycogen Storage Disease Type II (GSD-II), run by Genzyme, a Sanofi Company at 31 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in United States, Canada, Australia, France, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Jan 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) (time frame: Week 0 to 2.5 years). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Mar 2014.