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Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) Clinical Trials References presented on Clinical Trials Search isn't meant to be a substitute for proven healthcare advice, trips or professional assistance using a genuine physician. We are not docs. Always confer with your physician about Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) Clinical research trials and Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) healthcare trials happen in hundreds of localities throughout 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 usually evaluate the potency of new drugs. The propose of the studies / projects is to answer particular human health questions. Clinical trials are a popular way for mDs, government agencies, and private sector companies to detect cures for all sorts of conditions, such as Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II). Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) Clinical Trials and other clinical trials allow volunteers to acquire healthcare treatment choices before they are available to the general public. Some times the subjects recieve professional assistance for free, and every now and again they are compensated for their time. Sometimes there is a cost for a Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) clinical trial. Subjects frequently obtain the most expert healthcare possible for their Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) condition. Risks are a reality, nevertheless, and can include more or frequent doctor trips, medical risks (possibly life-threatening), and/or the treatment being uneffective. Trials are federally governed with stern guidelines to protect clinical trials patients.
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Home > "I" Clinical Trials Conditions > Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)  Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
For Condition: Mucopolysaccharidosis II
Status: No longer recruiting
Sponsor(s): Transkaryotic Therapies ,
Synopsis: The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II.
Details: MPS II is a rare, X-linked, lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase. Because of this deficiency, glycosaminoglycans (GAG) accumulate in multiple tissues and organs, resulting in progressive cellular and organ system dysfunction. The purpose of this study is to determine if one year of therapy with iduronate-2-sulfatase enzyme replacement therapy, at a dose of 0.5mg/kg, weekly or every other week, is safe, and results in clinically meaningful improvement in multiple organ function, compared with a placebo group. Upon completion of the study, patients will be eligible to enroll in an open-label maintenance study.
Eligibility:
Study Type: Interventional, Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study
Minimum Age/Maximum Age: 5 Years/25 Years
Genders: Male
Protocol Entry Criteria: Inclusion Criteria: To be eligible to participate in this study, patients must meet the following inclusion criteria prior to enrollment: 1. The diagnosis of MPS II will be determined by the investigator based upon both clinical and biochemical criteria. 2. All patients must have at least one of the following Clinical Criteria considered by the investigator to be MPS II-related: - Hepatosplenomegaly - Radiographic evidence of dysostosis multiplex - Valvular heart disease - Evidence of obstructive pulmonary disease 3. In addition, patients must have the following Biochemical Criteria: - Documented deficiency in iduronate-2-sulfastase enzyme activity of less than or equal to 10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on normal range of measuring laboratory). - A normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on normal range of measuring laboratory). 4. Must be male, 5 to 25 years of age. 5. Forced vital capacity of <80% of predicted obtained at the baseline evaluation of this study. 6. Must be able to adequately perform the testing required in this study, including reproducible pulmonary function testing by spirometry, as judged by the investigator. 7. Patient, patient’s parent(s), or legally authorized guardian must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. Exclusion Criteria: Patients meeting any of the following criteria are not eligible for participation in this study: 1. Patient has received treatment with another investigational therapy within the past 60 days. 2. Patient, patient’s parent(s), or patient’s legal guardian is unable to understand the nature, scope, and possible consequences of the study. 3. Patient is unable to comply with the protocol (e.g., due to a medical condition such as cervical cord compression or uncooperative attitude) or is unlikely to complete the study, as determined by the investigator. 4. Patient has a tracheostomy. 5. Patient has received a bone marrow or cord blood transplant. 6. Patient with known hypersensitivity to any of the components of iduronate-2-sulfatase.
Total Enrollment:
Location and Contact Information:
Hospital de Clinicas de Porto Alegre
Porto Alegre, ,
Brazil
Addenbrooke's Hospital
Cambridge, England, CB2 2QQ
United Kingdom
Texas Children's Hospital, Baylor College of Medicine
Houston, Texas, 77030
United States
Children's Hospital, Johannes-Gutenburg Universitaet Mainz
Mainz, ,
Germany
St. Louis Children's Hospital, Washington University
St. Louis, Missouri, 63110
United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
United States
Royal Manchester Children's Hospital
Manchester, England, M27 4HA
United Kingdom
Great Ormond Street Hospital for Sick Children
London, England, WC1N3JH
United Kingdom
Children's Hospital Oakland
Oakland, California, 94609
United States
Additional Information:
Study ID Numbers: TKT024;
Study Start Date: September 2003
Record last reviewed: February 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00069641
Other Mucopolysaccharidosis Ii Studies:
1. Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
2. Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene into Lymphocytes of Patients with Mucopolysaccharidosis II (Mild Hunter Syndrome)
Related Studies:
Other Mucopolysaccharidosis II Clinical Trials
Other England Clinical Trials
Other Manchester Clinical Trials
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
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