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A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II Clinical Trials Facts presented on Clinical Trials Search is not designed to be a substitute for certified medical advice, travels to or treatment with a real dr.. We aren't doctors. Always consult your mD on A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II conditions. Clinical Trials Search.org is a website dedicated to listing clinical research studies in human subjects. A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II Clinical research trials and A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II medical trials occur in many of places across the U.S.A.. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally assess the effectiveness of new does drugs. The role of the studies / undertakings is to figure out certain human healthcare questions. Clinical trials are a popular means for doctors, government agencies, and private sector corporations to locate treatments for all forms of circumstances, including A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II. A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II Clinical Trials and other clinical trials permit volunteers to get medical treatment options before they are available to the masses. Most times the human subjects acquire 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 pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II clinical trial. Participants oftentimes recieve the finest healthcare available for their A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II condition. Dangers are a reality, nonetheless, and might include extra or frequent physician calls, health hazards (potentially life-endangering), and/or the treatment being ineffectual. Trials are federally regulated with strict guidelines to protect clinical trials subjects.
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Home > "A" Clinical Trials Conditions > A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II  A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
For Condition: Pompe Disease,Glycogen Storage Disease Type II,Glycogenosis 2,Acid Maltase Deficiency Disease
Status: Completed
Sponsor(s): Genzyme ,
Synopsis: 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, pharmacokinetics, pharmacodynamics and efficacy of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for a pair of siblings with GSD-II. To be eligible for this study, a patient must have a confirmed diagnosis of GSD-II and have a sister or brother who also has a confirmed diagnosis of GSD-II.
Details:
Eligibility:
Study Type: Interventional, Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: Inclusion Criteria: 1. Written informed consent must be obtained from the parent or guardian prior to performing any study related procedures; 2. Patient must have a clinical diagnosis of GSD-II confirmed by endogenous GAA activity below normal in at least one tissue; 3. Patient must have a sibling with a clinical diagnosis of GSD-II confirmed by an endogenous GAA activity below normal in at least one tissue, who is eligible for participation in this study; 4. Patient must have a sibling with identical GAA mutations who is eligible for participation in this study; 5. Patient must have a sibling with evidence of different progression of GSD-II who is eligible for participation in this study; 6. The patient or his/her guardian(s) must have the ability to comply with the clinical protocol. Exclusion Criteria 1. Patient has 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, would preclude participation in the trial; 2. Patient is participating in another investigational study.
Total Enrollment: 2
Location and Contact Information:
Saint Peter's University Hospital
New Brunswick, New Jersey, 08903-0591
United States
Additional Information:
Study ID Numbers: AGLU01502;
Study Start Date: January 2003
Record last reviewed: March 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00051935
Other Glycogenosis 2 Studies:
1. A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease)
2. A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
3. A study of the safety and efficacy of rhGAA in patients with infantile-onset Pompe disease
4. Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
Related Studies:
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Other New Jersey Clinical Trials
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A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
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