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A Prospective, Observational Study in Patients with Late-Onset Pompe Disease Clinical Trials Info presented on Clinical Trials Search isn't intended to be a substitute for qualified medical advice, visits or professional assistance by using a real mD. We are not docs. Always confer with your physician about A Prospective, Observational Study in Patients with Late-Onset Pompe Disease conditions. Clinical Trials Search.org is a website committed to listing clinical research studies in human subjects. A Prospective, Observational Study in Patients with Late-Onset Pompe Disease Clinical research trials and A Prospective, Observational Study in Patients with Late-Onset Pompe Disease health trials occur in many of cities throughout the US. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally evaluate the effectivity of new does drugs. The intent of the studies / undertakings is to resolve particular human health questions. Clinical trials are a popular way for physicians, government agencies, and private sector companies to detect remedies for all sorts of conditions, including A Prospective, Observational Study in Patients with Late-Onset Pompe Disease. A Prospective, Observational Study in Patients with Late-Onset Pompe Disease Clinical Trials and other clinical trials permit volunteers to obtain healthcare treatment alternatives before they are available to the masses. Most times the participants undergo professional assistance for without cost, and occasionally they are compensated for their time. Occasionally there is a cost for a A Prospective, Observational Study in Patients with Late-Onset Pompe Disease clinical trial. Test subjects typically receive the most expert healthcare available for their A Prospective, Observational Study in Patients with Late-Onset Pompe Disease condition. Dangers are a reality, however, and may include more or frequent mD visits, healthcare dangers (perhaps life-endangering), and/or the treatment being ineffectual. Trials are federally regulated with rigid guidelines to protect clinical trials patients.
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Home > "A" Clinical Trials Conditions > A Prospective, Observational Study in Patients with Late-Onset Pompe Disease  A Prospective, Observational Study in Patients with Late-Onset Pompe Disease
A Prospective, Observational Study in Patients with Late-Onset Pompe Disease
For Condition: Glycogen Storage Disease Type II
Status: No longer recruiting
Sponsor(s): Genzyme ,
Synopsis: 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 collect prospective, observational data on patients with late-onset Pompe disease. Approximately 60 subjects with late-onset Pompe disease will be enrolled.
Details:
Eligibility:
Study Type: Observational,Natural History,Longitudinal,Defined Population,Prospective Study
Minimum Age/Maximum Age: 8 Years/
Genders: Both
Protocol Entry Criteria: Inclusion Criteria: - The patient must provide signed, informed consent prior to performing any study-related procedures. - The patient must have a diagnosis of Pompe disease based upon: a) documented marked deficiency of GAA activity by muscle biopsy, skin fibroblasts, or leukocytes OR b) documented GAA gene mutation by deoxyribonucleic acid (DNA) analysis - The patient must be greater than 8 years of age if enrolled at a site in the U.S. and greater than 18 years of age if enrolled at a site in Europe - The patient must have documented onset of symptoms of Pompe disease after 12 months of age - The patient must have at least 3 testable muscle groups in the arms and 3 testable muscle groups in the legs using quantitative muscle testing - The patient must be able to perform pulmonary and muscle function testing in the supine position - The patient must be able to provide reproducible muscle and pulmonary function test results within 10% of each other performed on Day 1 and Day 2 of the Screening/Baseline visit and forced vital capacity measurements within 10% of each other performed in the upright position on Day 1 and Day 2 of the Screening/Baseline visit - The patient must have the ability to comply with the clinical protocol Exclusion Criteria: - The patient is unable to ambulate (use of assistive devices, such as walker, cane, crutches, is permitted); - The patient requires the use of invasive ventilatory support. - The patient requires the use of noninvasive ventilatory support during waking hours. - The patient has received enzyme replacement therapy with acid alpha-glucosidase from any source - The patient has received an investigational drug within 30 days prior to study enrollment, or is currently enrolled in another study which involves clinical evaluations - The patient has a 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 - The patient has a major congenital abnormality - For female patients only, the patient is pregnant or lactating, or is unwilling to practice birth control methods during the course of the study
Total Enrollment: 60
Location and Contact Information:
Children's Hospital Medical Center
Washington D.C., District of Columbia, 20010
United States
Children's Hospital & Regional Medical Center
Seattle, Washington, 98105
United States
School of Medicine, Campus Box 8111
St. Louis, Missouri, 63110
United States
Universitair Medisch Centrum
Utrecht, , 3584CX
Netherlands
Institut de Myologie, Groupe Hospitalier Pitie-Salpetriere, Batimant Babinski
Paris 75651, , CEDEX 13
France
Additional Information:
Study ID Numbers: AGLU02303; "LOPOS"
Study Start Date:
Record last reviewed: May 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00077662
Other Glycogen Storage Disease Type Ii Studies:
1. A Prospective, Observational Study in Patients with Late-Onset Pompe Disease
2. A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
3. Genetic and Family Studies of Inherited Muscle Diseases
4. Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
5. Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease
Related Studies:
Other Glycogen Storage Disease Type II Clinical Trials
Other Missouri Clinical Trials
Other St. Louis Clinical Trials
A Prospective, Observational Study in Patients with Late-Onset Pompe Disease
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