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

Genzyme

Status and phase: Terminated; Phase 4

Conditions: Pompe Disease

Treatments: Drug: Alglucosidase alfa

Study type: Interventional

Funder types: Industry

Identifiers

NCT01526785

AGLU09411

EFC12720 (Other Identifier)

Details and patient eligibility

About

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

Enrollment: 113 patients

Sex: All

Ages: 1+ year old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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113 participants in 1 patient group

Alglucosidase alfa

Experimental group

Description:

Alglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.

Treatment:

Drug: Alglucosidase alfa