- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00074932
Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease
February 4, 2014 updated by: Genzyme, a Sanofi Company
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 protocol is to provide enzyme replacement therapy with alglucosidase alfa on an expanded access basis, to severely affected patients with late-onset Pompe disease for whom there is no alternative treatment and who do not meet the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored studies currently enrolling patients with late-onset Pompe disease.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
9
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Colorado
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Aurora, Colorado, United States
- Colorado Health Science Center
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Kansas
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Wichita, Kansas, United States
- Galichia Heart Hospital
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Massachusetts
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Cambridge, Massachusetts, United States, 02142
- Genzyme Medical Information
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Missouri
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Joplin, Missouri, United States
- Freeman Health Systems
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New York
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Buffalo, New York, United States
- The Women's and Children's Hospital of Buffalo
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Manhasset, New York, United States
- North Shore University Hospital
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Rochester Hills, New York, United States
- Macoumb/Oakland Adult Medicine
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Virginia
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Newport News, Virginia, United States
- Riverside Regional Medical Center
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- ADULT
- OLDER_ADULT
- CHILD
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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 has/had onset of symptoms compatible with Pompe disease after 12 months of age. Age at onset of symptoms must be documented in the patient's medical record(s).
- The patient has documented GAA deficiency consistent with a diagnosis of Pompe disease, or the patient has a confirmed diagnosis of Pompe disease by documented genotype. Tissues used for determination of GAA deficiency may include blood, muscle or skin fibroblasts.
- The patient must have the following conditions: a. The patient must be wheelchair bound (unable to ambulate with the use of assistive devices, such as walker, cane, or crutches) AND b. The patient requires the use of invasive ventilation (defined as the use of any form of ventilatory support applied through an endotracheal tube).
- Female patients of childbearing potential must have a documented negative pregnancy test prior to dosing each month. In addition, all female patients of childbearing potential must use a medically accepted method of contraception throughout the program. Male patients who are sexually active must use a barrier method of contraception.
Exclusion Criteria:
- Use of any investigational product within 30 days prior to program enrollment.
- Major congenital abnormality;
- Clinically significant organic disease (with the exception of symptoms relating to late-onset 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 study or potentially decrease survival.
- The patient meets the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored treatment study currently enrolling patients with late-onset Pompe disease.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NON_RANDOMIZED
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
OTHER: 1
|
20 mg/kg
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Provide ERT with Myozyme in severly affected patients with Late-onset Pompe disease
Time Frame: 52 weeks
|
52 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
November 1, 2004
Primary Completion (ACTUAL)
August 1, 2006
Study Completion (ACTUAL)
December 1, 2006
Study Registration Dates
First Submitted
December 23, 2003
First Submitted That Met QC Criteria
December 24, 2003
First Posted (ESTIMATE)
December 25, 2003
Study Record Updates
Last Update Posted (ESTIMATE)
February 6, 2014
Last Update Submitted That Met QC Criteria
February 4, 2014
Last Verified
February 1, 2014
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Carbohydrate Metabolism, Inborn Errors
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Lysosomal Storage Diseases, Nervous System
- Disease
- Glycogen Storage Disease Type II
- Glycogen Storage Disease
Other Study ID Numbers
- AGLU02603
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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