Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

ID: NCT01410890
Status: Recruiting
Phase: Phase 4
Start Date: November 01, 2014
First Submitted: August 02, 2011
Last Updated: February 22, 2018
Results: N/A
Organization: Sanofi
Sponsors & Collaborators: Genzyme, a Sanofi Company
Location: Bulgaria, India, Russian Federation, Ukraine, United Kingdom, United States
Conditions: Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II)
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.

Study Description

Brief Summary

- The primary objective of this study is to characterize the pharmacokinetics of alglucosidase alfa manufactured at the 4000 L scale in patients who have a confirmed diagnosis of Pompe disease.

- A secondary objective of this study is to evaluate and explore the relationship between anti-rhGAA antibody titers and the pharmacokinetics of alglucosidase alfa.

Detailed Description

The total study duration per patient will be 4 to 9 weeks that consist of a screening period (from 2 days to 4 weeks), treatment visit (1 day), and a follow up call (≥30 days).
Condition or disease Intervention/treatment Phase

Glycogen Storage Disease Type II (GSD II)

Pompe Disease (Late-Onset)

Biological: alglucosidase alfa
Other Names
Lumizyme
Phase 4

Tracking Information

First Submitted DateAugust 02, 2011
Last Update Posted DateFebruary 22, 2018
Start DateNovember 01, 2014
Anticipated Completion DateAugust 01, 2018
Primary Completion DateAugust 01, 2018
Results First Submitted DateN/A
Received Results Disposit DateN/A

Current Primary Outcome Measures

  • Maximum observed concentration (Cmax) [Time Frame: Day 1]

  • Actual sampling time to reach maximum observed concentration (Tmax) [Time Frame: Day 1]

  • Area under the concentration-time curve from 0 to the time of the last quantifiable concentration (AUC last) [Time Frame: Day 1]

  • Area under the concentration-time curve from time 0 and extrapolated to infinite time (AUC inf) [Time Frame: Day 1]

  • Terminal elimination half-life (T1/2) [Time Frame: Day 1]

  • Total systemic clearance (CL) [Time Frame: Day 1]

  • Volume of distribution (Vd) [Time Frame: Day 1]

Original Primary Outcome Measures

Not Available

Current Secondary Outcome Measures

  • Pharmacokinetic (PK) parameters assessed in relation to anti-rhGAA antibody titers [Time Frame: Day 1]

  • Pharmacokinetic (PK) parameters assessed in relation to inhibitory/neutralizing anti-rhGAA antibody titers [Time Frame: Day 1]

Original Secondary Outcome Measures

Not Available

Study Design

Brief TitlePharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Official TitleA Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Brief Summary

- The primary objective of this study is to characterize the pharmacokinetics of alglucosidase alfa manufactured at the 4000 L scale in patients who have a confirmed diagnosis of Pompe disease.

- A secondary objective of this study is to evaluate and explore the relationship between anti-rhGAA antibody titers and the pharmacokinetics of alglucosidase alfa.

Detailed Description

The total study duration per patient will be 4 to 9 weeks that consist of a screening period (from 2 days to 4 weeks), treatment visit (1 day), and a follow up call (≥30 days).

Study TypeInterventional
Study PhasePhase 4
Estimated Enrollment
20
Allocation
Not Available
Interventional Model
Single Group Assignment
Masking
None (Open Label)
Primary Purpose
Other
Conditions
Glycogen Storage Disease Type II (GSD II)
Pompe Disease (Late-Onset)
Target Follow-Up Duration N/A
Biospecimen:
N/A
Sampling MethodN/A
Study PopulationN/A
Intervention
Biological: alglucosidase alfa

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

Other Names
Lumizyme
Study Groups/Cohorts
alglucosidase alfa
alglucosidase alfa intravenous (IV) infusion of 20mg/kg body weight

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

Arm Intervention/Treatment
Experimental alglucosidase alfa
alglucosidase alfa intravenous (IV) infusion of 20mg/kg body weight
Biological : alglucosidase alfa

Recruitment Information

Recruitment Status:Recruiting
Enrollment20
Completion DateAugust 01, 2018
Eligibility Criteria: Inclusion Criteria:
A patient must meet all of the following criteria to be eligible for this study:
- The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent.
- The patient has a confirmed GAA enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.
- Infant and toddler Pompe disease patients can be included in the study only under condition (minimal body weight) that the trial-related blood loss (including any losses in the maneuver) will not exceed 3 percent of the total blood volume during a period of 4 weeks and will not exceed 1 percent at any single time.
- The patient, if female and of childbearing potential, must have a negative pregnancy test (urine beta-human chorionic gonadotropin) at screening. Note: All female patients of childbearing potential and sexually mature males must agree to use a medically accepted method of contraception throughout the study.
- For patients previously treated with alglucosidase alfa the patient has received alglucosidase alfa for at least 6 months.

Exclusion Criteria:
A patient who meets any of the following criteria will be excluded from this study:
- The patient is participating in another clinical study using an investigational product.
- The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
GenderAll
Age N/A to N/A
Accepts Healthy VolunteersNo
Contacts
Listed Location Countries
Bulgaria
India
Russian Federation
Ukraine
United Kingdom
United States
Germany

Administrative Information

NCT Number:NCT01410890
Other Study ID Numbers
AGLU07710
2010-022231-11
MSC12790
Has Data Monitoring CommitteeYes
U.S. FDA-regulated Product Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Device Product Not Approved or Cleared by U.S. FDA: No
IPD Sharing Statement
Not Available
Responsible Party,
Study Sponsor
Genzyme, a Sanofi Company
Collaborators
Not Available
Investigators
Study Director
Medical Monitor
Genzyme, a Sanofi Company