Alglucosidase Alfa Pompe Safety Sub-Registry

Pompe Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01710813
• Other study ID #: AGLU06909
• Secondary ID: LTS13930
• Status: Completed
• Start date: March 20, 2015
• Est. completion date: April 20, 2021

Study information

• Verified date: July 2021
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 110
• Est. completion date: April 20, 2021
• Est. primary completion date: April 20, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease

Intervention

Biological:
• alglucosidase alfa
Alglucosidase alfa IV infusion of 20 mg/kg; qow

Locations

Country	Name	City	State
Belgium	Investigational Site Number 056001	Gent
Belgium	Investigational Site Number 056002	Leuven
Belgium	Investigational Site Number 056003	Leuven
Czechia	Investigational Site Number 203001	Praha 2
Germany	Investigational Site Number 276002	Halle (Saale)
Italy	Investigational Site Number 380001	Brescia
Italy	Investigational Site Number 380002	Cagliari
Italy	Investigational Site Number 380006	Messina
Italy	Investigational Site Number 380005	Milano
Italy	Investigational Site Number 380004	Padova
Italy	Investigational Site Number 380003	Roma
Taiwan	Investigational Site Number 158001	Taipei
United States	Investigational Site Number 840002	Durham	North Carolina
United States	Investigational Site Number 840001	Fairfax	Virginia
United States	Investigational Site Number 840004	Hershey	Pennsylvania
United States	Investigational Site Number 840016	Phoenix	Arizona
United States	Investigational Site Number 840014	Pittsburgh	Pennsylvania
United States	Investigational Site Number 840008	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  Belgium,  Czechia,  Germany,  Italy,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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