Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
Hunter Syndrome, Mucopolysaccharidosis II, MPS II
About this trial
This is an interventional treatment trial for Hunter Syndrome focused on measuring Hunter syndrome, hunters syndrome, hunter's syndrome, hunter disease, hunters disease, hunter's disease, MPS II, MPSII, MPS2, MPS 2, mucopolysaccharides, lysosomal storage disease, lysosomal storage disorder, chronic ear infection, enlarged adenoids, mps symptoms, mps diagnosis, mps ii therapy, MPS II treatment, ert treatment, elaprase, idursulfase, iduronate sulfatase, iduronate 2 sulfatase, enzyme replacement therapy, hunter syndrome treatment, hunter's syndrome treatment, hunter syndrome therapy, hunter's disease treatment, mps society
Eligibility Criteria
Inclusion Criteria:
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 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.
Sites / Locations
- Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
- Instytut Pomnik Centrum Zdrowia Dziecka, Klinika Chorob Metaboliczynch, Endokrynologii i Diabetologii
- National Taiwan University Hospital, Dept. of Pediatrics and Medical Genetics
Arms of the Study
Arm 1
Other
Idursulfase
Open-label treatment with idursulfase