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Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease Clinical Trials Facts presented on Clinical Trials Search isn't designed to be a substitute for proven healthcare advice, calls or treatment using a real mD. We aren't mDs. Always confer with your physician on Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease conditions. Clinical Trials Search.org is a website dedicated to listing clinical research studies in human subjects. Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease Clinical research trials and Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease healthcare trials happen in a lot of of localities across the United States of America. 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 / undertakings is to answer particular human medical questions. Clinical trials are a popular manner for doctors, government agencies, and private sector corporations to discover remedies for all kinds of circumstances, such as Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease. Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease Clinical Trials and other clinical trials allow volunteers to get healthcare treatment alternatives before they are available to the general public. Most times the participants receive treatment for without cost, and occasionally they are paid for their time. Sometimes there is a cost for a Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease clinical trial. Human subjects often receive the most effective healthcare possible for their Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease condition. Risks are a reality, nonetheless, and may include more or frequent dr. calls, healthcare hazards (perhaps life-threatening), and/or the treatment being ineffective. Trials are federally governed with rigorous guidelines to protect clinical trials subjects.
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Home > "E" Clinical Trials Conditions > Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease  Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease
Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease
For Condition: Glycogenosis 2,Glycogen Storage Disease Type II
Status: Suspended
Sponsor(s): Genzyme ,
Synopsis: Pompe disease (also known as glycogen storage disease Type 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. The objective of this protocol is to provide enzyme replacement therapy with alglucosidase alfa on an expanded access basis, to severely affected patients with late-onset Pompe disease for whom there is no alternative treatment and who do not meet the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored studies currently enrolling patients with late-onset Pompe disease.
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: - The patient or the patient’s legal guardian(s) must provide written informed consent prior to any study-related procedures being performed. - The patient has/had onset of symptoms compatible with Pompe disease after 12 months of age. Age at onset of symptoms must be documented in the patient’s medical record(s). - The patient has documented GAA deficiency consistent with a diagnosis of Pompe disease, or the patient has a confirmed diagnosis of Pompe disease by documented genotype. Tissues used for determination of GAA deficiency may include blood, muscle or skin fibroblasts. - The patient must have the following conditions: a. The patient must be wheelchair bound (unable to ambulate with the use of assistive devices, such as walker, cane, or crutches) AND b. The patient requires the use of invasive ventilation (defined as the use of any form of ventilatory support applied through an endotracheal tube). - Female patients of childbearing potential must have a documented negative pregnancy test prior to dosing each month. In addition, all female patients of childbearing potential must use a medically accepted method of contraception throughout the program. Male patients who are sexually active must use a barrier method of contraception. Exclusion Criteria - Use of any investigational product within 30 days prior to program enrollment. - Major congenital abnormality; - Clinically significant organic disease (with the exception of symptoms relating to late-onset 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 study or potentially decrease survival. - The patient meets the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored treatment study currently enrolling patients with late-onset Pompe disease.
Total Enrollment:
Location and Contact Information:
Genzyme Medical Information
Cambridge, Massachusetts, 02142
United States
Additional Information:
Study ID Numbers: AGLU02603;
Study Start Date: December 2003
Record last reviewed: April 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00074932
Other Glycogenosis 2 Studies:
1. A study of the safety and pharmacokinetics of rhGAA in siblings with Glycogen Storage Disease Type II
2. A study of the safety and efficacy of rhGAA in patients with infantile-onset GSD-II (Pompe disease)
3. Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
4. Expanded access use of Myozyme (alglucosidase alfa) in patients with infantile-onset Pompe disease
5. Genetic and Family Studies of Inherited Muscle Diseases
Related Studies:
Other Glycogenosis 2 Clinical Trials
Other Massachusetts Clinical Trials
Other Cambridge Clinical Trials
Expanded Access Use of Myozyme (alglucosidase alfa) in Patients with Late-onset Pompe disease
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