A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00158600
• Date of registration: 08/09/2005
• Prospective Registration: No
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
• Scientific title: Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.
• Date of first enrolment: September 2005
• Target sample size: 90
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00158600
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

France, Netherlands, United States

Contacts

• Name: Medical Monitor
• Affiliation: Genzyme, a Sanofi Company

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patient must provide signed, informed consent prior to performing any study-related
procedures.

- Patient must have a diagnosis of Pompe disease based on deficient endogenous GAA
activity in cultured skin fibroblasts of less than or equal to 40% of the normal mean
of the testing laboratory and 2 confirmed GAA gene mutations;

- Patient must be greater than or equal to 8 years of age at the time of enrollment;

- Patient must be able to ambulate 40 meters (approximately 130 feet) in 6 minutes on
each test performed on two consecutive days (use of assistive devices such as a
walker, cane, or crutches, is permitted);

- Patient must have an FVC of greater than or equal to 30% and < 80% predicted in the
upright position;

- Patient must have a postural drop in FVC (liters) of at least 10% from the upright to
the supine position;

- Patient must have proximal muscle weakness in the lower limbs based on unilateral QMT
of the knee extensors defined as < 80% of the predicted value based on age, gender and
body size

- Patient must be able to tolerate pulmonary function testing (PFT) and muscle testing
in the supine position;

- Patient must have testable muscle in bilateral knee flexors and knee extensors, and
testable muscle in bilateral elbow flexors and elbow extensors;

- Patient must be able to provide reproducible muscle and pulmonary function test
results;

- Patient (and patient's legal guardian if patient is < 18 years of age) must have the
ability to comply with the clinical protocol;

- A female patient of childbearing potential must have a negative pregnancy test (urine)
at Baseline. Note: All female patients of childbearing potential and sexually mature
males must use a medically accepted method of contraception throughout the study.

Exclusion Criteria:

- Patient requires the use of invasive ventilatory support;

- Patient requires the use of noninvasive ventilatory support while awake and in an
upright position;

- Patient has received enzyme replacement therapy with GAA from any source;

- Patient has used an investigational product within 30 days prior to study enrollment,
or is currently enrolled in another study which involves clinical evaluations, unless
prior approval is given by Genzyme;

- Patient has a major congenital anomaly, medical condition, serious intercurrent
illness, or other extenuating circumstance that, in the opinion of the investigator,
may significantly interfere with study compliance, including all prescribed
evaluations and follow-up activities;



Age minimum: 8 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease (Late-onset)

Glycogen Storage Disease Type II (GSD-II)

Acid Maltase Deficiency Disease

Glycogenosis 2

Intervention(s)

Biological: alglucosidase alfa

Drug: Placebo

Primary Outcome(s)

Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC) [Time Frame: weeks 0, 12 and 52]

Summary of Patients Reporting Treatment-Emergent Adverse Events [Time Frame: weeks 0-78]

Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline [Time Frame: weeks 0, 78]

Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax) [Time Frame: weeks 0, 12, 52]

Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax) [Time Frame: weeks 0, 12, 52]

Percent of Predicted Forced Vital Capacity (FVC) [Time Frame: weeks 0, 78]

Secondary Outcome(s)

Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) [Time Frame: weeks 0, 78]

Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey [Time Frame: weeks 0, 78]

Secondary ID(s)

AGLU02704

2005-002759-42

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 23/07/2010
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00158600