A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy

Shire Human Genetic Therapies, Inc.0 个研究点目标入组 35 人开始时间: 2019年2月20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: Shire Human Genetic Therapies, Inc.
入组人数: 35

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

性别: All

入选标准

•Patients must meet all of the following criteria to be considered eligible for
•inclusion as a subject in the study:
•1. The subject must have a documented diagnosis of MLD (Groups A-D)
•a. Low ASA activity in leukocytes
•b. Elevated sulfatides in urine
•2. The subject must have a gait disorder due to spastic ataxia or weakness attributed to MLD by the investigator and documented by a pediatric neurologist or medical geneticist by 30 months of age (Groups A-C) or presymptomatic (Group D)
•3. The subject’s age at the time of informed consent, must be:
•? Group A: 18 to 48 months of age
•? Group B: 18 to 72 months of age
•? Group C: 18 to 72 months of age
•? Group D: <18 months of age
•4. The subject’s GMFC-MLD level at screening must be:
•? Group A: GMFC-MLD level of 1 or 2
•? Group B: GMFC-MLD level of 3
•? Group C: GMFC-MLD level of 4
•? Group D: presymptomatic, are younger siblings of enrolled subjects, and have the same ASA allelic constitution
•5. The subject and his/her parent/representative(s) must have the ability to comply with the clinical protocol
•6. Subject's parent or legally authorized representative(s) must provide written informed consent prior to performing any study-related activities. Study-related activities are any procedures that would not have been performed during normal management of the subject
•Inclusion criteria for matched historical controls
•Subjects must meet all of the following criteria to be considered eligible for inclusion as a matched historical control:
•1. The subject must have a documented diagnosis of MLD
•a. Low ASA activity in leukocytes
•b. Elevated sulfatides in urine
•2. Subjects must have a gait disorder due to spastic ataxia or weakness attributed to MLD by the investigator and documented at baseline
•3. Subjects must have at least 2 motor assessments by GMFC-MLD with the second assessment occurring at approximately 106 (±6) weeks after the first assessment or else a second assessment measured before Week 100 with a GMFC-MLD level 5 or 6. Subjects with GMFC-MLD data (pro or retrospectively determined) must have the earliest observation of level 1 or 2 (walking with support) in the data source-verified medical record
•4. Subjects must be 18 to 48 months of age at the earliest assessment
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 35
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

排除标准

•1. Multiple sulfatase disorder as determined by abnormal activity of another lysosomal sulfatase (based upon the reference laboratory’s normal range)
•2. History of hematopoietic stem cell transplantation (HSCT) or gene therapy or undergoes HSCT or gene therapy at any point during the study
•3. Initial presentation of behavioral or cognitive symptoms of MLD (per investigator’s clinical judgment)
•4. The subject has any known or suspected hypersensitivity to agents used for anesthesia or has history of difficult airway or potential for airway compromise
•5. Any other medical condition or serious comorbid illness that in the opinion of the investigator would preclude participation in the study
•6. The subject is enrolled in another clinical study that involves use of any investigational product (drug or device) within 30 days prior to study enrollment or at any time during the study
•7. The subject has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use (IFU)
•Exclusion criteria for matched historical controls
•1. History of hematopoietic stem cell transplantation (HSCT) or gene therapy or undergoes HSCT or gene therapy at any point during the study
•2. Initial presentation of behavioral or cognitive symptoms of MLD (per investigator’s
•clinical judgment)
•3. The subject is enrolled in another clinical study that involves use of any investigational product (drug or device)

研究者

发起方

Shire Human Genetic Therapies, Inc.

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