Trial · NCT01410890

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

NCT01410890, Phase 4, Completed, Genzyme, a Sanofi Company; results first posted 2021-06-11

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease. Trials, record NCT01410890. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01410890

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
NCT number: NCT01410890
Status: Completed
Phases: Phase 4
Enrollment: 21 participants, actual
Lead sponsor: Genzyme, a Sanofi Company
Conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Interventions: alglucosidase alfa
First posted: 2011-08-05
Last updated: 2022-03-28
Other registries' IDs: eudract:2010-022231-11
Results first posted: 2021-06-11

Source ClinicalTrials.gov

Registered outcomes14

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa	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.
Primary	Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa	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.
Primary	Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa	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.
Primary	Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa	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.
Primary	Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa	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.
Primary	Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa	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.
Primary	Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa	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	Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants	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.
Secondary	Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants	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.
Secondary	Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants	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.

1 to 10 of 14Source ClinicalTrials.gov

See all 14 registered outcomes

Outcomes14

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on all participants who received any amount of alglucosidase alfa.	nanograms per milliliter (ng/mL)	MEAN
PRIMARY	Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on all participants who received any amount of alglucosidase alfa.	hours	MEDIAN
PRIMARY	Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on all participants who received any amount of alglucosidase alfa.	hours per nanograms per milliliter	MEAN
PRIMARY	Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on all participants who received any amount of alglucosidase alfa.	hours per nanograms per milliliter	MEAN
PRIMARY	Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on all participants who received any amount of alglucosidase alfa.	hours	MEAN
PRIMARY	Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on all participants who received any amount of alglucosidase alfa.	Liters per hour	MEAN
PRIMARY	Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on all participants who received any amount of alglucosidase alfa.	Liters	MEAN
SECONDARY	Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.	ng/mL	MEAN
SECONDARY	Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.	hours	MEDIAN
SECONDARY	Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants	Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1	Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.	hours	MEAN

1 to 10 of 14Source ClinicalTrials.gov

See all 14 outcomes

Results by group28

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa	Alglucosidase Alfa: <18 Years	204000	Standard Deviation 94600	nanograms per milliliter (ng/mL)
Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa	Alglucosidase Alfa: >=18 Years	307000	Standard Deviation 143000	nanograms per milliliter (ng/mL)
Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa	Alglucosidase Alfa: <18 Years	4.23	Full Range 1.92 to 6.42	hours
Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa	Alglucosidase Alfa: >=18 Years	3.85	Full Range 1.42 to 5.33	hours
Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa	Alglucosidase Alfa: <18 Years	1110000	Standard Deviation 753000	hours per nanograms per milliliter
Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa	Alglucosidase Alfa: >=18 Years	1890000	Standard Deviation 969000	hours per nanograms per milliliter
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa	Alglucosidase Alfa: <18 Years	1040000	Standard Deviation 590000	hours per nanograms per milliliter
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa	Alglucosidase Alfa: >=18 Years	1840000	Standard Deviation 901000	hours per nanograms per milliliter
Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa	Alglucosidase Alfa: <18 Years	5.43	Standard Deviation 3.82	hours
Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa	Alglucosidase Alfa: >=18 Years	3.84	Standard Deviation 0.801	hours

1 to 10 of 28Source ClinicalTrials.gov

See all 28 results by group

Participant flow8

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Alglucosidase Alfa: <18 Years	10
Overall Study	STARTED	Alglucosidase Alfa: >=18 Years	11
Overall Study	Treated	Alglucosidase Alfa: <18 Years	10
Overall Study	Treated	Alglucosidase Alfa: >=18 Years	10
Overall Study	COMPLETED	Alglucosidase Alfa: <18 Years	10
Overall Study	COMPLETED	Alglucosidase Alfa: >=18 Years	10
Overall Study	NOT COMPLETED	Alglucosidase Alfa: <18 Years	0
Overall Study	NOT COMPLETED	Alglucosidase Alfa: >=18 Years	1

Source ClinicalTrials.gov

Adverse events26

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Fungal Skin Infection	Infections and infestations	Alglucosidase Alfa: <18 Years	0	10	0
other	Fungal Skin Infection	Infections and infestations	Alglucosidase Alfa: >=18 Years	1	10	1
other	Nasopharyngitis	Infections and infestations	Alglucosidase Alfa: <18 Years	2	10	2
other	Nasopharyngitis	Infections and infestations	Alglucosidase Alfa: >=18 Years	0	10	0
other	Rhinitis	Infections and infestations	Alglucosidase Alfa: <18 Years	1	10	1
other	Rhinitis	Infections and infestations	Alglucosidase Alfa: >=18 Years	0	10	0
other	Papule	Skin and subcutaneous tissue disorders	Alglucosidase Alfa: <18 Years	1	10	1
other	Papule	Skin and subcutaneous tissue disorders	Alglucosidase Alfa: >=18 Years	0	10	0
other	Urticaria	Skin and subcutaneous tissue disorders	Alglucosidase Alfa: <18 Years	2	10	2
other	Urticaria	Skin and subcutaneous tissue disorders	Alglucosidase Alfa: >=18 Years	0	10	0

1 to 10 of 26Source ClinicalTrials.gov

See all 26 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Alglucosidase Alfa: <18 Years	0	10
Total, all-cause mortality	Alglucosidase Alfa: >=18 Years	0	10
Total, other adverse events	Alglucosidase Alfa: <18 Years	4	10
Total, other adverse events	Alglucosidase Alfa: >=18 Years	3	10
Total, serious adverse events	Alglucosidase Alfa: <18 Years	1	10
Total, serious adverse events	Alglucosidase Alfa: >=18 Years	0	10

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
37705424	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Alglucosidase Alfa: <18 Years vs Alglucosidase Alfa: >=18 Years: deaths from any cause	Trial comparisons
Alglucosidase Alfa: <18 Years vs Alglucosidase Alfa: >=18 Years: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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