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

Information source: Sanofi
ClinicalTrials.gov processed this data on November 27, 2014
Link to the current ClinicalTrials.gov record.

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, a Sanofi Company

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

Overall contact:
For site information, send an email with site number to, Email: Contact-Us@sanofi.com

Summary

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 nave 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.

Clinical Details

Official title: A Phase IV Prospective Study to Characterize the Pharmacokinetics of Alglucosidase 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 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

Eligibility

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.

Locations and Contacts

For site information, send an email with site number to, Email: Contact-Us@sanofi.com

Investigational Site Number 1079, Mainz 55131, Germany; Recruiting

Investigational Site Number 1024, Moscow 119991, Russian Federation; Recruiting

Investigational Site Number 1076, Vynnitsa 21000, Ukraine; Recruiting

Additional Information

Starting date: August 2013
Last updated: September 15, 2014

Page last updated: November 27, 2014

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