A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Patients With Infantile-Onset Pompe Disease Who Have Never Been Treated - Full Text View

Purpose

A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 L and 4000 L scales in patients who have been diagnosed with infantile-onset Pompe disease. Patients will be treated with alglucosidase alfa 160 L scale product in the US and 4000 L scale product in the regions outside the US.

Condition	Intervention	Phase
Pompe Disease (Infantile-Onset) Glycogen Storage Disease Type II (GSD II) Glycogenosis 2 Acid Maltase Deficiency	Biological: alglucosidase alfa	Phase 4

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
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

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

U.S. FDA Resources

Further study details as provided by Genzyme:

Primary Outcome Measures:

Change from baseline in cardiac function as measured by the left ventricular mass Z-score(LVM-Z) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Estimated probability of survival [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Probability of invasive ventilator-free survival [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Change in motor development status as assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Number of Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs) [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	24
Study Start Date:	August 2012
Estimated Study Completion Date:	August 2017
Estimated Primary Completion Date:	July 2017 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: alglucosidase alfa (4000 L material) Non-US patients will receive alglucosidase alfa (4000 L material)	Biological: alglucosidase alfa Intravenous (IV) infusions of alglucosidase alfa (4000 L material) at a dose of 20 mg/kg of body weight every other week (qow) Other Names: Myozyme Lumizyme
Active Comparator: alglucosidase alfa (160 L material) US patients will receive alglucosidase alfa (160 L material)	Biological: alglucosidase alfa Intravenous (IV) infusions of alglucosidase alfa (160 L material) at a dose of 20 mg/kg of body weight every other week (qow) Other Name: Myozyme

Eligibility

Ages Eligible for Study:	up to 12 Months
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01597596

Contacts

Contact: Medical Information

medinfo@genzyme.com

Locations

United States, California
	Recruiting
Oakland, California, United States
United States, Illinois
	Recruiting
Chicago, Illinois, United States
United States, Massachusetts
	Recruiting
Cambridge, Massachusetts, United States
United States, Michigan
	Recruiting
Detroit, Michigan, United States
United States, Nevada
	Recruiting
Las Vegas, Nevada, United States
United States, North Carolina
	Recruiting
Durham, North Carolina, United States

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Medical Monitor

Genzyme

More Information

No publications provided

Responsible Party:	Genzyme
ClinicalTrials.gov Identifier:	NCT01597596 History of Changes
Other Study ID Numbers:	AGLU07510, 2011-005595-42
Study First Received:	May 10, 2012
Last Updated:	May 2, 2013
Health Authority:	United States: Food and Drug Administration European Union: European Medicines Agency

Additional relevant MeSH terms:

Glycogen Storage Disease Type II
Glycogen Storage Disease
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
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 16, 2013