AGLU03306: Dose and Dose Interval Study of Myozyme®

This study is currently recruiting patients.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00483379

Purpose

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 objective of this extension study is to evaluate the safety and efficacy of alternative dosing regimens of Myozyme® in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg qow after a minimum of 6 months treatment.

Condition	Intervention	Phase
Pompe Disease Glycogen Storage Disease Type II (GSD-II) Glycogenesis 2 Acid Maltase Deficiency	Drug: Myozyme® (Alglucosidase Alfa)	Phase 4

Study Type: Interventional

Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Official Title: An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (Alglucosidase Alfa) Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen

Further Study Details:

Primary Outcome Measures:
Evaluate safety and efficacy of alternate Myozyme® dosing regimens;
Evaluate difference in efficacy in 2 dosing arms

Expected Total Enrollment: 12

Study start: 2007-05

Eligibility

Ages Eligible for Study: 6 Months and above

Genders Eligible for Study: Both

Criteria

Inclusion Criteria:

The patient or patient’s legal guardian must provide signed, informed consent prior to performing any study-related procedures;

The patient must have a clinical diagnosis of Pompe disease as defined by documented GAA deficiency in skin fibroblasts or blood;

The patient must have been compliant with the standard dosing regimen of Myozyme® (20 mg/kg qow) for a minimum of 6 months prior to study entry

The patient must have clinical decline or sub-optimal improvement in at least one of the following parameters as compared to their condition prior to the beginning Myozyme® treatment:

- Cardiac: Left Ventricular Mass (LVM) Z-score ≥6 or LVM index ≥150 g/m2 after a minimum of 6 months of treatment with Myozyme®; OR

- Respiratory: New development of respiratory failure requiring the use of ventilatory assistance (invasive or non-invasive) after a minimum of 6 months of treatment with Myozyme. Ventilatory assistance must have been required for at least 4 weeks prior to study enrollment; OR

- Motor Skills: For patients ≤ 2 years of age at study entry, failure to acquire at least 2 new gross motor milestones after a minimum of 6 months with Myozyme; OR For patients > 2 years of age at study entry, evidence of motor impairment as established using age appropriate tests after a minimum of 6 months with Myozyme®; OR For patients previously ambulatory, progression to use of an assistive device for ambulation due to worsening of proximal lower extremity muscle weakness after a minimum of 6 months of treatment with Myozyme®.

Exclusion Criteria

Any medical condition which, in the opinion of the Investigator, could interfere with treatment or evaluation of safety and/or efficacy of Myozyme®;

The patient has major congenital abnormality;

The patient has used any investigational product (other than alglucosidase alfa in those regions where the product is not commercially available) within 30 days prior to study enrollment;

The patient is pregnant or lactating.

Location and Contact Information

Medical Information 800-745-4447 medinfo@genzyme.com
Medical Information 617-252-7832 medinfo@genzyme.com

Alabama
Birmingham, Alabama, United States; Recruiting

North Carolina
Durham, North Carolina, United States; Recruiting

Canada, Alberta
Calgary, Alberta, T3B 6A8, Canada; Recruiting

More Information

Myozyme® FDA-approved labeling information

Study ID Numbers: AGLU03306
ClinicalTrials.gov Identifier: NCT00483379
Health Authority: United States: Food and Drug Administration