NL-OMON45009已完成2 期
An Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Mucopolysaccharidosis Type IIIA Disease. - San Filippo SHP-610-201
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
Trial ended prematurely
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 11（—）
入选标准
- •Patients must meet all of the following criteria to be considered eligible for enrollment:;1. Patient has completed through at least the Week 48 visit of Study HGT-SAN-093.
- •2. The patient*s parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board- (IRB-)/Independent Ethics Committee- (IEC-) 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, as relevant, must be obtained.
排除标准
- •Patients will be excluded from the study if any of the following criteria are met:;1. The patient, if randomized to treatment in Study HGT-SAN-093, has experienced a decline of more than 20 points in the BSID-III cognitive DQ score between Baseline and the Week 48 visit in Study HGT-SAN-093, AND, upon individual evaluation by the Investigator, has been deemed a treatment failure*.
- •2. The patient has experienced, in the opinion of the Investigator, a safety or medical issue that contraindicates treatment with HGT-1410, including but not limited to clinically relevant intracranial hypertension, severe infusion-related reactions after treatment with HGT-1410, uncontrollable seizure disorder.
- •3. The patient has a known hypersensitivity to any of the components of
- •HGT-1410. Patients with documented infusion-related reactions that are
- •clinically manageable (for example, with pre-medication or slowing
- •infusion rate) are not necessarily excluded based on the assessment of
- •the investigator.
- •4. The patient is enrolled in another clinical study, other than HGT-SAN-093, that involves clinical investigations 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.
- •5. 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.
- •6. The patient has a condition that is contraindicated as described in the SOPH-APORT ® 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*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
- •7. The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator.;*All treated patients in Study HGT-SAN-093 will have their cognitive development assessed at the Week 48 Visit in Study HGT-SAN-093. If a decline from Baseline of 20 points or less in the BSID-III DQ score is observed, then the patient may proceed into the Study SHP-610-201 without further evaluation. If a decline from Baseline of more than 20 points in DQ score is observed, then an individual evaluation by the Investigator will occur to determine if the patient is a treatment failure. This individual evaluation will take into account the DQ scores, VABS-II score, physical status, and any other information available for that patient at that time. If the Investigator deems the patient to
- •be a treatment failure, then the patient may not enter the Study SHP-610-201.
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