Clinical trial NCT00125879: Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602
Official title: A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Glycogen Storage Disease Type II
Keywords: Glycogen Storage Disease Type II
GSD-II
Pompe Disease
Pompe Disease (Late-onset)
Acid Maltase Deficiency Disease
Glycogenosis 2
Glycogen Storage Disease Type II (GSD-II)
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2005-08-02
Enrollment: 16
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Pompe disease (also known as glycogen storage disease type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.


Eligibility criteria: Inclusion Criteria:

* The patient's legal guardian(s) must provide written informed consent prior to any study-related procedures being performed
* The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol
* The patient must have completed Protocol AGLU01602.

Exclusion Criteria:

* Patient has experienced any unmanageable adverse event (AE) in Protocol AGLU01602 due to Myozyme that would preclude continuing treatment with Myozyme

Primary outcomes:
measure: Long-term Safety and Efficacy
timeFrame: 52 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Gainesville
country: United States
facility: Shands Hospital at the University of Florida
state: Florida
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Decatur
country: United States
facility: Emory University Medical Genetics
state: Georgia
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Children's Hospital Medical Center
state: Ohio
city: Amiens
country: France
facility: CHU Amiens
city: Caen
country: France
facility: CHU Cote de Nacre
city: Mainz
country: Germany
facility: Universitats-Kinderklinik Mainz
city: Haifa
country: Israel
facility: Rambam Medical Center
city: Monza
country: Italy
facility: San Gerardo Hospital
city: Rotterdam
country: Netherlands
facility: Erasmus MC University
city: Hua-lien
country: Taiwan
facility: Tzu-Chi General Hospital
city: Tainan
country: Taiwan
facility: Chi-Mei Medical Center Dept of Pediatrics
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References:
citation: Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, Bali D, Smith SA, Li JS, Mandel H, Koeberl D, Rosenberg A, Chen YT. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab. 2010 Jan;99(1):26-33. doi: 10.1016/j.ymgme.2009.08.003.