---
title: Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
nct_id: NCT01410890
overall_status: COMPLETED
phase: PHASE4
sponsor: Genzyme, a Sanofi Company
study_type: INTERVENTIONAL
primary_condition: Pompe Disease
countries: United States, Bulgaria, India, Russia, Ukraine, United Kingdom
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT01410890.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT01410890"
ct_last_update_post_date: 2022-03-28
last_seen_at: "2026-05-12T06:05:46.785Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

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

**NCT ID:** [NCT01410890](https://clinicaltrials.gov/study/NCT01410890)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE4
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 21
- **Lead Sponsor:** Genzyme, a Sanofi Company
- **Conditions:** Pompe Disease, Glycogen Storage Disease Type II (GSD II)
- **Start Date:** 2014-11-03
- **Completion Date:** 2020-11-20
- **CT.gov Last Update:** 2022-03-28

## Brief 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.

## 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.

## Eligibility

- **Sex:** ALL
- **Healthy Volunteers:** No

```
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.
```

## Arms

- **Alglucosidase alfa** (EXPERIMENTAL) — 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.

## Interventions

- **alglucosidase alfa** (BIOLOGICAL) — Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)

## Primary Outcomes

- **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.
- **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.
- **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.
- **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.
- **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.
- **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.
- **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

- **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)_
- **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)_
- **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)_
- **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)_
- **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)_
- **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)_
- **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)_

## Locations (12)

- Investigational Site Number 840008, Valhalla, New York, United States
- Investigational Site Number 840007, Cincinnati, Ohio, United States
- Investigational Site Number 840005, Salt Lake City, Utah, United States
- Investigational Site Number 840003, Fairfax, Virginia, United States
- Investigational Site Number 1028, Sofia, Bulgaria
- Investigational Site Number 356001, New Delhi, India
- Investigational Site Number 356002, Vellore, India
- Investigational Site Number 643001, Moscow, Russia
- Investigational Site Number 643002, Moscow, Russia
- Investigational Site Number 804001, Kiev, Ukraine
- Investigational Site Number 826003, Birmingham, United Kingdom
- Investigational Site Number 826002, Salford, United Kingdom

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.investigational site number 840008|valhalla|new york|united states` — added _(2026-05-12)_
- `locations.investigational site number 840007|cincinnati|ohio|united states` — added _(2026-05-12)_
- `locations.investigational site number 840005|salt lake city|utah|united states` — added _(2026-05-12)_
- `locations.investigational site number 840003|fairfax|virginia|united states` — added _(2026-05-12)_
- `locations.investigational site number 1028|sofia||bulgaria` — added _(2026-05-12)_
- `locations.investigational site number 356001|new delhi||india` — added _(2026-05-12)_
- `locations.investigational site number 356002|vellore||india` — added _(2026-05-12)_
- `locations.investigational site number 643001|moscow||russia` — added _(2026-05-12)_
- `locations.investigational site number 643002|moscow||russia` — added _(2026-05-12)_
- `locations.investigational site number 804001|kiev||ukraine` — added _(2026-05-12)_
- `locations.investigational site number 826003|birmingham||united kingdom` — added _(2026-05-12)_
- `locations.investigational site number 826002|salford||united kingdom` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT01410890.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT01410890*  
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