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

This study has been terminated.
(Terminated due to approved label expansion)
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01526785
First received: February 2, 2012
Last updated: November 9, 2015
Last verified: November 2015
  Purpose
The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.

Condition Intervention Phase
Pompe Disease
Drug: Alglucosidase alfa
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Percentage of Participants Who Were Clinically Stable or Improved at Week 52 [ Time Frame: Week 52 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Week 52 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Week 52 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, Week 52 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, Week 52 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, Week 52 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, Week 52 ] [ Designated as safety issue: No ]
    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%.


Enrollment: 113
Study Start Date: March 2012
Study Completion Date: December 2014
Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Alglucosidase alfa
Alglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.
Drug: Alglucosidase alfa
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.
Other Name: Lumizyme®

  Eligibility

Ages Eligible for Study:   1 Year and older   (Child, Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01526785

  Show 51 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT01526785     History of Changes
Other Study ID Numbers: AGLU09411  EFC12720 
Study First Received: February 2, 2012
Results First Received: September 29, 2015
Last Updated: November 9, 2015
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on September 27, 2016