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

Purpose

The objective of this study is to evaluate the efficacy and safety of treatment with 4000 L alglucosidase alfa (Lumizyme®) in Pompe patients.

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:

Genetics Home Reference related topics: glycogen storage disease type IX Pompe disease Schindler disease succinic semialdehyde dehydrogenase deficiency

Drug Information available for: Alglucosidase Alfa

Genetic and Rare Diseases Information Center resources: Glycogen Storage Disease Type 2

U.S. FDA Resources

Further study details as provided by Sanofi:

Primary Outcome Measures:

Percentage of participants who are clinically stable or improved at Week 52 [ Time Frame: Week 52 ] [ Designated as safety issue: No ]
Clinical stability defined as the absence of any of the following definitions of clinical worsening:
- death due to disease progression or new dependency on invasive ventilation;
- decline in cardiac status from baseline with an increase in left ventricular mass Z-score of >1 Z-score above a Z-score of 2;
- decline in motor function from baseline as measured by an absolute decrease in Gross Motor Function Measure-88 Total Percent Score of ≥8%;
- decline in pulmonary function from baseline with a decrease of ≥15% in forced vital capacity % predicted in the sitting position.

Secondary Outcome Measures:

Percent survival at Week 52 [ Time Frame: Week 52 ] [ Designated as safety issue: No ]
Kaplan-Meier methodology will be applied to estimate the percentage of patients treated with 4000 L alglucosidase alfa who are alive at Week 52.
Percentage of participants who are invasive ventilator-free at Week 52 [ Time Frame: Week 52 ] [ Designated as safety issue: No ]
Invasive ventilation is defined as mechanical ventilatory support applied with the use of an endotracheal tube or tracheostomy. Invasive ventilator dependence will be defined as the time to the first use of invasive ventilation which is not followed by an invasive ventilator-free period of at least 14 consecutive days. Kaplan-Meier methodology will be applied to estimate the percentage of patients who are alive and free of invasive ventilator dependence at Week 52.
Change from baseline on left ventricular mass Z-score (LVM-Z) [ Time Frame: Baseline and Week 52 ] [ Designated as safety issue: No ]
Change from baseline on Gross Motor Function Measure-88 (GMFM-88) Total Percent Score [ Time Frame: Baseline and Week 52 ] [ Designated as safety issue: No ]
The GMFM-88 consists of 88 items organized into 5 categories: lying and rolling, sitting, crawling and kneeling, standing, walking, running and jumping. Each item is scored on a 4-point Likert scale (i.e., 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 is expressed as a percentage of the maximum score for that dimension. The total score is obtained by adding the percentage scores for each dimension and dividing the sum by the total number of dimensions.
Change from baseline in forced vital capacity (FVC) % predicted [ Time Frame: Baseline and Week 52 ] [ Designated as safety issue: No ]
FVC % predicted in a sitting position will be measured in accordance with the American Thoracic Society guidelines.


Enrollment:	114
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	Drug: Alglucosidase alfa 4000 L alglucosidase alfa administered by intravenous 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
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

A patient must meet all of the following criteria to be eligible for this study.

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

Exclusion Criteria:

A patient who meets any of the following criteria will be excluded from this study.

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

No publications provided


Responsible Party:	Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:	NCT01526785 History of Changes
Other Study ID Numbers:	AGLU09411, EFC12720
Study First Received:	February 2, 2012
Last Updated:	January 30, 2015
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:


Glycogen Storage Disease Type II Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Genetic Diseases, Inborn	Glycogen Storage Disease Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases

ClinicalTrials.gov processed this record on March 03, 2015