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Alglucosidase Alfa Pompe Safety Sub-Registry

This study is currently recruiting participants.
See Contacts and Locations
Verified June 2017 by Sanofi ( Genzyme, a Sanofi Company )
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company ) Identifier:
First received: October 17, 2012
Last updated: June 7, 2017
Last verified: June 2017
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.

Condition Intervention
Pompe Disease Biological: alglucosidase alfa

Study Type: Observational [Patient Registry]
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 4 Years
Official Title: 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

Resource links provided by NLM:

Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):

Primary Outcome Measures:
  • 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

Estimated Enrollment: 100
Study Start Date: March 20, 2015
Estimated Study Completion Date: April 2021
Estimated Primary Completion Date: March 2021 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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 Name: Myozyme; Lumizyme


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01710813

Contact: For site information, send an email with site number to
Contact: Medical Information 6172527832

United States, Arizona
Investigational Site Number 840016 Recruiting
Phoenix, Arizona, United States, 85013
United States, North Carolina
Investigational Site Number 840002 Recruiting
Durham, North Carolina, United States, 27710
United States, Pennsylvania
Investigational Site Number 840004 Recruiting
Hershey, Pennsylvania, United States, 17033
Investigational Site Number 840014 Recruiting
Pittsburgh, Pennsylvania, United States, 15213
United States, Utah
Investigational Site Number 840008 Recruiting
Salt Lake City, Utah, United States, 84113
United States, Virginia
Investigational Site Number 840001 Recruiting
Fairfax, Virginia, United States, 22030
Investigational Site Number 056001 Recruiting
Gent, Belgium, 9000
Investigational Site Number 056002 Recruiting
Leuven, Belgium, 3000
Investigational Site Number 056003 Recruiting
Leuven, Belgium, 3000
Investigational Site Number 203001 Recruiting
Praha 2, Czechia, 12808
Investigational Site Number 276002 Recruiting
Halle (Saale), Germany, 06120
Investigational Site Number 380001 Recruiting
Brescia, Italy, 25123
Investigational Site Number 380002 Recruiting
Cagliari, Italy, 09126
Investigational Site Number 380006 Recruiting
Messina, Italy, 98125
Investigational Site Number 380005 Recruiting
Milano, Italy, 20133
Investigational Site Number 380004 Recruiting
Padova, Italy, 35128
Investigational Site Number 380003 Recruiting
Roma, Italy, 00168
Investigational Site Number 158001 Active, not recruiting
Taipei, Taiwan, 10043
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Genzyme, a Sanofi Company Identifier: NCT01710813     History of Changes
Other Study ID Numbers: AGLU06909
LTS13930 ( Other Identifier: Other company study code )
Study First Received: October 17, 2012
Last Updated: June 7, 2017

Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
pompe disease
alglucosidase alfa
severe cutaneous
systemic immune complex-mediated reactions

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Antigen-Antibody Complex
Immunologic Factors
Physiological Effects of Drugs processed this record on September 19, 2017