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A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease Clinical Trials Information presented on Clinical Trials Search is not intended to be a substitute for qualified health advice, trips or treatment by using a genuine doctor. We aren't doctors. Always consult your mD on A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease conditions. Clinical Trials Search.org is a site committed to listing clinical research studies in human subjects. A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease Clinical research trials and A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease health trials take place in a lot of of cities across the US. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally measure the potency of new drugs. The aim of the studies / projects is to answer specific human medical questions. Clinical trials are a popular manner for physicians, government agencies, and private sector corporations to discover remedies for all kinds of circumstances, like A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease. A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease Clinical Trials and other clinical trials allow for volunteers to have health treatment alternatives before they are available to the general public. Many times the test subjects obtain treatment for without cost, and occasionally they are paid for their time. Sometimes there is a cost for a A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease clinical trial. Subjects oftentimes recieve the most effective healthcare possible for their A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease condition. Hazards are a reality, however, and could include additional or frequent doctor visits, healthcare dangers (perhaps life-threatening), 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 Pompe disease  A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease
A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease
For Condition: Glycogen storage disease type II (GSD-II),Glycogenosis 2,Acid Maltase Deficiency Disease,Pompe Disease
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 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.
Details:
Eligibility:
Study Type: Interventional,Treatment,Randomized,Open Label,Historical Control,Factorial Assignment,Safety/Efficacy Study
Minimum Age/Maximum Age: /26 Weeks
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 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.
Total Enrollment: 16
Location and Contact Information:
Duke University Medical Center
Durham, North Carolina, 27710
United States
University of Utah Medical Center
Salt Lake City, Utah, 84132
United States
University of Florida College of Medicine
Gainesville, Florida, 32610-00266
United States
Rambam Medical Center
Haifa, , 31096
Israel
Pediatrique Hopital deBrousse
Lyon, ,
France
Royal Manchester Children's Hospital
Manchester, ,
United Kingdom
National Taiwan University Hospital
Taipei, , 100
Taiwan, Province of China
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Additional Information:
Study ID Numbers: AGLU01602;
Study Start Date: April 2003
Record last reviewed: May 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00059280
Other Glycogenosis 2 Studies:
1. Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease
2. A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease)
3. A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease
4. Expanded access use of Myozyme (alglucosidase alfa) in patients with infantile-onset Pompe disease
5. A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
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A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease
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