Alglucosidase Alfa Pompe Safety Sub-Registry

NCT01710813 | Completed DMC

• 2 other identifiers: AGLU06909LTS13930
• Study Type: observational
• Target: 110
• Locations: 6 countries
• Sites: 18

Brief Summary

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.

Conditions

Pompe Disease

Keywords

pompe disease; alglucosidase alfa; anaphylaxis; severe cutaneous; systemic immune complex-mediated reactions

Outcome Measures

Primary Outcomes (1)

• number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions

  collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa

  4 Years

Study Arms (1)

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

Interventions

alglucosidase alfa BIOLOGICAL

Alglucosidase alfa IV infusion of 20 mg/kg; qow

Also known as: Myozyme; Lumizyme

pompe safety sub-registry

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (18)

Investigational Site Number 840016

Phoenix, Arizona, 85013, United States

Location

Investigational Site Number 840002

Durham, North Carolina, 27710, United States

Location

Investigational Site Number 840004

Hershey, Pennsylvania, 17033, United States

Location

Investigational Site Number 840014

Pittsburgh, Pennsylvania, 15213, United States

Location

Investigational Site Number 840008

Salt Lake City, Utah, 84113, United States

Location

Investigational Site Number 840001

Fairfax, Virginia, 22030, United States

Location

Investigational Site Number 056001

Ghent, 9000, Belgium

Location

Investigational Site Number 056002

Leuven, 3000, Belgium

Location

Investigational Site Number 056003

Leuven, 3000, Belgium

Location

Investigational Site Number 203001

Prague, 12808, Czechia

Location

Investigational Site Number 276002

Halle, 06120, Germany

Location

Investigational Site Number 380001

Brescia, 25123, Italy

Location

Investigational Site Number 380002

Cagliari, 09126, Italy

Location

Investigational Site Number 380006

Messina, 98125, Italy

Location

Investigational Site Number 380005

Milan, 20133, Italy

Location

Investigational Site Number 380004

Padua, 35128, Italy

Location

Investigational Site Number 380003

Roma, 00168, Italy

Location

Investigational Site Number 158001

Taipei, 10043, Taiwan

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type II; Anaphylaxis

Interventions

GAA protein, human

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Study Officials

• Medical Monitor

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 4 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2012
• First Posted: October 19, 2012
• Study Start: March 20, 2015
• Primary Completion: April 20, 2021
• Study Completion: April 20, 2021
• Last Updated: July 13, 2021

Record last verified: 2021-07

Locations

DE (1); IT (6); CZ (1); US (6); BE (3); TW (1)