Health Condition 1: E740- Glycogen storage disease Health Condition 2: null- The patient is â�¥8 and â�¤18 years of age with confirmed acid �±-glucosidase [GAA] enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.
Conditions
Interventions
Intervention1: Alglucosidase Alfa: Eligible patients will receive an intravenous (IV) infusion of alglucosidase alfa of 20 mg/kg of body weight every other week (qow) for 26 weeks
Intervention2: alglu
Sponsors
Genzyme Corporation
SANOFISynthelabo India Limited
Eligibility
Inclusion criteria
Inclusion criteria: A patient must meet all of the following criteria to be eligible for this study. 1. The patient and/or the patientââ?¬•s parent/legal guardian is willing and able to provide signed informed consent. 2. The patient is ââ?°Â¥8 and ââ?°Â¤18 years of age with confirmed acid Ã?±-glucosidase [GAA] enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations. 3. The patient, if female and of childbearing potential, must have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: All female patients of childbearing potential and sexually mature males
Exclusion criteria
Exclusion criteria: A patient who meets any of the following criteria will be excluded from this study. 1. The patient has had previous treatment with alglucosidase alfa. 2. The patient is participating in another clinical study using an investigational product. 3. The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective of this study is to characterize the pharmacokinetics of alglucosidase alfa manufactured at the 4000 L scale in patients ranging from 8 to 18 years of age who have a confirmed diagnosis of Pompe disease.Timepoint: ââ?¬Â¢Maximum observed concentration (Cmax) [Time Frame: Day 1,wk12,wk26] ââ?¬Â¢Actual sampling time to reach maximum observed concentration (Tmax)[Time Frame:Day1,Wk12,Wk26] ââ?¬Â¢Area under the concentration-time curve from 0 to the time of the last quantifiable concentration (AUC last) ââ?¬Â¢Area under the concentration-time curve from time 0 and extrapolated to infinite time(AUC inf) ââ?¬Â¢Terminal elimination half-life(T1/2) ââ?¬Â¢Total systemic clearance (CL) ââ?¬Â¢Volume of distribution (Vd) | — |
Secondary
| Measure | Time frame |
|---|---|
| A secondary objective of this study is to evaluate the impact of immunogenicity (anti-recombinant human acid Ã?±-glucosidase [rhGAA] immunoglobulin G [IgG] titers and inhibitory/neutralizing antibodies) on the pharmacokinetics of alglucosidase alfa.Timepoint: ââ?¬Â¢ Impact of anti-rhGAA IgG antibodies on pharmacokinetic (PK) profile as measured by change in assessed PK parameters including clearance [ Time Frame: Day 1, Week 12, Week 26 ] ââ?¬Â¢ Impact of inhibitory/neutralizing antibodies on pharmacokinetic (PK) profile as measured by change in assessed PK parameters including clearance [ Time Frame: Day 1, Week 12, Week 26 ] | — |
Countries
China, India, Russian Federation, United States of America
Contacts
Public ContactMs Salini James
Genzyme India Pvt. Ltd , A sanofi Company
Outcome results
None listed