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Completed

NCT Number: NCT01410890

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

* 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.

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Key information

Conditions

Pompe Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Glycogen Storage Disease Type II Glycogen Storage Disease Type II (GSD II) Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Investigational Site Number 1028, Sofia, Bulgaria

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About this study

The total study duration per participant was 4 to 8 weeks that consisted of a screening period (from 2 days to 4 weeks), treatment visit (1 day), and a follow up call (greater than or equal to 30 days). Two participants enrolled prior to protocol amendment 2 (dated 17 December 2015), which changed the study to single-dose, and were treated for 26 weeks, with a 4-week follow up.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

alglucosidase alfa

Biological

Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)

Other names: Lumizyme

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

Sponsors and collaborators

Lead sponsor

Genzyme, a Sanofi Company

Industry

Registry information

Official study title

A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

Acronym: PAPAYA

Important dates

Study start
2014
Primary completion
2020
Study completion
2020
First posted
Aug 5, 2011
Registry last updated
Mar 28, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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