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A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-Onset Pompe Disease
This study has been completed.
Study NCT00059280   Information provided by Genzyme
First Received: April 22, 2003   Last Updated: July 6, 2009   History of Changes

April 22, 2003
July 6, 2009
April 2003
June 2005   (final data collection date for primary outcome measure)
  • Evaluate the safety profile of MZ [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • To estimate the proportion of patients treated w/ MZ who were alive and free of ventilator support at 12 months of age; compared to historical cohort [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Determine PK/PD profile of MZ [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Determine effect of different doses of MZ on safety and efficacy [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT00059280 on ClinicalTrials.gov Archive Site
 
 
 
A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-Onset Pompe Disease
An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-Onset Pompe Disease

Pompe disease (also known as glycogen storage disease type II, "GSD-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. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied.

 
Phase II, Phase III
Interventional
Treatment, Randomized, Open Label, Historical Control, Factorial Assignment, Safety/Efficacy Study
Glycogen Storage Disease Type II
Biological: Myozyme
 
 

*   Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
 
Completed
16
September 2005
June 2005   (final data collection date for primary outcome measure)

Inclusion criteria:

  • The patient or the patient's legal guardian(s) must provide written informed consent prior to any study-related procedures being performed;
  • The patient must have clinical symptoms (documented in his or her medical record) of infantile-onset Pompe disease. In addition, the patient must have: a. an endogenous GAA activity less than 1% of the mean of the normal range as assessed in cultured skin fibroblasts; AND b. cardiomyopathy (LVMI greater than 65 g/m2) by echocardiography;
  • The patient must be no older than 26 weeks and 0 days, when he/she receives the first dose of rhGAA;
  • The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol.

Exclusion criteria:

  • Symptoms of respiratory insufficiency, including: a. Oxygen saturation less than 90% in room air as measured by pulse oximetry; OR b. venous PCO2 greater than 55 mmHg on room air OR arterial PCO2 greater than 40 mmHg on room air; c. any ventilator use at the time of enrollment;
  • Major congenital abnormality;
  • Clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival;
  • Use of any investigational product within 30 days prior to study enrollment;
  • Received enzyme replacement therapy with GAA from any source.
Both
up to 26 Weeks
No
Contact information is only displayed when the study is recruiting subjects
United States,   France,   Israel,   Taiwan,   United Kingdom
 
NCT00059280
Medical Monitor, Genzyme Corporation
AGLU01602
Genzyme
 
Study Director: Medical Monitor Genzyme
Genzyme
July 2006

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP