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

March 15, 2022 updated by: Genzyme, a Sanofi Company

A Phase 3/4 Prospective Study to Characterize the 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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
21

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Bulgaria
      • Sofia, Bulgaria, 1113
        • Investigational Site Number 1028
  • India
      • New Delhi, India, 110 029
        • Investigational Site Number 356001
      • Vellore, India, 632004
        • Investigational Site Number 356002
  • Russian Federation
      • Moscow, Russian Federation, 125367
        • Investigational Site Number 643001
      • Moscow, Russian Federation, 125412
        • Investigational Site Number 643002
  • Ukraine
      • Kiev, Ukraine, 01135
        • Investigational Site Number 804001
  • United Kingdom
      • Birmingham, United Kingdom, B4 6NH
        • Investigational Site Number 826003
      • Salford, United Kingdom, M6 8HD
        • Investigational Site Number 826002
  • United States
    • New York
      • Valhalla, New York, United States, 10595
        • Investigational Site Number 840008
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • Investigational Site Number 840007
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • Investigational Site Number 840005
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • Investigational Site Number 840003

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: OTHER
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.
Biological: alglucosidase alfa
Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
Other Names:
  • Lumizyme

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
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.
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 3, 2014

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 20, 2020

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 20, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 2, 2011

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 4, 2011

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 5, 2011

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 28, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 15, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AGLU07710
  • 2010-022231-11 (EUDRACT_NUMBER)
  • MSC12790 (OTHER: Sanofi)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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