Alglucosidase Alfa Pompe Safety Sub-Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01710813
First received: October 17, 2012
Last updated: March 6, 2014
Last verified: March 2014
  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 Phase
Pompe Disease
Biological: Alglucosidase alfa
Phase 4

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
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 manufactured at the 4000-L scale


Estimated Enrollment: 100
Study Start Date: April 2013
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
IV infusion of 20 mg/kg; qow
Other Name: Myozyme; Lumizyme

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Patients who 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;
  • 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 treatment with alglucosidase alfa (4000-L), plan to be treated with alglucosidase alfa, or have been treated with alglucosidase alfa for less than 2 years.

Exclusion Criteria:

  • if the patient does not plan to receive treatment with alglucosidase alfa (4000-L scale)
  • patients who have received an investigational drug (excluding alglucosidase alfa) within 30 days prior to signing a sub-registry consent form
  • if they are taking or plan to take any investigational product while enrolled in the 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: Medical Information 8007454447 medinfo@genzyme.com
Contact: Medical Information 6172527832 medinfo@genzyme.com

Locations
United States, Massachusetts
Recruiting
Cambridge, Massachusetts, United States
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Sanofi ( 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: March 6, 2014
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
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
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
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 15, 2014