Pompe Disease Clinical Trial
— PAPAYAOfficial title:
A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Verified date | March 2022 |
Source | Sanofi |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
- 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.
Status | Completed |
Enrollment | 21 |
Est. completion date | November 20, 2020 |
Est. primary completion date | November 20, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Bulgaria | Investigational Site Number 1028 | Sofia | |
India | Investigational Site Number 356001 | New Delhi | |
India | Investigational Site Number 356002 | Vellore | |
Russian Federation | Investigational Site Number 643001 | Moscow | |
Russian Federation | Investigational Site Number 643002 | Moscow | |
Ukraine | Investigational Site Number 804001 | Kiev | |
United Kingdom | Investigational Site Number 826003 | Birmingham | |
United Kingdom | Investigational Site Number 826002 | Salford | |
United States | Investigational Site Number 840007 | Cincinnati | Ohio |
United States | Investigational Site Number 840003 | Fairfax | Virginia |
United States | Investigational Site Number 840005 | Salt Lake City | Utah |
United States | Investigational Site Number 840008 | Valhalla | New York |
Lead Sponsor | Collaborator |
---|---|
Genzyme, a Sanofi Company |
United States, Bulgaria, India, Russian Federation, Ukraine, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa | 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 | |
Primary | Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa | 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 | |
Primary | Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa | 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 | |
Primary | Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa | 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 | |
Primary | Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa | 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 | |
Primary | Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa | 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 | |
Primary | Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa | 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 | Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants | 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 | |
Secondary | Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants | 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 | |
Secondary | Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants | 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 | |
Secondary | 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 | 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 | |
Secondary | 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 | 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 | |
Secondary | Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants | 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 | |
Secondary | Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants | 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 |
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