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History of Changes for Study: NCT00158600
Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients with Late-Onset Pompe Disease.
Latest version (submitted April 6, 2015) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 September 8, 2005 None (earliest Version on record)
2 September 29, 2005 Contacts/Locations and Study Status
3 October 14, 2005 Study Status and Eligibility
4 November 10, 2005 Study Status, Study Identification, Contacts/Locations and Conditions
5 December 6, 2005 Study Status and Contacts/Locations
6 December 8, 2005 Contacts/Locations and Study Status
7 December 23, 2005 Contacts/Locations and Study Status
8 January 25, 2006 Contacts/Locations, Study Status and Oversight
9 January 31, 2006 Contacts/Locations and Study Status
10 February 10, 2006 Contacts/Locations and Study Status
11 March 9, 2006 Contacts/Locations and Study Status
12 March 26, 2006 Recruitment Status, Contacts/Locations, Study Status and Outcome Measures
13 September 20, 2006 Study Status
14 April 18, 2007 Study Status and Study Design
15 August 29, 2007 Outcome Measures, Study Status and Study Identification
16 December 14, 2007 Study Status
17 December 22, 2007 Recruitment Status, Outcome Measures, Study Status, Arms and Interventions, Study Design, Oversight and Sponsor/Collaborators
18 September 23, 2008 Study Status and Study Design
19 October 14, 2008 Outcome Measures and Study Status
20 July 7, 2009 Contacts/Locations and Study Status
21 June 24, 2010 Arms and Interventions, Study Status, Outcome Measures, Study Identification, Results, Eligibility, Conditions and Study Description
22 April 6, 2015 Study Status, Study Identification and Baseline Characteristics
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Study NCT00158600
Submitted Date:  September 8, 2005 (v1)

Open or close this module Study Identification
Unique Protocol ID: AGLU02704
Brief Title: Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients with Late-Onset Pompe Disease.
Official Title: Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients with Late-Onset Pompe Disease.
Secondary IDs:
Open or close this module Study Status
Record Verification: September 2005
Overall Status: Recruiting
Study Start: September 2005
Primary Completion:
Study Completion: December 2006
First Submitted: September 8, 2005
First Submitted that
Met QC Criteria:
September 8, 2005
First Posted: September 12, 2005 [Estimate]
Last Update Submitted that
Met QC Criteria:
September 8, 2005
Last Update Posted: September 12, 2005 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Genzyme, a Sanofi Company
Responsible Party:
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring:
Open or close this module Study Description
Brief Summary: 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 is to evaluate the safety, efficacy, and pharmacokinetics (PK) of Myozyme treatment in patients with late-onset Pompe disease as compared to placebo.
Detailed Description:
Open or close this module Conditions
Conditions: Pompe Disease (Late-onset)
Glycogen storage disease type II (GSD-II)
Acid Maltase Deficiency Disease
Glycogenosis 2
Keywords: Glycogen Storage Disease Type II
GSD-II
Pompe Disease
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 3
Interventional Study Model: Factorial Assignment
Number of Arms:
Masking: Double (masked roles unspecified)
Allocation: Randomized
Enrollment: 72
Open or close this module Arms and Interventions
Intervention Details:
Drug: Myozyme (alglucosidase alfa)
Open or close this module Outcome Measures
Primary Outcome Measures:
1. 1. to evaluate the safety profile of Myozyme
2. 2. to determine the effect of Myozyme treatment on as measured by the Six Minute Walk Test (6MWT) at Week 52
3. 3. to determine the effect of Myozyme treatment as measured by Forced Vital Capacity at Week 52
4. 4. to determine the PK profile of Myozyme
Open or close this module Eligibility
Minimum Age: 8 Years
Maximum Age:
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
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 have 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 be able to provide reproducible muscle and pulmonary function test results;
  • patient must have an FVC of greater than or equal to 30% and < 80% predicted in the upright;
  • patient must have an FEV1/FVC value of greater than or equal to 70% 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 testable muscle in bilateral knee flexors and knee extensors, and testable muscle in bilateral elbow flexors and elbow extensors;
  • patient must be able to ambulate 40 meters (approximately 130 feet) in 6 minutes on each of 2 consecutive tests performed on the same day (use of assistive devices such as a walker, cane, or crutches, is permitted);
  • patient and patient's legal guardian if patient must have the ability to comply with the clinical protocol;
  • A female patient of childbearing potential must have a negative pregnancy test 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;
  • patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities;
  • patient has a major congenital anomaly.
Open or close this module Contacts/Locations
Central Contact Person: Medical Information
Telephone: 800-745-4447
Email: medinfo@genzyme.com
Central Contact Backup: Medical Information
Telephone: 617-252-7832
Email: medinfo@genzyme.com
Study Officials: Deya Corzo, M.D.
Study Director
Genzyme, a Sanofi Company
Locations: United States, Missouri
Washington University Medical Center
[Recruiting]
St. Louis, Missouri, United States, 63110
Contact:Contact: Charlie Wulf 314-362-6980
Contact:Contact: Julaine Florence, DPT 314-362-6983
Contact:Principal Investigator: Alan Pestronk, M.D.
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links:
Available IPD/Information:

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