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Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease (PAPAYA)

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ClinicalTrials.gov Identifier: NCT01410890
Recruitment Status : Recruiting
First Posted : August 5, 2011
Last Update Posted : February 8, 2018
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:
  • 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.

Condition or disease Intervention/treatment Phase
Pompe Disease (Late-Onset) Glycogen Storage Disease Type II (GSD II) Biological: alglucosidase alfa Phase 4

Detailed Description:
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).

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Study Start Date : November 2014
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : August 2018

Arm Intervention/treatment
Experimental: alglucosidase alfa
alglucosidase alfa intravenous (IV) infusion of 20mg/kg body weight
Biological: alglucosidase alfa
Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
Other Name: Lumizyme

Primary Outcome Measures :
  1. Maximum observed concentration (Cmax) [ Time Frame: Day 1 ]
  2. Actual sampling time to reach maximum observed concentration (Tmax) [ Time Frame: Day 1 ]
  3. Area under the concentration-time curve from 0 to the time of the last quantifiable concentration (AUC last) [ Time Frame: Day 1 ]
  4. Area under the concentration-time curve from time 0 and extrapolated to infinite time (AUC inf) [ Time Frame: Day 1 ]
  5. Terminal elimination half-life (T1/2) [ Time Frame: Day 1 ]
  6. Total systemic clearance (CL) [ Time Frame: Day 1 ]
  7. Volume of distribution (Vd) [ Time Frame: Day 1 ]

Secondary Outcome Measures :
  1. Pharmacokinetic (PK) parameters assessed in relation to anti-rhGAA antibody titers [ Time Frame: Day 1 ]
  2. Pharmacokinetic (PK) parameters assessed in relation to inhibitory/neutralizing anti-rhGAA antibody titers [ Time Frame: Day 1 ]

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 screening. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01410890

Contact: For site information, send an email with site number to Contact-Us@sanofi.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com

Investigational Site Number 1028 Recruiting
Sofia, Bulgaria, 1113
Russian Federation
Investigational Site Number 643001 Active, not recruiting
Moscow, Russian Federation, 125367
Investigational Site Number 643002 Active, not recruiting
Moscow, Russian Federation, 125412
Investigational Site Number 804001 Recruiting
Kiev, Ukraine, 01135
Investigational Site Number 804002 Recruiting
Vynnitsa, Ukraine, 21000
United Kingdom
Investigational Site Number 826003 Recruiting
Birmingham, United Kingdom, B4 6NH
Investigational Site Number 826002 Recruiting
Salford, United Kingdom, M6 8HD
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT01410890     History of Changes
Other Study ID Numbers: AGLU07710
2010-022231-11 ( EudraCT Number )
MSC12790 ( Other Identifier: Sanofi )
First Posted: August 5, 2011    Key Record Dates
Last Update Posted: February 8, 2018
Last Verified: February 2018

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases