Glycogen Storage Disease Type II (GSD-II)ATAPNCT00520143
Alglucosidase Alfa Temporary Access Program
A study testing alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]) in people with Glycogen Storage Disease Type II (GSD-II) and Pompe Disease (Late-Onset), run by Genzyme, a Sanofi Company at 74 sites in United States.
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 18 years and older
- Conditions the study is for
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease
- What is being tested?
- alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 74 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures.
- The patient must reside in the US.
- The patient must have a confirmed diagnosis of Pompe disease defined as documented acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or GAA gene mutations.
- The patient must have/had documented clinical signs and symptoms of Pompe disease.
- The patient must have/had prior treatment with alglucosidase alfa produced at commercial scale OR be naive to enzyme replacement therapy (ERT) for the treatment of Pompe disease and meet at least 1 of the following criteria: require a wheelchair OR require some respiratory assistance for any number of hours (including night time) through non-invasive ventilation.
- The patient must be capable of complying with the required program schedule of assessments.
Who cannot join
- Females who are pregnant or lactating
- The patient has a clinical condition unrelated to Pompe disease that would interfere with program assessments.
- The patient is currently enrolled in any clinical studies.
Trial locations and contacts
74 sites in 1 country.
74 sites in 1 country
United States74 sites
Study site
Albany, New York
Study site
Albuquerque, New Mexico
Study site
Anchorage, Alaska
Study site
Asheville, North Carolina
Study site
Augusta, Maine
Study site
Aurora, Colorado
Study site
Bakersfield, California
Study site
Baltimore, Maryland
Study site
Beverly Hills, California
Study site
Boise, Idaho
Study site
Boston, Massachusetts
Study site
Bradenton, Florida
Study site
Charlottesville, Virginia
Study site
Chesapeake, Virginia
Study site
Chicago, Illinois
Study site
Cincinnati, Ohio
Study site
Cleveland, Ohio
Study site
Coral Springs, Florida
Study site
Corning, New York
Study site
Daytona Beach, Florida
Study site
Decatur, Georgia
Study site
Detroit, Michigan
Study site
Durham, North Carolina
Study site
Evansville, Indiana
Study site
Gainsville, Florida
Study site
Grand Rapids, Michigan
Study site
Green Bay, Wisconsin
Study site
Greenville, Ohio
Study site
Greenville, South Carolina
Study site
Houston, Texas
Study site
Iowa City, Iowa
Study site
Jacksonville, North Carolina
Study site
Kansas City, Kansas
Study site
Lake Orion, Michigan
Study site
Loma Linda, California
Study site
Los Angeles, California
Study site
Louisville, Kentucky
Study site
Manhasset, New York
Study site
Midland Park, New Jersey
Study site
Minneapolis, Minnesota
Study site
Missoula, Montana
Study site
Morgantown, West Virginia
Study site
New Orleans, Louisiana
Study site
New York, New York
Study site
Newport News, Virginia
Study site
Oklahoma City, Oklahoma
Study site
Omaha, Nebraska
Study site
Philadelphia, Pennsylvania
Study site
Phoenix, Arizona
Study site
Pittsburgh, Pennsylvania
Study site
Portland, Oregon
Study site
Prescott, Arizona
Study site
Pullman, Washington
Study site
Richmond, Virginia
Study site
Rochester Hills, Michigan
Study site
Sacramento, California
Study site
Saint Joseph, Michigan
Study site
Salt Lake City, Utah
Study site
San Antonio, Texas
Study site
San Diego, California
Study site
Scottsdale, Arizona
Study site
Seattle, Washington
Study site
Sheffield, Alabama
Study site
Shreveport, Louisiana
Study site
Springfield, Massachusetts
Study site
St. Petersburg, Florida
Study site
Stanford, California
Study site
Toledo, Ohio
Study site
Tucson, Arizona
Study site
Tulsa, Oklahoma
Study site
Waco, Texas
Study site
Washington D.C., District of Columbia
Study site
West Colombia, South Carolina
Study site
Winchester, Virginia
About this study
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 expanded access study is to provide patients with Pompe disease in the United States (US), access to alglucosidase alfa produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration.
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
- Being tested
- alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
- Keywords
- GSD-II, Pompe Disease, Glycogen Storage Disease II
- 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 alfa (recombinant human acid alpha-glucosidase [rhGAA])Biological
Also called Alglucosidase alfa
IV infusion: 20mg/kg qow
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2007 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage Disease Type II (GSD-II)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
- Expanded access
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
- NCT00520143
- Sponsor's protocol ID
- AGLU03907
- Acronym
- ATAP
- Status last verified
- Feb 2014
- First submitted
- 21 Aug 2007
- Last updated
- 6 Feb 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Alglucosidase Alfa Temporary Access Program
- ClinicalTrials.gov ID
- NCT00520143
- Acronym / sponsor ID
- ATAP / AGLU03907
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
- Keywords
- GSD-II, Pompe Disease, Glycogen Storage Disease II
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- First posted
- 23 Aug 2007 (estimated)
- Results
- Not posted in the source record
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 expanded access study is to provide patients with Pompe disease in the United States (US), access to alglucosidase alfa produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures.
- The patient must reside in the US.
- The patient must have a confirmed diagnosis of Pompe disease defined as documented acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or GAA gene mutations.
- The patient must have/had documented clinical signs and symptoms of Pompe disease.
- The patient must have/had prior treatment with alglucosidase alfa produced at commercial scale OR be naive to enzyme replacement therapy (ERT) for the treatment of Pompe disease and meet at least 1 of the following criteria: require a wheelchair OR require some respiratory assistance for any number of hours (including night time) through non-invasive ventilation.
Key exclusion criteria (3 listed)
- Females who are pregnant or lactating
- The patient has a clinical condition unrelated to Pompe disease that would interfere with program assessments.
- The patient is currently enrolled in any clinical studies.
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 alfa (recombinant human acid alpha-glucosidase [rhGAA])Also: Alglucosidase alfaIV infusion: 20mg/kg qow | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2014.
Related trials for Glycogen Storage Disease Type II (GSD-II)
RecruitingObservational study
Pompe Pregnancy Sub-Registry
Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset)
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
74 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]) in people with Glycogen Storage Disease Type II (GSD-II) and Pompe Disease (Late-Onset), run by Genzyme, a Sanofi Company at 74 sites in United States.
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 6 inclusion and 3 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: status not confirmed, as of the sponsor's last update on 6 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 74 sites in United States. 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.
Are results available for this study?
Results are not available in the source record.
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.
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 6 Feb 2014; AzTrials last refreshed its copy on 28 Oct 2026.