Phase 4
N=21
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Pompe Disease · Glycogen Storage Disease Type II (GSD II)
Bottom Line
View on ClinicalTrials.gov: NCT01410890 ↗Enrolled (actual)
21
Serious AEs
5.0%
Results posted
Jun 2021
Primary outcome: Primary: Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa — 204000; 307000 nanograms per milliliter (ng/mL)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- alglucosidase alfa (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Genzyme, a Sanofi Company
- Primary completion
- Nov 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa |
204000; 307000 | — |
| PRIMARY Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa |
4.23; 3.85 | — |
| PRIMARY Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa |
1110000; 1890000 | — |
| PRIMARY Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa |
1040000; 1840000 | — |
| PRIMARY Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa |
5.43; 3.84 | — |
| PRIMARY Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa |
0.529; 1.26 | — |
| PRIMARY Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa |
2.35; 5.59 | — |
| SECONDARY Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants |
256000; 262000 | — |
| SECONDARY Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants |
3.94; 4.33 | — |
| SECONDARY Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants |
4.68; 4.79 | — |
| 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 |
1410000; 1610000 | — |
| 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 |
1450000; 1700000 | — |
| SECONDARY Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants |
0.765; 1.21 | — |
| SECONDARY Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants |
5.82; 7.26 | — |
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT01410890). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.