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A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease) Clinical Trials Information presented on Clinical Trials Search isn't designed to be a substitute for certified healthcare advice, travels to or professional assistance using a genuine medical doctor. We are not physicians. Always confer with your dr. about A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease) conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease) Clinical research trials and A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease) medical trials happen in hundreds of places across the United States. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials usually measure the effectualness of new drugs. The intention of the studies / undertakings is to solve certain human healthcare questions. Clinical trials are a popular manner for mDs, government agencies, and private sector companies to locate treatments for all forms of circumstances, such as A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease). A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease) Clinical Trials and other clinical trials allow for volunteers to undergo medical treatment choices before they are available to the general public. Some times the human subjects get treatment for free of charge, and sometimes they are paid for their time. Occasionally there is a cost for a A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease) clinical trial. Participants frequently get the best healthcare available for their A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease) condition. Risks are a reality, nonetheless, and can include extra or frequent physician trips, medical risks (possibly life-jeopardising), and/or the treatment being ineffective. Trials are federally governed with exacting guidelines to protect clinical trials subjects.
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Home > "A" Clinical Trials Conditions > A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease)  A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease)
A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease)
For Condition: Acid Maltase Deficiency Disease,Glycogen Storage Disease Type II,Pompe Disease,Glycogenosis 2
Status: No longer recruiting
Sponsor(s): Genzyme ,
Synopsis: Glycogen Storage Disease Type II ("GSD-II"; also known as Pompe disease) 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 GSD-II, 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 GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.
Details:
Eligibility:
Study Type: Interventional, Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Minimum Age/Maximum Age: 6 Months/36 Months
Genders: Both
Protocol Entry Criteria: 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 a clinical diagnosis of infantile GSD-II as defined by: (a) the patient has/had documented (in a medical record) onset of symptoms compatible with GSD-II by 12 months of age; (b) the patient has documented GAA deficiency as illustrated by an endogenous GAA activity less than or equal to 2% of the mean of the normal range as assessed in cultured skin fibroblasts; AND (c) the patient has a Left Ventricular Mass Index greater than 2 standard deviations above the mean for age - The patient is greater than 6 months old and less than or equal to 36 months old at the time of 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: - Signs and symptoms of cardiac failure and an ejection fraction less than 40% - Major congenital abnormality - Clinically significant organic disease (with the exception of symptoms relating to GSD-II), 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
Total Enrollment: 20
Location and Contact Information:
Pediatrique Hopital de Brousse
Lyon, ,
France
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Duke University Medical Center
Durham, North Carolina, 27710
United States
Rambam Medical Center
Haifa, , 31096
Israel
University of Florida College of Medicine
Gainesville, Florida, 32610
United States
Royal Manchester Children's Hospital
Manchester, , M27 4 HA
United Kingdom
Additional Information:
Study ID Numbers: AGLU01702;
Study Start Date:
Record last reviewed: May 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00053573
Other Glycogenosis 2 Studies:
1. A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease)
2. Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
3. A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
4. A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease
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A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease)
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