search
Back to results

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Primary Purpose

Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2

Status
Terminated
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
alglucosidase alfa
alglucosidase alfa
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pompe Disease (Infantile-Onset)

Eligibility Criteria

undefined - 12 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • The participant's parent/legal guardian was willing and able to provide signed informed consent.
  • The participant might be less than or equal to 12 months of age.
  • The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue.
  • The participant might be naïve to treatment with alglucosidase alfa.

Exclusion Criteria:

  • The participant was cross-reactive immunologic material negative.
  • The participant required invasive ventilator support at the time of enrollment.
  • The participant had decompensated clinical heart failure.
  • The participant had a major congenital abnormality, excluding cardiac hypertrophy.
  • The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
  • The participant was currently receiving any investigational product.
  • The participant was participating in another clinical study.
  • The participant and/or the patient's parent/legal guardian was unable to adhere to the requirements of the study.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Alglucosidase Alfa 4000 L material (Non-US participants)

Alglucosidase Alfa 160 L material (US participants)

Arm Description

Alglucosidase alfa 4000 L material for 52 weeks.

Alglucosidase alfa 160 L material for 52 weeks.

Outcomes

Primary Outcome Measures

Change From Baseline in Cardiac Function at Week 52
Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.

Secondary Outcome Measures

Percentage of Participants With Estimated Probability of Survival
Number of Participants With Invasive Ventilator-Free Survival
Invasive ventilator-free survival was defined as the time during which the participant is alive and not invasively ventilated. Number of Participants with invasive ventilator-free survival were reported.
Change From Baseline in Motor Development Status at Week 52
Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item was scored on a 4-point Likert scale (0 = cannot do; 1 = initiates [<10% of the task]; 2 = partially completes [10% to <100% of the task]; 3 = task completion). The score for each dimension was expressed as a percentage of the maximum score for that dimension. Total score ranges from 0% to 100%, where higher scores indicate better motor functions.

Full Information

First Posted
May 10, 2012
Last Updated
December 14, 2015
Sponsor
Genzyme, a Sanofi Company
search

1. Study Identification

Unique Protocol Identification Number
NCT01597596
Brief Title
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Official Title
A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Study Type
Interventional

2. Study Status

Record Verification Date
December 2015
Overall Recruitment Status
Terminated
Why Stopped
Study was stopped due to approved label expansion of alglucosidase alfa.
Study Start Date
August 2012 (undefined)
Primary Completion Date
December 2014 (Actual)
Study Completion Date
December 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Alglucosidase Alfa 4000 L material (Non-US participants)
Arm Type
Experimental
Arm Description
Alglucosidase alfa 4000 L material for 52 weeks.
Arm Title
Alglucosidase Alfa 160 L material (US participants)
Arm Type
Active Comparator
Arm Description
Alglucosidase alfa 160 L material for 52 weeks.
Intervention Type
Biological
Intervention Name(s)
alglucosidase alfa
Other Intervention Name(s)
Lumizyme
Intervention Description
Intravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW)
Intervention Type
Biological
Intervention Name(s)
alglucosidase alfa
Other Intervention Name(s)
Myozyme
Intervention Description
IV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW.
Primary Outcome Measure Information:
Title
Change From Baseline in Cardiac Function at Week 52
Description
Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.
Time Frame
Baseline, Week 52
Secondary Outcome Measure Information:
Title
Percentage of Participants With Estimated Probability of Survival
Time Frame
Up to Week 52
Title
Number of Participants With Invasive Ventilator-Free Survival
Description
Invasive ventilator-free survival was defined as the time during which the participant is alive and not invasively ventilated. Number of Participants with invasive ventilator-free survival were reported.
Time Frame
Up to Week 52
Title
Change From Baseline in Motor Development Status at Week 52
Description
Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item was scored on a 4-point Likert scale (0 = cannot do; 1 = initiates [<10% of the task]; 2 = partially completes [10% to <100% of the task]; 3 = task completion). The score for each dimension was expressed as a percentage of the maximum score for that dimension. Total score ranges from 0% to 100%, where higher scores indicate better motor functions.
Time Frame
Baseline, Week 52

10. Eligibility

Sex
All
Maximum Age & Unit of Time
12 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: The participant's parent/legal guardian was willing and able to provide signed informed consent. The participant might be less than or equal to 12 months of age. The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue. The participant might be naïve to treatment with alglucosidase alfa. Exclusion Criteria: The participant was cross-reactive immunologic material negative. The participant required invasive ventilator support at the time of enrollment. The participant had decompensated clinical heart failure. The participant had a major congenital abnormality, excluding cardiac hypertrophy. The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease). The participant was currently receiving any investigational product. The participant was participating in another clinical study. The participant and/or the patient's parent/legal guardian was unable to adhere to the requirements of the study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
City
Little Rock
State/Province
Arkansas
Country
United States
City
Oakland
State/Province
California
Country
United States
City
Gainsville
State/Province
Florida
Country
United States
City
Decatur
State/Province
Georgia
Country
United States
City
Chicago
State/Province
Illinois
Country
United States
City
Cambridge
State/Province
Massachusetts
Country
United States
City
Detroit
State/Province
Michigan
Country
United States
City
Las Vegas
State/Province
Nevada
Country
United States
City
New Brunswick
State/Province
New Jersey
Country
United States
City
New York
State/Province
New York
Country
United States
City
Durham
State/Province
North Carolina
Country
United States
City
Cincinnati
State/Province
Ohio
Country
United States
City
Fort Worth
State/Province
Texas
Country
United States
City
Seattle
State/Province
Washington
Country
United States
City
Geiben
Country
Germany
City
Mainz
Country
Germany
City
Taipei
Country
Taiwan

12. IPD Sharing Statement

Learn more about this trial

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

We'll reach out to this number within 24 hrs