Alglucosidase Alfa Pompe Safety Sub-Registry

Completed

• Conditions: Pompe Disease
• Interventions: Biological: alglucosidase alfa

• Registration Number: NCT01710813
• Lead Sponsor: Genzyme, a Sanofi Company

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-10-17
• Study First Submitted QC: 2012-10-17
• Study First Posted: 2012-10-19
• Study Start: 2015-03-20
• Primary Completion: 2021-04-20
• Study Completion: 2021-04-20
• Status Verified: 2021-07
• Last Update Submitted: 2021-07-12
• Last Update Posted: 2021-07-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 110

Inclusion Criteria

• 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.

Exclusion Criteria

• 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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
pompe safety sub-registry	alglucosidase alfa	patients are selected from those who are enrolled in the Pompe Registry, and will be followed for safety evaluation in this sub-registry

Primary Outcome Measures

Name	Time	Method
number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions	4 Years	collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa

Trial Locations

Locations (18)

Investigational Site Number 056002; 🇧🇪 Leuven, Belgium

Investigational Site Number 158001; 🇨🇳 Taipei, Taiwan

Investigational Site Number 380006; 🇮🇹 Messina, Italy

Investigational Site Number 203001; 🇨🇿 Praha 2, Czechia

Investigational Site Number 276002; 🇩🇪 Halle (Saale), Germany

Investigational Site Number 380001; 🇮🇹 Brescia, Italy

Investigational Site Number 380005; 🇮🇹 Milano, Italy

Investigational Site Number 840004; 🇺🇸 Hershey, Pennsylvania, United States

Investigational Site Number 840014; 🇺🇸 Pittsburgh, Pennsylvania, United States

Investigational Site Number 380002; 🇮🇹 Cagliari, Italy

Investigational Site Number 056001; 🇧🇪 Gent, Belgium

Investigational Site Number 840001; 🇺🇸 Fairfax, Virginia, United States

Investigational Site Number 056003; 🇧🇪 Leuven, Belgium

Investigational Site Number 380004; 🇮🇹 Padova, Italy

Investigational Site Number 840016; 🇺🇸 Phoenix, Arizona, United States

Investigational Site Number 840002; 🇺🇸 Durham, North Carolina, United States

Investigational Site Number 840008; 🇺🇸 Salt Lake City, Utah, United States

Investigational Site Number 380003; 🇮🇹 Roma, Italy