Status:
COMPLETED
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
Lead Sponsor:
Shire
Collaborating Sponsors:
Covance
PharmaNet
Conditions:
Hunter Syndrome
Mucopolysaccharidosis II
Eligibility:
MALE
Up to 5 years
Phase:
PHASE4
Brief Summary
The objective of this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years old.
Detailed Description
This study will provide a basis for evaluating the safety of idursulfase administered to Hunter syndrome patients who are ≤ 5 years old. Additionally, this study will provide a basis for evaluating th...
Eligibility Criteria
Inclusion
- The patient has a diagnosis of Hunter syndrome based upon biochemical criteria either documented in their medical history or established at Screening:
- 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
- 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
- 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.
Key Trial Info
Start Date :
December 31 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 8 2011
Estimated Enrollment :
28 Patients enrolled
Trial Details
Trial ID
NCT00607386
Start Date
December 31 2007
End Date
July 8 2011
Last Update
June 8 2021
Active Locations (3)
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1
Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
2
Instytut Pomnik Centrum Zdrowia Dziecka, Klinika Chorob Metaboliczynch, Endokrynologii i Diabetologii
Warsaw, Poland, 04-730
3
National Taiwan University Hospital, Dept. of Pediatrics and Medical Genetics
Taipei, Taiwan, 10016