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

Genzyme

Status and phase

Terminated

Phase 4

Conditions

Pompe Disease (Infantile-Onset)

Acid Maltase Deficiency

Glycogenosis 2

Glycogen Storage Disease Type II (GSD II)

Treatments

Biological: alglucosidase alfa

Study type

Interventional

Funder types

Industry

Identifiers

NCT01597596

AGLU07510

2011-005595-42 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment

4 patients

Sex

All

Ages

Under 12 months old

Volunteers

No Healthy Volunteers

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.