a study on Hunter Syndrome
Study HGT-HIT-094 is a multicenter study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter syndrome and cognitive impairment who have previously received and tolerated a minimum of 4 months of therapy with Elaprase.
A Controlled, Randomized, Two-arm, Open-label, Assessor-blinded, Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Elaprase, a large molecular protein, is not expected to cross the blood brain barrier when administered intravenously. A revised formulation of idursulfase, idursulfase-IT, that differs from that of the intravenous (IV) formulation, Elaprase, has been developed to be suitable for delivery into the cerebrospinal fluid (CSF) via intrathecal administration.
Mucopolysaccharidosis II (MPS II) is a rare, X-linked, inherited disease that affects males nearly exclusively. The disease is caused by the absence of, or deficiency in, the activity of the lysosomal enzyme, iduronate-2-sulfatase (I2S) which acts to cleave O-linked sulfate moieties from the glycosaminoglycan (GAG) molecules dermatan sulfate and heparan sulfate.
Study HGT-HIT-094 is a controlled, randomized, two-arm, open-label, assessor-blinded, multicenter study to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter syndrome and cognitive impairment who have previously received and tolerated a minimum of 4 months of therapy with Elaprase.
Pediatric patients under 3 years of age will be enrolled into a separate substudy to evaluate the safety and efficacy of idursulfase-IT. The separate substudy is open label and single arm. Patients who are enrolled in the substudy will receive idursulfase-IT treatment and follow the same schedule of study visits.
Hunter Syndrome hunters disease MPS II lysosomal storage disorder hunters syndrome ert treatment hunter's disease iduronate sulfatase MPS society mps 2 enlarged adenoids hunter's syndrome iduronate 2 sulfatase chronic ear infection mucopolysaccharides hunter syndrome therapy hunter's syndrome treatment enzyme replacement therapy hunter syndrome treatment elaprase MPSII lysosomal storage disease mps diagnosis mps symptoms idursulfase hunter disease hunter's disease treatment MPS2
Open to males ages up to 18 years
Inclusion Criteria for the Pivotal Study
Patients must meet all of the following criteria to be considered eligible for randomization in the pivotal study:
The patient is male and is ≥3 and <18 years of age at the time of informed consent.
(Patients who are younger than 3 years of age may be enrolled in a separate substudy provided that they meet other inclusion criteria, provided below.)
The patient has evidence at Screening of Hunter syndrome-related cognitive impairment defined as follows:
A patient who is ≥3 and <13 years of age must have one of the following criteria (3a
If the patient has a GCA score at Screening>85, there must be evidence of a decrease in GCA score of ≥10 points over 12 months from a previously documented test result in observational study HGT-HIT-090.
A patient who is ≥13 and <18 years of age must have both of the following criteria (3c AND 3d):
The patient's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, if applicable, must be obtained prior to the start of any study procedures.
Inclusion Criteria for the Substudy
Patients must meet all of the following criteria to be considered eligible for enrollment in the separate substudy:
Patients who meet any of the following criteria are not eligible to be randomized into the pivotal study or enrolled in the separate substudy:
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