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

• Register: ClinicalTrials.gov
• Last refreshed on: 1 April 2013
• Main ID: NCT01410890
• Date of registration: 02/08/2011
• Primary sponsor: Genzyme
• Public title: Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8-18 Years of Age PAPAYA
• Scientific title: A Phase IV Prospective Study to Characterize the Pharmacokinetics of Alglucosidase in Patients Aged 8-18 Years of Age
• Date of first enrolment: August 2011
• Target sample size: 20
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT01410890
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label

Countries of recruitment

Germany, India, United States

Contacts

• Name: Medical Information

• Name: Medical Monitor
• Affiliation: Genzyme

Key inclusion & exclusion 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 a-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.



Age minimum: 8 Years
Age maximum: 18 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Acid Maltase Deficiency

Glycogen Storage Disease Type II (GSD II)

Glycogenesis 2

Pompe Disease (Late-Onset)

Intervention(s)

Biological: alglucosidase alfa

Primary Outcome(s)

Actual sampling time to reach maximum observed concentration (Tmax) [Time Frame: Day 1, Week 12, Week 26]

Area under the concentration-time curve from 0 to the time of the last quantifiable concentration (AUC last) [Time Frame: Day 1, Week 12, Week 26]

Area under the concentration-time curve from time 0 and extrapolated to infinite time (AUC inf) [Time Frame: Day 1, Week 12, Week 26]

Maximum observed concentration (Cmax) [Time Frame: Day 1, Week 12, Week 26]

Terminal elimination half-life (T1/2) [Time Frame: Day 1, Week 12, Week 26]

Total systemic clearance (CL) [Time Frame: Day 1, Week 12, Week 26]

Volume of distribution (Vd) [Time Frame: Day 1, Week 12, Week 26]

Secondary Outcome(s)

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]

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]

Secondary ID(s)

2010-022231-11

AGLU07710

Source(s) of Monetary Support

Please refer to primary and secondary sponsors