Alglucosidase Alfa Pompe Safety Sub-Registry

Sponsor

Genzyme, a Sanofi Company (Industry)

Overall Status

Completed

CT.gov ID

NCT01710813

Collaborator

(none)

110

Enrollment

Locations

Actual Duration (Months)

6.1

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Pompe Disease	Biological: alglucosidase alfa

Study Design

Study Type:

Observational [Patient Registry]

Actual Enrollment :

110 participants

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

Actual Study Start Date :

Mar 20, 2015

Actual Primary Completion Date :

Apr 20, 2021

Actual Study Completion Date :

Apr 20, 2021

Arms and Interventions

Arm	Intervention/Treatment
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 Names: Myozyme; Lumizyme

Arm

Intervention/Treatment

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 Names:

Myozyme; Lumizyme

Outcome Measures

Primary Outcome Measures

number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions [4 Years]
collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	Investigational Site Number 840016	Phoenix	Arizona	United States	85013
2	Investigational Site Number 840002	Durham	North Carolina	United States	27710
3	Investigational Site Number 840004	Hershey	Pennsylvania	United States	17033
4	Investigational Site Number 840014	Pittsburgh	Pennsylvania	United States	15213
5	Investigational Site Number 840008	Salt Lake City	Utah	United States	84113
6	Investigational Site Number 840001	Fairfax	Virginia	United States	22030
7	Investigational Site Number 056001	Gent		Belgium	9000
8	Investigational Site Number 056002	Leuven		Belgium	3000
9	Investigational Site Number 056003	Leuven		Belgium	3000
10	Investigational Site Number 203001	Praha 2		Czechia	12808
11	Investigational Site Number 276002	Halle (Saale)		Germany	06120
12	Investigational Site Number 380001	Brescia		Italy	25123
13	Investigational Site Number 380002	Cagliari		Italy	09126
14	Investigational Site Number 380006	Messina		Italy	98125
15	Investigational Site Number 380005	Milano		Italy	20133
16	Investigational Site Number 380004	Padova		Italy	35128
17	Investigational Site Number 380003	Roma		Italy	00168
18	Investigational Site Number 158001	Taipei		Taiwan	10043