Trial record 1 of 1 for:    NCT01410890
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Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8-18 Years of Age (PAPAYA)

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01410890
First received: August 2, 2011
Last updated: September 15, 2014
Last verified: September 2014

August 2, 2011
September 15, 2014
August 2013
February 2017   (final data collection date for primary outcome measure)
  • Maximum observed concentration (Cmax) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
  • Actual sampling time to reach maximum observed concentration (Tmax) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Area under the concentration-time curve from time 0 and extrapolated to infinite time (AUC inf) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
  • Terminal elimination half-life (T1/2) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
  • Total systemic clearance (CL) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
  • Volume of distribution (Vd) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT01410890 on ClinicalTrials.gov Archive Site
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8-18 Years of Age
A Phase IV Prospective Study to Characterize the Pharmacokinetics of Alglucosidase in Patients Aged 8-18 Years of Age

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.

Not Provided
Interventional
Phase 4
Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
  • Pompe Disease (Late-Onset)
  • Glycogen Storage Disease Type II (GSD II)
  • Glycogenesis 2
  • Acid Maltase Deficiency
Biological: alglucosidase alfa
Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
Other Name: Lumizyme
Experimental: alglucosidase alfa
alglucosidase alfa intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
Intervention: Biological: alglucosidase alfa
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
20
February 2017
February 2017   (final data collection date for primary outcome measure)

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.
Both
8 Years to 18 Years
No
Contact: For site information, send an email with site number to Contact-Us@sanofi.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com
Germany,   Russian Federation,   Ukraine
 
NCT01410890
AGLU07710, 2010-022231-11, MSC12790
Yes
Sanofi ( Genzyme, a Sanofi Company )
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
September 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP