Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Completed · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Mucopolysaccharidosis II

In brief

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.

Key facts

Study ID

NCT00069641

Run by

Shire

People needed

96

Starts

2003-09-18

Expected to finish

2005-03-16

Last updated by the study team

2021-06-10

Who can join

Age: 5 and older, up to 25. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• To be eligible to participate in this study, patients must meet the following inclusion criteria prior to enrollment:
• The diagnosis of MPS II will be determined by the investigator based upon both clinical and biochemical criteria.
• 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
• 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).
• Must be male, 5 to 25 years of age.
• Forced vital capacity of <80% of predicted obtained at the baseline evaluation of this study.
• Must be able to adequately perform the testing required in this study, including reproducible pulmonary function testing by spirometry, as judged by the investigator.
• 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.

You may not qualify if…

• Patients meeting any of the following criteria are not eligible for participation in this study:
• Patient has received treatment with another investigational therapy within the past 60 days.
• Patient, patient's parent(s), or patient's legal guardian is unable to understand the nature, scope, and possible consequences of the study.
• 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.
• Patient has a tracheostomy.
• Patient has received a bone marrow or cord blood transplant.
• Patient with known hypersensitivity to any of the components of iduronate-2-sulfatase.

Where it is running

• Children's Hospital Oakland — Oakland, California, United States
• St. Louis Children's Hospital, Washington University — St Louis, Missouri, United States
• University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
• Texas Children's Hospital, Baylor College of Medicine — Houston, Texas, United States
• Hospital de Clinicas de Porto Alegre — Porto Alegre, Brazil
• Children's Hospital, Johannes-Gutenburg Universitaet Mainz — Mainz, Germany
• Addenbrooke's Hospital — Cambridge, England, United Kingdom
• Great Ormond Street Hospital for Sick Children — London, England, United Kingdom
• Royal Manchester Children's Hospital — Manchester, England, United Kingdom

Full record on ClinicalTrials.gov

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