NL-OMON40318已完成2 期
A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type IIIA Disease - HGT-SAN-093
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 2
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 11（—）
入选标准
- •1. Documented MPS IIIA diagnosis:
- •a) All patients must show a documented deficiency in sulfamidase
- •enzyme activity of *10% of the lower limit of the normal range as
- •measured in fibroblasts or leukocytes
- •b) patients must show documented mutations in each SGSH allele OR there must be
- •documentation of mutations in each SGSH allele in a sibling affected by
- •MPSIIIA, provided parental consent is obtained to use this information.
- •2. Age *12 months and * 48 months
- •3. The patient has a Developmental Quotient score *60%, assessed by
- •cognitive evaluation at screening assessment using the Bayley Scales of
- •Infant and Toddler Development *3rd Edition (BSID-III)
- •4. The patient is medically stable, in the opinion of the Investigator, and
- •able to accommodate the protocol requirements, including travel,
- •assessments, and IDDD surgery, without placing an undue burden on the
- •patient/patient's family
- •5. The patient, and patient's parent(s) or legal guardian must have
- •voluntarily signed an Independent Ethics Committee-approved informed
- •consent form after all relevant aspects of the study have been explained
- •and discussed with the patient's parent(s), or legal guardian. Consent of
- •the patient's parent(s) or legally authorized guardian(s) must be
- •obtained prior to the start of any study procedures.
排除标准
- •1. The presence of significant non-MPS IIIA related central nervous
- •system (CNS) impairment or behavioral disturbances that would
- •confound the scientific integrity or interpretation of study assessments,
- •as determined by the Investigator.
- •2. The presence of the S298P mutation in either or both SGSH alleles,
- •associated with attenuated disease OR there must be documentation of
- •mutations in each SGSH allele in a sibling affected by MPSIIIA, provided
- •parental consent is obtained to use this information.
- •3. The presence of relatively attenuated MPS IIIA disease in an older
- •sibling, defined as preservation of any comprehensible speech beyond
- •the age of 10 years
- •4. Visual or hearing impairment, in the clinical judgement of the
- •investigator, sufficient to preclude cooperation with neurodevelopmental
- •5. In the opinion of the Investigator, the patient is assessed as having an
- •unacceptably high risk for anesthesia due to airway compromise, drug
- •hypersensitivity, or other conditions (such as neuroleptic malignant
- •syndrome, malignant hyperthermia, or other anesthesia-related
- •6. The patient has a history of poorly controlled seizure disorder.
- •7. The patient is currently receiving psychotropic or other medications,
- •which in the Investigator's opinion would be likely to substantially
- •confound test results.
- •8. The patient has a history of bleeding disorder or is unable to abstain
- •from medications that, in the opinion of the investigator, place them at
- •risk of bleeding following surgery or lumbar puncture..
- •9. The patient participated in a clinical trial of another investigational
- •medicinal product, within the 30 days prior to the study (or within 5
- •elimination half lives of the investigational product), or is currently
- •enrolled in another study that involves an investigational drug or device.
- •NOTE: Nutritional supplements, including genistein are permitted if they
- •are taken or administered outside the context of a formal investigation.
- •10. The patient has received a hematopoietic stem cell or bone marrow
- •transplant, or gene therapy.
- •11. The patient has a condition that is contraindicated as described in
- •the SOPH-A-PORT Mini S IDDD Instructions for Use, including:
- •a) The patient has had 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-APORT
- •Mini S Access Port, as judged by the Investigator
- •c) The patient has a known or suspected local or general infection
- •d) The patient has one or more spinal abnormalities that could
- •complicate safe implantation or fixation
- •e) The patient has a functioning CSF shunt device
- •f) The patient has shown an intolerance to an implanted device
- •12. The patient's parent(s) or patient's legal guardian(s) is/are unable
- •to understand the nature, scope, and possible consequences of the
- •study, or do/does not agree to comply with the protocol defined
- •schedule of assessments.
- •13. The patient is unable to comply with the protocol (eg, has a clinically
- •relevant medical condition making implementation of the protocol
- •difficult, unstable social situation, or otherwise unlikely to complete the
- 另有 2 项未显示
研究者
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