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A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

Primary Purpose

Pompe Disease

Status
Terminated
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
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

Eligibility Criteria

1 Year - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

A participant might meet all of the following criteria to be eligible for this study.

  1. The participant and/or their parent/legal guardian was willing and able to provide signed informed consent.
  2. The participant might be at least 1 year of age at the time of informed consent.
  3. The participant had a diagnosis of Pompe disease and might have received treatment with 160 L alglucosidase alfa prior to screening.
  4. The participant, if female and of childbearing potential, might have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: all female participants of childbearing potential and sexually mature males might agree to use a medically accepted method of contraception throughout the study.

Exclusion Criteria:

A participant who met any of the following criteria were to be excluded from this study.

  1. The participant had within the past 3 months received or was currently receiving any investigational product other than 160 L alglucosidase alfa or was currently participating in another clinical treatment study.
  2. The participant, in the opinion of the Investigator, was clinically unstable and would not be expected to survive to completion of the 52-week treatment period.
  3. The participant and/or their parent/legal guardian, in the opinion of the Investigator, was unable to adhere to the requirements of the study.

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Alglucosidase alfa

Arm Description

Alglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.

Outcomes

Primary Outcome Measures

Percentage of Participants Who Were Clinically Stable or Improved at Week 52
Clinical stability was defined as absence of death due to disease progression or new dependency on invasive ventilation and; decline in cardiac status, motor function, and pulmonary function from baseline.

Secondary Outcome Measures

Survival Rate at Week 52
Percentage of participants who were alive at Week 52, were reported. Survival rate was calculated by Kaplan-Meier estimate.
Invasive Ventilator-Free Survival Rate at Week 52
Percentage of participants, who were invasive ventilator-free at week 52, are reported. Invasive ventilation was defined as mechanical ventilatory support applied with the use of an endotracheal tube or tracheostomy. Invasive ventilator-free survival rate was calculated by Kaplan-Meier estimate.
Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52
Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative 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.
Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52
GMFM-88 (88-item measure to detect gross motor function) consists of 5 components, each measured on a 4-point Likert scale.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.
Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position
Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.
Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Sitting Position
Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.

Full Information

First Posted
February 2, 2012
Last Updated
November 9, 2015
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT01526785
Brief Title
A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
Official Title
A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale
Study Type
Interventional

2. Study Status

Record Verification Date
November 2015
Overall Recruitment Status
Terminated
Why Stopped
Terminated due to approved label expansion
Study Start Date
March 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
The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
113 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Alglucosidase alfa
Arm Type
Experimental
Arm Description
Alglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.
Intervention Type
Drug
Intervention Name(s)
Alglucosidase alfa
Other Intervention Name(s)
Lumizyme®
Intervention Description
4000 L alglucosidase alfa administered by IV infusion at the same dose and dose regimen used for the patient's routine treatment prior to the study for 52 weeks.
Primary Outcome Measure Information:
Title
Percentage of Participants Who Were Clinically Stable or Improved at Week 52
Description
Clinical stability was defined as absence of death due to disease progression or new dependency on invasive ventilation and; decline in cardiac status, motor function, and pulmonary function from baseline.
Time Frame
Week 52
Secondary Outcome Measure Information:
Title
Survival Rate at Week 52
Description
Percentage of participants who were alive at Week 52, were reported. Survival rate was calculated by Kaplan-Meier estimate.
Time Frame
Week 52
Title
Invasive Ventilator-Free Survival Rate at Week 52
Description
Percentage of participants, who were invasive ventilator-free at week 52, are reported. Invasive ventilation was defined as mechanical ventilatory support applied with the use of an endotracheal tube or tracheostomy. Invasive ventilator-free survival rate was calculated by Kaplan-Meier estimate.
Time Frame
Week 52
Title
Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52
Description
Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative 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
Title
Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52
Description
GMFM-88 (88-item measure to detect gross motor function) consists of 5 components, each measured on a 4-point Likert scale.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
Title
Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position
Description
Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.
Time Frame
Baseline, Week 52
Title
Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Sitting Position
Description
Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.
Time Frame
Baseline, Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: A participant might meet all of the following criteria to be eligible for this study. The participant and/or their parent/legal guardian was willing and able to provide signed informed consent. The participant might be at least 1 year of age at the time of informed consent. The participant had a diagnosis of Pompe disease and might have received treatment with 160 L alglucosidase alfa prior to screening. The participant, if female and of childbearing potential, might have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: all female participants of childbearing potential and sexually mature males might agree to use a medically accepted method of contraception throughout the study. Exclusion Criteria: A participant who met any of the following criteria were to be excluded from this study. The participant had within the past 3 months received or was currently receiving any investigational product other than 160 L alglucosidase alfa or was currently participating in another clinical treatment study. The participant, in the opinion of the Investigator, was clinically unstable and would not be expected to survive to completion of the 52-week treatment period. The participant and/or their parent/legal guardian, in the opinion of the Investigator, 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
Birmingham
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Alabama
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Phoenix
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Little Rock
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Miami
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Louisville
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Rochester
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Syracuse
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Valhalla
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Asheville
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Providence
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Norfolk
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Morgantown
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Miliwaukee
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United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
36214004
Citation
Duong T, Kishnani PS, An Haack K, Foster MC, Gibson JB, Wilson C, Hahn SH, Hillman R, Kronn D, Leslie ND, Pena LDM, Sparks SE, Stockton DW, Tanpaiboon P, Day JW; Pompe ADVANCE Study Consortium. Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort. J Neuromuscul Dis. 2022;9(6):713-730. doi: 10.3233/JND-210784.
Results Reference
derived
PubMed Identifier
34420548
Citation
Byrne BJ, Colan SD, Kishnani PS, Foster MC, Sparks SE, Gibson JB, An Haack K, Stockton DW, Pena LDM, Hahn SH, Johnson J, Tanpaiboon PX, Leslie ND, Kronn D, Hillman RE, Wang RY; Pompe ADVANCE Study Consortium. Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort. Cardiol Young. 2022 Mar;32(3):364-373. doi: 10.1017/S1047951121002079. Epub 2021 Aug 23.
Results Reference
derived
PubMed Identifier
31086307
Citation
Kishnani PS, Gibson JB, Gambello MJ, Hillman R, Stockton DW, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Day JW, Wang RY, Goldstein JL, An Haack K, Sparks SE, Zhao Y, Hahn SH; Pompe ADVANCE Study Consortium. Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease. Genet Med. 2019 Nov;21(11):2543-2551. doi: 10.1038/s41436-019-0527-9. Epub 2019 May 14.
Results Reference
derived
PubMed Identifier
29565424
Citation
Hahn SH, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Gambello MJ, Gibson JB, Hillman R, Stockton DW, Day JW, Wang RY, An Haack K, Shafi R, Sparks S, Zhao Y, Wilson C, Kishnani PS; Pompe ADVANCE Study Consortium. Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study. Genet Med. 2018 Oct;20(10):1284-1294. doi: 10.1038/gim.2018.2. Epub 2018 Mar 22.
Results Reference
derived

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A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

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