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Alglucosidase Alfa Pompe Safety Sub-Registry

NCT ID: NCT01710813

Last Updated: 2021-07-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

110 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-03-20

Study Completion Date

2021-04-20

Brief Summary

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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.

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Detailed Description

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Conditions

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Pompe Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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pompe safety sub-registry

patients are selected from those who are enrolled in the Pompe Registry, and will be followed for safety evaluation in this sub-registry

alglucosidase alfa

Intervention Type BIOLOGICAL

Alglucosidase alfa IV infusion of 20 mg/kg; qow

Interventions

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alglucosidase alfa

Alglucosidase alfa IV infusion of 20 mg/kg; qow

Intervention Type BIOLOGICAL

Other Intervention Names

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Myozyme; Lumizyme

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

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Investigational Site Number 840016

Phoenix, Arizona, United States

Site Status

Investigational Site Number 840002

Durham, North Carolina, United States

Site Status

Investigational Site Number 840004

Hershey, Pennsylvania, United States

Site Status

Investigational Site Number 840014

Pittsburgh, Pennsylvania, United States

Site Status

Investigational Site Number 840008

Salt Lake City, Utah, United States

Site Status

Investigational Site Number 840001

Fairfax, Virginia, United States

Site Status

Investigational Site Number 056001

Ghent, , Belgium

Site Status

Investigational Site Number 056002

Leuven, , Belgium

Site Status

Investigational Site Number 056003

Leuven, , Belgium

Site Status

Investigational Site Number 203001

Prague, , Czechia

Site Status

Investigational Site Number 276002

Halle, , Germany

Site Status

Investigational Site Number 380001

Brescia, , Italy

Site Status

Investigational Site Number 380002

Cagliari, , Italy

Site Status

Investigational Site Number 380006

Messina, , Italy

Site Status

Investigational Site Number 380005

Milan, , Italy

Site Status

Investigational Site Number 380004

Padua, , Italy

Site Status

Investigational Site Number 380003

Roma, , Italy

Site Status

Investigational Site Number 158001

Taipei, , Taiwan

Site Status

Countries

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United States Belgium Czechia Germany Italy Taiwan

Other Identifiers

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LTS13930

Identifier Type: OTHER

Identifier Source: secondary_id

AGLU06909

Identifier Type: -

Identifier Source: org_study_id

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