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AGLU03105: A Prospective, Open-Label Study of the Effect and Safety of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support.
This study has been completed.
Sponsored By:
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Genzyme
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Information Provided By:
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Genzyme
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ClinicalTrials.gov Identifier:
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NCT00268944
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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 overall objective is to evaluate the safety and efficacy of rhGAA in patients with advanced Late-onset Pompe disease.
Condition
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Intervention
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Phase
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Pompe Disease (Late-Onset)
Glycogen Storage Disease Type II (GSD-II)
Acid Maltase Deficeincy Disease
Glycogenosis 2
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Drug: Myozyme, rhGAA (alglucosidase alfa)
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2
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Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group
Assignment, Safety/Efficacy Study
Further Study Details:
Primary Outcomes:
 | Treatment effect on muscle strength and functional status. [Time Frame: six months and one year];
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| Treatment effect on pulmonary function and/or ventilation conditions. [Time Frame: six months and one year];
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| Treatment effect on cardiomyopathy noted at inclusion [Time Frame: six months and one year];
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| Treatment effect on fatigue. [Time Frame: six months and one year];
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| Treatment effect on quality of life. [Time Frame: six months and one year];
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| Treatment effect on muscular atrophy. [Time Frame: six months and one year];
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| Treatment effect on muscle glycogen. [Time Frame: six months and one year];
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| Overall patient satisfaction with treatment (visual analog scale). [Time Frame: six months and one year];
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| Pharmacodynamics assessment [Time Frame: six months and one year]. |
Total Enrollment: 5
Study Start: 2005-12; Study Completion: 2007-03
Eligibility
Ages Eligible for Study: 18 Years - N/A, Genders Eligible for Study: Both
Criteria
Inclusion Criteria:
| male or female aged greater than or equal to 18 years
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| patient’s legally authorized guardian(s) must provide signed, informed consent prior to initiation of study; patient’s signature required if patient understands informed consent
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| patient must have a documented deficit in acid alpha-glucosidase (GAA) activity , corresponding to the diagnosis of Pompe disease confirmed by documented genotyping
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| patient presents with advanced documented symptoms of the disease defined as follows:
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| patient is in a wheel chair and presents diaphragmatic dysfunction and requires invasive ventilation or non invasive ventilation (12 or more hours daily) |
Exclusion Criteria:
| patient has received enzyme replacement therapy with GAA from any source
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| patient has taken an experimental drug in the 30 days prior to study enrollment, or is currently included in another study involving clinical evaluations; If this is the case, inclusion of the patient in the present study will be subject to prior agreement by Genzyme
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| major congenital anomaly
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| clinically important organic disease (except for symptoms related to Pompe disease) or any other medical condition, serious intercurrent illness, or other extenuating circumstance that, in the physician’s opinion should preclude the patient’s participation in the study or may reduce survival
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| pregnancy and breastfeeding (women of childbearing age must use a medically accepted method of contraception throughout the entire duration of the trial. Male patients must use a medically accepted birth control method throughout the entire duration of the study) |
Location and Contact Information
Medical Information 800-745-4447 medinfo@genzyme.com
Medical Information 617-252-7832 medinfo@genzyme.com
France
Hopital Raymond Poincare, Garches, 92380, France; Recruiting
More Information
Myozyme® FDA-approved labeling information
Study ID Numbers AGLU03105
ClinicalTrials.gov Identifier NCT00268944
Health Authority: France: Afssaps - French Health Products Safety Agency
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Contact Information
Genzyme Corporation
500 Kendall Street
Cambridge, MA 02142
T:617 252 7500
F:617 252 7600
Contact Us
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