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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT00069641 |
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Date of registration:
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29/09/2003 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
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Scientific title:
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A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II |
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Date of first enrolment:
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September 2003 |
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Target sample size:
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96 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00069641 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 2/Phase 3
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Countries of recruitment
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Brazil
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Germany
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United Kingdom
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United States
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Contacts
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Name:
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Michael Beck, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's Hospital, Johannes-Gutenburg Universitaet Mainz |
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Name:
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Edmund Wraith, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Royal Manchester Children's Hospital |
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Name:
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Ashok Vellodi, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Great Ormond Street Hospital for Sick Children |
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Name:
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Christine Eng, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Texas Children's Hospital, Baylor College of Medicine |
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Name:
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Joseph Muenzer, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of North Carolina, Chapel Hill |
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Name:
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Roberto Giugliani, MD, PhD |
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Address:
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Email:
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Affiliation:
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Hospital de Clinicas de Porto Alegre |
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Name:
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Uma Ramaswami, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Cambridge University Hospitals NHS Foundation Trust |
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Name:
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Rick A. Martin, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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St. Louis Children's Hospital, Washington University |
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Name:
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Paul Harmatz, MD |
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Address:
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Email:
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Affiliation:
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Children's Hospital & Research Center Oakland |
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Key inclusion & exclusion criteria
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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.
Age minimum:
5 Years
Age maximum:
25 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidosis II
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Intervention(s)
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Biological: Iduronate-2-sulfatase enzyme replacement therapy
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Biological: Placebo
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Primary Outcome(s)
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Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53
[Time Frame: Baseline, Week 53]
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Secondary Outcome(s)
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Mean Combined Liver and Spleen Volume at Baseline
[Time Frame: Baseline]
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Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53
[Time Frame: Baseline, Week 53]
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Change From Baseline in Mean Normalized Urine Glycosaminoglycan (GAG) Levels at Week 53
[Time Frame: Baseline, Week 53]
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Percent Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 53
[Time Frame: Baseline, Week 53]
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Mean Cardiac Left Ventricular Mass Index (LVMI) at Baseline
[Time Frame: Baseline]
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Percent Change From Baseline in Mean Combined Liver and Spleen Volume at Week 53
[Time Frame: Baseline, Week 53]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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