An expanded access record providing alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]) in Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset) and Acid Maltase Deficiency Disease, sponsored by Genzyme, a Sanofi Company. Approved for marketing at 74 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2014-02-06.
Sponsored by Genzyme, a Sanofi Company · Expanded access
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.
151 studies on the registry are indexed under Glycogen Storage Disease Type II; 30 are open to participants now.
Browse Glycogen Storage Disease Type II studies →Genzyme, a Sanofi Company is the lead sponsor of 303 studies on the registry; 5 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 19 (79%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
IV infusion: 20mg/kg qow
Also known as: Alglucosidase alfa
Expanded access is arranged between your doctor and the company. Ask your care team to contact the provider listed on this record.
No contact was published for this record. The registry link below has the sponsor’s details.
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Glycogen Storage Disease Type II→
Genzyme, a Sanofi Company