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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: |
NCT00607386 |
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Date of registration:
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22/01/2008 |
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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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Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
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Scientific title:
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A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy |
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Date of first enrolment:
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December 2007 |
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Target sample size:
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28 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00607386 |
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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 4
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Countries of recruitment
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Brazil
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Poland
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Taiwan
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Contacts
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Name:
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Anna Tylki-Szymanska, 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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Instytut Pomnik Centrum Zdrowia Dziecka |
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Name:
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Roberto Giugliani, 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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Hospital de Clinicas de Porto Alegre |
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Name:
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Arian Pano, MD, MPH |
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Address:
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Telephone:
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Email:
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Affiliation:
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Shire Human Genetic Therapies, Inc. |
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Name:
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Wuh-Liang Hwu, 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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National Taiwan University Hospital |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- The patient has a diagnosis of Hunter syndrome based upon biochemical criteria either
documented in their medical history or established at Screening:
1. A deficiency in iduronate-2-sulfatase (I2S) enzyme activity of = 10 % of the
lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes
(based on normal range of measuring laboratory)
AND
2. A normal enzyme activity level of one other sulfatase as measured in plasma,
fibroblasts, or leukocytes (based on normal range of measuring laboratory).
- The patient is 5 years of age and under.
- The patient is male.
- The patient's parent(s), or patient's legal guardian must have voluntarily signed an
Institutional Review Board approved informed consent form after all relevant aspects
of the study have been explained and discussed with the patient's parent(s), or the
patient's legal guardian.
Exclusion Criteria:
- The patient has received treatment with another investigational therapy within 30 days
prior to enrollment.
- The patient has clinically relevant medical condition(s) making implementation of the
protocol difficult.
- The patient has previously received idursulfase.
- The patient has known hypersensitivity to any of the components of idursulfase.
- The patient has had a tracheostomy.
Age minimum:
N/A
Age maximum:
5 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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MPS II
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Mucopolysaccharidosis II
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Hunter Syndrome
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Intervention(s)
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Biological: Idursulfase
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Primary Outcome(s)
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Safety Evaluation
[Time Frame: From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks]
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Secondary Outcome(s)
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Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to the Final Time Point With a Concentration of at Least Lower Limit of Quantitation (AUClast)
[Time Frame: Weeks 1 and 27]
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Single- and Repeat-Dose Pharmacokinetics - Clearance (CL)
[Time Frame: Weeks 1 and 27]
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Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to Infinity (AUCinf)
[Time Frame: Weeks 1 and 27]
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Single- and Repeat-Dose Pharmacokinetics - Volume of Distribution at Steady State (Vss)
[Time Frame: Weeks 1 and 27]
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Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels
[Time Frame: Baseline, Weeks 18, 36 and 53]
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Single- and Repeat-Dose Pharmacokinetics - Elimination Half-Life (t1/2)
[Time Frame: Weeks 1 and 27]
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Single- and Repeat-Dose Pharmacokinetics - Mean Residence Time From Time 0 to Infinity (MRTinf)
[Time Frame: Weeks 1 and 27]
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Single- and Repeat-Dose Pharmacokinetics - Time of Maximum Observed Serum Concentration (Tmax)
[Time Frame: Weeks 1 and 27]
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Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax)
[Time Frame: Weeks 1 and 27]
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Secondary ID(s)
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HGT-ELA-038
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2007-006044-22
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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