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An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease Clinical Trials Data presented on Clinical Trials Search is not meant to be a substitute for qualified health advice, visits or treatment with a real mD. We are not doctors. Always consult your doctor about An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease Clinical research trials and An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease healthcare trials happen in many 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 generally assess the effectivity of new drugs. The purpose of the studies / projects is to solve particular human medical questions. Clinical trials are a popular way for doctors, government agencies, and private sector companies to discover cures for all varieties of conditions, such as An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease. An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease Clinical Trials and other clinical trials allow volunteers to have health treatment alternatives before they are available to the masses. Some times the human subjects obtain treatment for without cost, and sometimes they are compensated for their time. Occasionally there is a cost for a An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease clinical trial. Test subjects oftentimes receive the most effective healthcare possible for their An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease condition. Dangers are a reality, however, and may include extra or frequent physician visits, healthcare dangers (possibly life-jeopardising), and/or the treatment being uneffective. Trials are federally governed with rigorous guidelines to protect clinical trials patients.
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Home > "A" Clinical Trials Conditions > An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease  An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease
An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease
For Condition: Fabry Disease
Status: Recruiting
Sponsor(s): National Institute of Neurological Disorders and Stroke (NINDS) ,
Synopsis: This study will evaluate the safety of multiple biweekly intravenous doses of Replagal over 26 weeks in 25 children with Fabry disease and the way in which that agent can improve the health of this patient population. Fabry disease is a genetic disorder inherited as an X-linked recessive trait. It causes a deficiency in the enzyme alpha galactosidase, which normally breaks down a lipid, or fatty substance, called ceramidetrihexosidase, a building block in all cells of the body. The deficiency in breaking down the lipid eventually causes that lipid to accumulate and injure cells. Problems in the blood vessels, kidneys, heart, and nerves are the result. The disease typically occurs in childhood or adolescence, with repeated episodes of severe pain in the extremities and other symptoms. There is no definitive treatment, but pain management is important in caring for patients with Fabry disease. Although it is not known exactly how lipid accumulation brings about such problems, studies of another lipid storage disorder, Gaucher's disease, have shown that the illness can be reversed if the lipid is removed when an appropriate enzyme, Replagal, is given intravenously. In this study, the gene response of the body's cells to Fabry disease will be described, as will any gene responses that change when the enzyme is used. Patients 7 to 17 years of age who have Fabry disease may be eligible for this study. They will undergo the following tests and procedures: -Physical examination. -Neurological examination. -Vital signs. -Urinalysis. -Blood tests to determine complete blood count and chemistries. -Questionnaire on pain. -Tests pertaining to sweating. -Electrocardiogram. -Doppler blood flow study. -Diary for recording symptoms and the use of pain medications. Participants will go through the evaluation, over a period of about 5 days, either as an inpatient or outpatient. Participants will receive an intravenous infusion of Replagal every other week, at the dose of 0.2 mg/kg of body weight. Vital signs will be measured before the infusion and immediately and after and 1 hour afterward. There will be careful monitoring for allergic reactions and side effects. The infusion time takes approximately 40 minutes. This study will last 6 months, with the possibility of being extended another 6 months-a maintenance study in which patients will continue to receive Replagal at the same dose every 2 weeks.
Details: The objectives of this clinical trial are to evaluate: 1) the safety of multiple biweekly intravenous (IV) doses of Replagal over 26 weeks in 25 children ages 7-17 years old with Fabry Disease, and 2) the pharmacokinetics of Replagal in this patient population. Safety will be determined by standard clinical and laboratory measurements.
Eligibility:
Study Type: Interventional, Treatment, Safety/Efficacy
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: INCLUSION CRITERIA: Male hemizygote with Fabry Disease as documented by clinical evidence and by laboratory evidence of alpha-galactosidase A deficiency. Or Female heterozygote with Fabry Disease as documented by gene analysis showing a mutation of the alpha-galactosidase A gene. Female patients of child-bearing potential must have a negative pregnancy test at baseline and agree to the use of effective contraception such as oral contraceptive or double barrier method for study entry and while participating in the study. 7-17 years of age. Adequate general health (as determined by the investigators) to undergo the specified phlebotomy regimen and protocol related procedures. The child must assent to participate in the protocol and the parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee (IRB/IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed with the child and the child's parent(s) or legal guardian(s). EXCLUSION CRITERIA: Patient has previously participated in a multi-dose clinical study of an investigational therapeutic agent for Fabry Disease. Patient and/or the patient's parent(s) or legal guardian(s) are unable to understand the nature, scope, and possible consequences of the study. Patient is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the investigator or the medical monitor.
Total Enrollment: 25
Location and Contact Information:
National Institute of Neurological Disorders and Stroke (NINDS) *Recruiting*
Bethesda, Maryland, 20892
United States
Recruiting Patient and Public Liaison Office 1-800-411-1222
Additional Information:
Study ID Numbers: 040029; 04-N-0029
Study Start Date: October 30, 2003
Record last reviewed: October 27, 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00071877
Other Fabry Disease Studies:
1. A Study of Fabrazyme in Pediatric Patients with Fabry Disease
2. Alpha-Galactosidase A Replacement Therapy for Fabry Disease
3. PET Scans in Normal Volunteers and Patients with Fabry Disease
4. A Study of the Safety and Efficacy of Fabrazyme in Patients with Fabry Disease
5. A Study of the Safety and Efficacy of Fabrazyme in Patients with Fabry Disease
Related Studies:
Other Fabry Disease Clinical Trials
Other Maryland Clinical Trials
Other Bethesda Clinical Trials
An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years with Fabry Disease
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