EUCTR2011-002044-28-DK进行中（未招募）1 期
A Phase I/II, Multicenter, Open-label, Dose Escalation Study of HGT-1110 Administered Intrathecally in Children with Metachromatic Leukodystrophy
Shire Human Genetics Therapies Inc0 个研究点目标入组 24 人开始时间: 2011年8月16日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 24
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
- •For Cohorts 1-4:
- •1. Confirmed diagnosis of metachromatic leukodystrophy by both:
- •Arylsulfatase A (ASA) deficiency by assay in leukocytes
- •Elevated sulfatide in urine
- •2. Appearance of the first symptoms of disease at or before 30 months of age.
- •For Cohorts 1-3 only:
- •3. Ambulatory at the time of screening. The minimum level of function required to meet this criterion is defined as the ability to walk forward 10 steps with one hand held.
- •4. The patient is less than 12 years of age at the time of screening.
- •For Cohorts 4 only:
- •3.1 Minimum motor function requirements:
- •a. A total GMFM-88 (percent) score =40 at the screening examination and a total GMFM-88 (percent) score =35 at the baseline examination,
- •b. GMFM-88 Dimension E: Walking, Running & Jumping, item 68 (walk forward 10 steps with one hand held score of at least 1 initiates at the screening and baseline examinations (if applicable).
- •4.1 The patient is less than 8 years of age at the time of screening.
- •For Cohorts 1-4:
- •5. Neurological signs of MLD must be present at the screening examination.
- •6. The patient and his/her parent/representative(s) must have the ability to comply with the clinical protocol.
- •7. Patient’s parent(s) 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 patient.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 24
- •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
- •Patients will be excluded from the study if there is evidence or history of any of the following criteria at screening:
- •For Cohorts 1-4:
- •1. History of hematopoietic stem cell transplantation.
- •2. The patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to airway compromise or other conditions.
- •3. Any other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial.
- •4. The patient is enrolled in another clinical study that involves the use of any investigational product (drug or device) other than HGT-1110 or the IDDD used in this study within 30 days prior to study enrollment or at any time during the study.
- •5. The patient is pregnant or breastfeeding.
- •6. 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, 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 has a known or suspected local or general infection.
- •d The patient is at risk of abnormal bleeding due to a medical condition or therapy.
- •e The patient has one or more spinal abnormalities that could complicate safe implantation or fixation.
- •f The patient has a functioning CSF shunt device.
- •g The patient has shown an intolerance to an implanted device.
研究者
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