Trial record 1 of 1 for:    NCT01410890
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Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease (PAPAYA)

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2016 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01410890
First received: August 2, 2011
Last updated: August 11, 2016
Last verified: August 2016

August 2, 2011
August 11, 2016
November 2014
February 2018   (final data collection date for primary outcome measure)
  • Maximum observed concentration (Cmax) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Actual sampling time to reach maximum observed concentration (Tmax) [ Time Frame: Day 1 ] [ 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 ] [ 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 ] [ Designated as safety issue: No ]
  • Terminal elimination half-life (T1/2) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Total systemic clearance (CL) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Volume of distribution (Vd) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • 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 ]
Complete list of historical versions of study NCT01410890 on ClinicalTrials.gov Archive Site
  • Pharmacokinetic (PK) parameters assessed in relation to anti-rhGAA antibody titers [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Pharmacokinetic (PK) parameters assessed in relation to inhibitory/neutralizing anti-rhGAA antibody titers [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • 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 ]
Not Provided
Not Provided
 
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
  • The primary objective of this study is to characterize the pharmacokinetics of alglucosidase alfa manufactured at the 4000 L scale in patients who have a confirmed diagnosis of Pompe disease.
  • A secondary objective of this study is to evaluate and explore the relationship between anti-rhGAA antibody titers and the pharmacokinetics of alglucosidase alfa.
The total study duration per patient will be 4 to 9 weeks that consist of a screening period (from 2 days to 4 weeks), treatment visit (1 day), and a follow up call (≥30 days).
Interventional
Phase 4
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
  • Pompe Disease (Late-Onset)
  • Glycogen Storage Disease Type II (GSD II)
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
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 2018
February 2018   (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 has a confirmed GAA enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.
  • Infant and toddler Pompe disease patients can be included in the study only under condition (minimal body weight) that the trial-related blood loss (including any losses in the maneuver) will not exceed 3 percent of the total blood volume during a period of 4 weeks and will not exceed 1 percent at any single time.
  • 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.
  • For patients previously treated with alglucosidase alfa the patient has received alglucosidase alfa for at least 6 months.

Exclusion Criteria:

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

  • 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.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Both
Child, Adult, Senior
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
Bulgaria,   Germany,   Russian Federation,   Ukraine
India,   United States
 
NCT01410890
AGLU07710, 2010-022231-11, MSC12790
Yes
Not Provided
Not Provided
Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
August 2016

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