Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8-18 Years of Age

Condition(s) targeted: Pompe Disease (Late-Onset); Glycogen Storage Disease Type II (GSD II); Glycogenesis 2; Acid Maltase Deficiency

Intervention: alglucosidase alfa (Biological)

Phase: Phase 4

Status: Recruiting

Sponsored by: Genzyme

Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme

Overall contact:
Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com

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 and are naïve to treatment with 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.

Official title: A Phase 4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8-18 Years of Age

Study design: Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Basic Science

Primary outcome:

Maximum observed concentration (Cmax)

Actual sampling time to reach maximum observed concentration (Tmax)

Area under the concentration-time curve from 0 to the time of the last quantifiable concentration (AUC last)

Area under the concentration-time curve from time 0 and extrapolated to infinite time (AUC inf)

Terminal elimination half-life (T1/2)

Total systemic clearance (CL)

Volume of distribution (Vd)

Secondary outcome:

Impact of anti-rhGAA IgG antibodies on pharmacokinetic (PK) profile as measured by change in assessed PK parameters including clearance

Impact of inhibitory/neutralizing antibodies on pharmacokinetic (PK) profile as measured by change in assessed PK parameters including clearance

Minimum age: 8 Years. Maximum age: 18 Years. Gender(s): Both.

Criteria:

Inclusion Criteria:

A patient must meet all of the following criteria to be eligible for this study.

- The patient and/or the patient's parent/legal guardian is willing and able to provide

signed informed consent.

- The patient is ≥8 and ≤18 years of age with confirmed acid α-glucosidase [GAA] enzyme

deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.

- 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 must agree to use a medically accepted method of contraception throughout the study.

Exclusion Criteria:

A patient who meets any of the following criteria will be excluded from this study.

- The patient has had previous treatment with alglucosidase alfa.

- The patient is participating in another clinical study using an investigational

product.

- The patient, in the opinion of the Investigator, is unable to adhere to the

requirements of the study.

Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com

Cambridge, Massachusetts, United States; Recruiting

Starting date: August 2011
Last updated: January 2, 2013