CompletedPhase 2/3

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

United States, Brazil96 participantsStarted 2003-09-18

Plain-language summary

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.

Who can participate

Age range5 Years – 25 Years

SexMALE

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Inclusion criteria

✓. 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:
✓. In addition, patients must have the following Biochemical Criteria:
✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53

Timeframe: Baseline, Week 53

Trial details

NCT IDNCT00069641

SponsorShire

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2005-03-16

Results submitted2014-01-16

View on ClinicalTrials.gov More Mucopolysaccharidosis II trials