Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease (PAPAYA)
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ClinicalTrials.gov Identifier: NCT01410890 |
Recruitment Status :
Completed
First Posted : August 5, 2011
Results First Posted : June 11, 2021
Last Update Posted : March 28, 2022
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- The primary objective of this study was to characterize the pharmacokinetics (PK) of alglucosidase alfa manufactured at the 4000 L scale in participants who had a confirmed diagnosis of Pompe disease.
- A secondary objective of this study was to evaluate and explore the relationship between anti-recombinant human acid alpha-glucosidase antibody titers and the PK of alglucosidase alfa.
Condition or disease | Intervention/treatment | Phase |
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Pompe Disease Glycogen Storage Disease Type II (GSD II) | Biological: alglucosidase alfa | Phase 4 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 21 participants |
Allocation: | N/A |
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 |
Actual Study Start Date : | November 3, 2014 |
Actual Primary Completion Date : | November 20, 2020 |
Actual Study Completion Date : | November 20, 2020 |

Arm | Intervention/treatment |
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Experimental: Alglucosidase alfa
Participants received intravenous (IV) infusion of Alglucosidase alfa 20 milligrams per kilogram (mg/kg) body weight on Day 1. Infusion was administered at an initial rate of approximately 1 milligram per kilogram per hour (mg/kg/hr) with allowed rate increased of 2 mg/kg/hr every 30 minutes, if there were no signs of infusion-associated reactions (IARs), until a maximum rate of approximately 7 mg/kg/hr was reached.
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Biological: alglucosidase alfa
Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
Other Name: Lumizyme |
- Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]Cmax was defined as maximum observed plasma concentration.
- Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]Tmax was defined as time to reach maximum observed plasma concentration.
- Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]AUC was defined as area under the plasma concentration-time curve from time 0 to 24 hours post-dose.
- Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]AUC0-last was defined as area under the concentration-time curve from time 0 to the time of the last quantifiable concentration.
- Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration.
- Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.
- Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]Volume of distribution (Vd) is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state.
- Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]Cmax was defined as maximum observed plasma concentration.
- Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]Tmax was defined as time to reach maximum observed plasma concentration.
- Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration.
- Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]AUC0-last was defined as area under the plasma concentration-time curve from time 0 to the time of the last quantifiable concentration.
- Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 and Extrapolated to Infinite Time (AUC0-inf) in Anti-rhGAA Antibody Positive and Negative Participants [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]AUC0-inf was defined as area under the concentration-time curve from time 0 extrapolated to infinite time.
- Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.
- Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants [ Time Frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 ]Vd is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state.

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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
A participant was to meet all of the following criteria to be eligible for this study:
- The participant and/or the participant's parent/legal guardian was willing and able to provide signed informed consent.
- The participant had a confirmed acid alpha-glucosidase (GAA) enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.
- Infant and toddler Pompe disease participants could be included in the study only under condition (minimal body weight) that the trial-related blood loss (including any losses in the maneuver) would not exceed 3 percent (%) of the total blood volume during a period of 4 weeks and would not exceed 1 % at any single time.
- The participant, if female and of childbearing potential, must have had a negative pregnancy test (urine beta-human chorionic gonadotropin) at screening. Note: All female participants of childbearing potential and sexually mature males must have agreed to use a medically accepted method of contraception throughout the study.
- For participants previously treated with alglucosidase alfa the participant had received alglucosidase alfa for at least 6 months.
Exclusion Criteria:
A participant who met any of the following criteria was excluded from this study:
- The participant was participating in another clinical study using an investigational product.
- The participant, in the opinion of the Investigator, was unable to adhere to the requirements of the study.
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

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
United States, New York | |
Investigational Site Number 840008 | |
Valhalla, New York, United States, 10595 | |
United States, Ohio | |
Investigational Site Number 840007 | |
Cincinnati, Ohio, United States, 45219 | |
United States, Utah | |
Investigational Site Number 840005 | |
Salt Lake City, Utah, United States, 84108 | |
United States, Virginia | |
Investigational Site Number 840003 | |
Fairfax, Virginia, United States, 22030 | |
Bulgaria | |
Investigational Site Number 1028 | |
Sofia, Bulgaria, 1113 | |
India | |
Investigational Site Number 356001 | |
New Delhi, India, 110 029 | |
Investigational Site Number 356002 | |
Vellore, India, 632004 | |
Russian Federation | |
Investigational Site Number 643001 | |
Moscow, Russian Federation, 125367 | |
Investigational Site Number 643002 | |
Moscow, Russian Federation, 125412 | |
Ukraine | |
Investigational Site Number 804001 | |
Kiev, Ukraine, 01135 | |
United Kingdom | |
Investigational Site Number 826003 | |
Birmingham, United Kingdom, B4 6NH | |
Investigational Site Number 826002 | |
Salford, United Kingdom, M6 8HD |
Study Director: | Medical Monitor | Genzyme, a Sanofi Company |
Documents provided by Sanofi ( Genzyme, a Sanofi Company ):
Responsible Party: | Genzyme, a Sanofi Company |
ClinicalTrials.gov Identifier: | NCT01410890 |
Other Study ID Numbers: |
AGLU07710 2010-022231-11 ( EudraCT Number ) MSC12790 ( Other Identifier: Sanofi ) |
First Posted: | August 5, 2011 Key Record Dates |
Results First Posted: | June 11, 2021 |
Last Update Posted: | March 28, 2022 |
Last Verified: | March 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org |
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 |