Alglucosidase Alfa Pompe Safety Sub-Registry

Completed

Conditions studied: Pompe Disease

In brief

To collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune complex-mediated reactions following treatment with alglucosidase alfa.

Key facts

Study ID

NCT01710813

Run by

Genzyme, a Sanofi Company

People needed

110

Starts

2015-03-20

Expected to finish

2021-04-20

Last updated by the study team

2021-07-13

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• The patient must be enrolled in the Pompe Registry;
• Provide a signed patient information and authorization form;
• Have a confirmed diagnosis of Pompe disease (confirmation of diagnosis is defined as documented GAA enzyme deficiency from any tissue source and/or documentation of 2 GAA gene mutations);
• Be naïve to and plan to be treated with alglucosidase alfa at or prior to enrollment, or are being treated with alglucosidase alfa.

You may not qualify if…

• Patients will be excluded if they have received an investigational drug (excluding alglucosidase alfa) within 30 days prior to signing a Safety Sub-Registry Patient Information and Authorization form, or if they are taking or plan to take any investigational product while enrolled in the Safety Sub-Registry.

Where it is running

• Investigational Site Number 840016 — Phoenix, Arizona, United States
• Investigational Site Number 840002 — Durham, North Carolina, United States
• Investigational Site Number 840004 — Hershey, Pennsylvania, United States
• Investigational Site Number 840014 — Pittsburgh, Pennsylvania, United States
• Investigational Site Number 840008 — Salt Lake City, Utah, United States
• Investigational Site Number 840001 — Fairfax, Virginia, United States
• Investigational Site Number 056001 — Ghent, Belgium
• Investigational Site Number 056002 — Leuven, Belgium
• Investigational Site Number 056003 — Leuven, Belgium
• Investigational Site Number 203001 — Prague, Czechia
• Investigational Site Number 276002 — Halle, Germany
• Investigational Site Number 380001 — Brescia, Italy
• Investigational Site Number 380002 — Cagliari, Italy
• Investigational Site Number 380006 — Messina, Italy
• Investigational Site Number 380005 — Milan, Italy
• Investigational Site Number 380004 — Padova, Italy
• Investigational Site Number 380003 — Roma, Italy
• Investigational Site Number 158001 — Taipei, Taiwan

Full record on ClinicalTrials.gov

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