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

NCT ID: NCT01597596

Last Updated: 2016-01-18

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-08-31
• Study Completion Date: 2014-12-31

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.

Conditions

Pompe Disease (Infantile-Onset); Glycogen Storage Disease Type II (GSD II); Glycogenosis 2; Acid Maltase Deficiency

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Alglucosidase Alfa 4000 L material (Non-US participants)

Alglucosidase alfa 4000 L material for 52 weeks.

Group Type: EXPERIMENTAL

alglucosidase alfa

Intervention Type: BIOLOGICAL

Intravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW)

Alglucosidase Alfa 160 L material (US participants)

Alglucosidase alfa 160 L material for 52 weeks.

Group Type: ACTIVE_COMPARATOR

alglucosidase alfa

Intervention Type: BIOLOGICAL

IV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW.

Interventions

alglucosidase alfa

Intravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW)

Intervention Type: BIOLOGICAL

alglucosidase alfa

IV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW.

Intervention Type: BIOLOGICAL

Other Intervention Names

Lumizyme; Myozyme

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.

• Maximum Eligible Age: 12 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

Little Rock, Arkansas, United States

Oakland, California, United States

Gainsville, Florida, United States

Decatur, Georgia, United States

Chicago, Illinois, United States

Cambridge, Massachusetts, United States

Detroit, Michigan, United States

Las Vegas, Nevada, United States

New Brunswick, New Jersey, United States

New York, New York, United States

Durham, North Carolina, United States

Cincinnati, Ohio, United States

Fort Worth, Texas, United States

Seattle, Washington, United States

Geiben, , Germany

Mainz, , Germany

Taipei, , Taiwan

Countries

United States; Germany; Taiwan

Other Identifiers

2011-005595-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

AGLU07510

Identifier Type: -

Identifier Source: org_study_id