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A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II Clinical Trials Data presented on Clinical Trials Search isn't meant to be a substitute for qualified health advice, calls or treatment using a genuine doctor. We are not docs. Always consult your dr. 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 site 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 healthcare trials occur in a lot of of places throughout the United States. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally assess the potency of new drugs. The intent of the studies / undertakings is to figure out certain human medical questions. Clinical trials are a popular means for mDs, government agencies, and private sector corporations to locate remedies for all kinds 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 allow volunteers to obtain health treatment alternatives before they are available to the masses. Many times the participants undergo treatment for free, 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 typically obtain the most effective 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 can include extra or frequent mD trips, medical hazards (potentially life-endangering), and/or the treatment being uneffective. Trials are federally regulated with rigid guidelines to protect clinical trials patients.
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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 Acid Maltase Deficiency Disease Studies:
1. Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile 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. A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
Related Studies:
Other Acid Maltase Deficiency Disease Clinical Trials
Other New Jersey Clinical Trials
Other New Brunswick Clinical Trials
A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
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