Clinical trial NCT01710813: Alglucosidase Alfa Pompe Safety Sub-Registry
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pompe Disease
Keywords: pompe disease
alglucosidase alfa
anaphylaxis
severe cutaneous
systemic immune complex-mediated reactions
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2012-10-19
Enrollment: 110
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


Eligibility 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.

Primary outcomes:
measure: number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions
timeFrame: 4 Years
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Investigational Site Number 840016
state: Arizona
city: Durham
country: United States
facility: Investigational Site Number 840002
state: North Carolina
city: Hershey
country: United States
facility: Investigational Site Number 840004
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Investigational Site Number 840014
state: Pennsylvania
city: Salt Lake City
country: United States
facility: Investigational Site Number 840008
state: Utah
city: Fairfax
country: United States
facility: Investigational Site Number 840001
state: Virginia
city: Gent
country: Belgium
facility: Investigational Site Number 056001
city: Leuven
country: Belgium
facility: Investigational Site Number 056002
city: Leuven
country: Belgium
facility: Investigational Site Number 056003
city: Praha 2
country: Czechia
facility: Investigational Site Number 203001
city: Halle (Saale)
country: Germany
facility: Investigational Site Number 276002
city: Brescia
country: Italy
facility: Investigational Site Number 380001
city: Cagliari
country: Italy
facility: Investigational Site Number 380002
city: Messina
country: Italy
facility: Investigational Site Number 380006
city: Milano
country: Italy
facility: Investigational Site Number 380005
city: Padova
country: Italy
facility: Investigational Site Number 380004
city: Roma
country: Italy
facility: Investigational Site Number 380003
city: Taipei
country: Taiwan
facility: Investigational Site Number 158001
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References: