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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01410890
Other study ID # AGLU07710
Secondary ID 2010-022231-11MS
Status Completed
Phase Phase 4
First received
Last updated
Start date November 3, 2014
Est. completion date November 20, 2020

Study information

Verified date March 2022
Source Sanofi
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

- 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.


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.


Recruitment information / eligibility

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.

Study Design


Intervention

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

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  Bulgaria,  India,  Russian Federation,  Ukraine,  United Kingdom, 

Outcome

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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