Pompe disease MedDRA version: 20.1 Level: PT Classification code 10053185 Term: Glycogen storage disease type II System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Myozyme
Pharmaceutical Form: Powder for concentrate for solution for injection/infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Current Sponsor code: GZ419829
Conce
Sponsors
Genzyme Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures; The patient must have a confirmed diagnosis of Pompe disease as determined by deficient endogenous acid alpha-glucosidase (GAA) activity or GAA mutation analysis; and The patient must be =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: The patient is participating in another clinical study using alglucosidase alfa or any investigational therapy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The overall objective is to evaluate long-term growth and development of patients with infantile-onset Pompe disease who begin treatment before 1 year of age. Patients will be followed for a 10-year period. An additional objective is to collect long-term safety data on patients with infantile-onset Pompe disease. ;Secondary Objective: Not applicable;Primary end point(s): 1/ Long-term growth and development as measured by recumbent length/height, weight and head circumference 2/ Change from baseline in motor development and function, as measured by changes in the motor subscale of the Bayley Scales of Infant and Toddler Development (Bayley-III) (up to 42 months of age) at 10 years 3/ Change from baseline in motor development and function, as measured by changes in the total score of the Gross Motor Function Measure (GMFM-88) at 10 years 4/ Change from baseline in the raw scores, normative standard scores and scaled scores for the Functional Skills Mobility and Self-Care domains of the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at 10 years 5/ Change from baseline in Cognitive Development, as measured by changes in the raw scores, scaled scores and composite scores for the cognitive and language subscales of the Bayley Scales of Infant and Toddler Development (Bayley-III) at 10 years 6/ Change from baseline in Cognitive Development, as measured by changes in raw and scaled subscale scores and composite scores and percentiles of Brief Scale IQ test of Leiter-R and/or Nonverbal IQ test of Leiter-3 (starting at 42 months of age) 7/ Summary of Adverse Events ;Timepoint(s) of evaluation of this end point: 1/ Every 3 Months for up to 10 years ] 2/ to 7/: Up to 10 years | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): NA;Timepoint(s) of evaluation of this end point: NA | — |
Countries
United States
Contacts
Public Contact-
Genzyme Corporation
Outcome results
None listed