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.

Shire HGT Inc0 个研究点目标入组 49 人开始时间: 2013年9月11日最近更新: 2019年6月30日

适应症

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方
入组人数: 49

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional clinical trial of medicinal product

入排标准

入选标准

•Inclusion Criteria for the Pivotal Study:
•- The patient is male and is =3 and <18 years of age at the time of informed consent.
•- The patient must have a documented diagnosis of MPS II. Of the three criteria below, the combinations (2a AND 2b) or (2a AND 2c) will be accepted as diagnostic of MPS II:
•a. The patient has a deficiency in iduronate-2-sulfatase enzyme activity of =10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory’s normal range).
•b.The patient has a documented mutation in the iduronate-2-sulfatase gene that leaves the FMR1 and FMR2 genes intact.
•c.The patient has a normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on the normal range of measuring laboratory).
•- 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 OR 3b):
•a. A GCA score =55 and =85
•b. 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):
•a. A GCA score of =55 and =85.
•b. 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.
•- The patient has received and tolerated a minimum of 4 months of therapy with Elaprase during the period immediately prior to Screening.
•- The patient must have sufficient auditory capacity, with a hearing aid(s), if needed, in the Investigator’s judgment to complete the required protocol testing and must be compliant with wearing the hearing aid(s), if needed, on scheduled testing days.
•Inclusion criteria for the sub-study:
•- The patient is male and is <3 years of age at the time of informed consent.
•- The patient must have a documented diagnosis of MPS II. Of the three criteria below, the combinations (2a AND 2b) or (2a AND 2c) will be accepted as diagnostic of MPS II:
•a. The patient has a deficiency in iduronate-2-sulfatase enzyme activity of =10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory’s normal range).
•b. The patient has a documented mutation in the iduronate-2-sulfatase gene that leaves the FMR1 and FMR2 genes intact.
•c. The patient has a normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on the normal range of measuring laboratory).
•- The patient has evidence at Screening of Hunter syndrome-related cognitive impairment as assessed using the BSID-III and defined as a composite score on the cognitive scale =55 and =85.
•- The patient has received and

排除标准

•- The patient has clinically significant non-Hunter syndrome-related CNS involvement (such as Fragile-X syndrome) which is judged by the Investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
•- The patient has a large chromosomal deletion or complex rearrangement that includes a deletion of the FMR1 and/or FMR2 genes.
•- The patient has a significant medical or psychiatric comorbidity(ies) that might affect study data or confound the integrity of study results.
•- The patient has contra-indications for performance of lumbar puncture such as musculoskeletal/spinal abnormalities or risk of abnormal bleeding.
•- The patient has a history of complications from previous lumbar punctures or technical challenges in conducting lumbar punctures such that the potential risks would exceed possible benefits for the patient.
•- The patient has an opening CSF pressure upon lumbar puncture that exceeds 30.0 cm H2O.
•- The patient has experienced infusion-related anaphylactoid event(s) or has evidence of consistent severe adverse events related to treatment with Elaprase which, in the Investigator’s opinion, may pose an unnecessary risk to the patient.
•- The patient has received a cord blood or bone marrow transplant at any time or has received blood product transfusions within 90 days prior to Screening.
•- The patient has a history of poorly controlled seizure disorder.
•- The patient is unable to comply with the protocol (eg, has significant hearing or vision impairment, a clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, known clinically significant psychiatric/behavioral instability, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator.
•- The patient is enrolled in another clinical study that involves clinical investigation or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrollment or at any time during the study.
•- The patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to compromised airways or other conditions.
•- The patient has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use (IFU), including:
•a. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device
•b. The patient’s body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the Investigator
•c. The patient’s drug therapy requires substances known to be incompatible with the materials of construction
•d. The patient has a known or suspected local or general infection
•e. The patient is at risk of abnormal bleeding due to a medical condition or therapy
•f. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
•g. The patient has a functioning CSF shunt device
•h. The patient has shown an intolerance to an implanted device

研究者

发起方

Shire HGT Inc

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