An Open-Label Extension for Subjects in Studies HGT-HIT-046 and SHP609-302 Evaluating Long-Term Safety of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Subjects With Hunter Syndrome and Cognitive Impairment
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 6
- 试验地点
- 4
- 主要终点
- Number of Participants with Adverse Events (AEs) by Type and Severity
研究概览
简要总结
The study is an extension of two previous studies (HGT-HIT-046 [NCT01506141] and SHP609-302 [NCT02412787]). Participants must have completed one of the previous studies. The main aim of this study is to collect more information about the safety of the treatments, idursulfase-IT and elaprase, in children and adults with Hunter syndrome and cognitive impairment. Participants will receive the same treatment as in the previous studies.
详细描述
The drug being tested in this study is called idursulfase. Idursulfase is being tested for long term safety in participants with Hunter syndrome and cognitive impairment.
The study will enroll up to approximately 8 patients. Participants will receive idursulfase-IT via intrathecal drug delivery device (IDDD) once monthly along with elaprase intravenous (IV) infusion, weekly.
This multi-center trial will be conducted in France and Canada. The overall time to participate in this study is approximately 4.8 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 19 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •The participant must have completed end of study assessments in studies HGT-HIT-046 [NCT01506141] or SHP609-302 [NCT02412787] and received a clinical benefit from idursulfase-IT in the opinion of the investigator.
- •The participant, or participant's legally designated representative, must have been informed of the nature of this open-label extension and 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. Written consent of the participant's legally designated representative (if applicable) and the participant's consent/assent, as relevant, must be obtained.
- •The participant has continued to receive elaprase on a regular basis in studies HGT-HIT-046 or SHP609-302.
排除标准
- •The participant has experienced, in the opinion of the investigator, a safety or medical issue that contraindicates treatment with idursulfase-IT, including, but not limited to, uncontrolled seizure disorder, bleeding disorder, and clinically relevant hypertension.
- •The participant has clinically relevant intracranial hypertension.
- •The participant is enrolled in another clinical study, other than studies HGT-HIT-046 or SHP609-302, that involves clinical investigations or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrolment or at any time during the study.
研究组 & 干预措施
Idursulfase-IT
Participants will receive idursulfase-IT once monthly and weekly IV infusions of elaprase at the dose used in previous studies (HGT-HIT-045/SHP609-302) via IDDD until benefit is no longer derived from the treatment, or treatment is no longer tolerable, or up to approximately 4.8 years.
干预措施: Idursulfase-IT (Drug)
Idursulfase-IT
Participants will receive idursulfase-IT once monthly and weekly IV infusions of elaprase at the dose used in previous studies (HGT-HIT-045/SHP609-302) via IDDD until benefit is no longer derived from the treatment, or treatment is no longer tolerable, or up to approximately 4.8 years.
干预措施: Elaprase (Drug)
结局指标
主要结局
Number of Participants with Adverse Events (AEs) by Type and Severity
时间窗: Up to approximately 4.8 years
An adverse event (AE) means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. The severity of AEs will be determined per the investigator's assessment.
Number of Participants with AEs Related to Idursulfase-IT
时间窗: Up to approximately 4.8 years
An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to idursulfase-IT will be reported in this outcome measure.
Number of Participants with AEs Related to the IDDD
时间窗: Up to approximately 4.8 years
An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to the IDDD will be reported in this outcome measure.
Number of Participants with AEs Related to Device Surgical Procedure
时间窗: Up to approximately 4.8 years
An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to device surgical procedure will be reported in this outcome measure.
Number of Participants with AEs Related to IT Administration Process
时间窗: Up to approximately 4.8 years
An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to IT administration process will be reported in this outcome measure.
Number of Participants with AEs Related to IV Elaprase Infusion
时间窗: Up to approximately 4.8 years
An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to IV elaprase infusion will be reported in this outcome measure.
次要结局
未报告次要终点