Alglucosidase Alfa Pompe Safety Sub-Registry

July 12, 2021 updated by: Genzyme, a Sanofi Company

A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune Complex Mediated Reactions With Alglucosidase Alfa Treatment

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

110

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Gent, Belgium, 9000
        • Investigational Site Number 056001
      • Leuven, Belgium, 3000
        • Investigational Site Number 056003
      • Leuven, Belgium, 3000
        • Investigational Site Number 056002
  • Czechia
      • Praha 2, Czechia, 12808
        • Investigational Site Number 203001
  • Germany
      • Halle (Saale), Germany, 06120
        • Investigational Site Number 276002
  • Italy
      • Brescia, Italy, 25123
        • Investigational Site Number 380001
      • Cagliari, Italy, 09126
        • Investigational Site Number 380002
      • Messina, Italy, 98125
        • Investigational Site Number 380006
      • Milano, Italy, 20133
        • Investigational Site Number 380005
      • Padova, Italy, 35128
        • Investigational Site Number 380004
      • Roma, Italy, 00168
        • Investigational Site Number 380003
  • Taiwan
      • Taipei, Taiwan, 10043
        • Investigational Site Number 158001
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Investigational Site Number 840016
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Investigational Site Number 840002
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Investigational Site Number 840004
      • Pittsburgh, Pennsylvania, United States, 15213
        • Investigational Site Number 840014
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Investigational Site Number 840008
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • Investigational Site Number 840001

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

Patients who are enrolled in the Pompe Registry will be enrolled in this sub-registry include patients with infantile-onset Pompe disease, as well as those with late-onset Pompe disease.

An approximately equal proportion of currently treated and treatment-naïve patients is targeted for enrollment at each site.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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
Biological: alglucosidase alfa
Alglucosidase alfa IV infusion of 20 mg/kg; qow
Other Names:
  • Myozyme; Lumizyme

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 20, 2015

Primary Completion (Actual)

April 20, 2021

Study Completion (Actual)

April 20, 2021

Study Registration Dates

First Submitted

October 17, 2012

First Submitted That Met QC Criteria

October 17, 2012

First Posted (Estimate)

October 19, 2012

Study Record Updates

Last Update Posted (Actual)

July 13, 2021

Last Update Submitted That Met QC Criteria

July 12, 2021

Last Verified

July 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AGLU06909
  • LTS13930 (Other Identifier: Other company study code)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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