Glycogen Storage Disease Type II (Pompe's Disease) MedDRA version: 9.1 Level: LLT Classification code 10036143 Term: Pompe's disease
Conditions
Interventions
Product Name: alglucosidase alfa
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: alglucosidase alfa
CAS Number: n.a.
Current Sponsor code: rhGAA
Other descriptive name: algl
Sponsors
Erasmus MC Sophia
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Patients who previously received alglucosidase alfa manufactured in a 2000 liter bioreactor or are naïve to alglucosidase alfa treatment. • Patients should have a proven diagnosis of Pompe disease, which means that the diagnosis is confirmed by deficiency of ?-glucosidase activity in leukocytes, fibroblasts or skeletal muscle, or by a documented mutation in the ?-glucosidase gene. A skin biopsy is required to assess the level of residual ?-glucosidase activity. • Patients should be = 18 years. • Female patients of childbearing potential who are naïve to alglucosidase alfa are required to have a negative pregnancy test • Written informed consent must be obtained from the patients. • Patients are also enrolled in protocol Pompe phys 02 to assess long term efficacy of alglucosidase alfa Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: all infants and children (<18 year)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of alglucosidase alfa manufactured in a 4000 liter bioreactor and to transition Dutch patients safely and gradually from alglucosidase alfa manufactured in a 2000 liter bioreactor to alglucosidase alfa manufactured in a 4000 liter bioreactor;Secondary Objective: Farmacokinetics of alglucosidase alfa produced in a 4000 liter bioreactor;Primary end point(s): No efficacy endpoints are defined as assessment of safety is the primary goal of the study. | — |
Countries
Netherlands
Outcome results
None listed