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Alglucosidase Alfa Pompe Safety Sub-Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2016 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01710813
First received: October 17, 2012
Last updated: September 15, 2016
Last verified: September 2016
  Purpose
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:

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 ] [ Designated as safety issue: No ]
    collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa


Estimated Enrollment: 100
Study Start Date: March 2015
Estimated Study Completion Date: September 2022
Estimated Primary Completion Date: August 2022 (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

  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Genders Eligible for Study:   Both
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.

Criteria

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 ClinicalTrials.gov identifier: NCT01710813

Contacts
Contact: For site information, send an email with site number to Contact-Us@sanofi.com
Contact: Medical Information 6172527832 medinfo@genzyme.com

Locations
United States, Pennsylvania
Investigational Site Number 840152323 Recruiting
Hershey, Pennsylvania, United States, 17033
Investigational Site Number 840152318 Recruiting
Pittsburgh, Pennsylvania, United States, 15213
United States, Utah
Investigational Site Number 150594 Recruiting
Salt Lake City, Utah, United States, 84113
United States, Virginia
Investigational Site Number 840151747 Recruiting
Fairfax, Virginia, United States, 22030
Belgium
Investigational Site Number 056001 Recruiting
Gent, Belgium, 9000
Investigational Site Number 056002 Recruiting
Leuven, Belgium, 3000
Investigational Site Number 056003 Recruiting
Leuven, Belgium, 3000
Germany
Investigational Site Number 276001 Recruiting
Halle (Saale), Germany, 06120
Investigational Site Number 276002 Recruiting
Mainz, Germany, 55131
Italy
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
Taiwan
Investigational Site Number 158001 Recruiting
Taipai, Taiwan, 10043
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT01710813     History of Changes
Other Study ID Numbers: AGLU06909  LTS13930 
Study First Received: October 17, 2012
Last Updated: September 15, 2016
Health Authority: United States: Food and Drug Administration
European Union: European Medicines Agency

Keywords provided by Sanofi:
pompe disease
alglucosidase alfa
anaphylaxis
severe cutaneous
systemic immune complex-mediated reactions

Additional relevant MeSH terms:
Anaphylaxis
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
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Antigen-Antibody Complex
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 26, 2016