Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Genzyme

Status and phase

Completed

Phase 3

Phase 2

Conditions

Glycogen Storage Disease Type II

Treatments

Biological: Myozyme

Study type

Interventional

Funder types

Industry

Identifiers

NCT00125879

AGLU02403

Details and patient eligibility

About

Pompe disease (also known as glycogen storage disease type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.

Enrollment

16 patients

Sex

All

Volunteers

No Healthy Volunteers

Inclusion criteria

The patient's legal guardian(s) must provide written informed consent prior to any study-related procedures being performed
The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol
The patient must have completed Protocol AGLU01602.

Exclusion criteria

Patient has experienced any unmanageable adverse event (AE) in Protocol AGLU01602 due to Myozyme that would preclude continuing treatment with Myozyme