CTIS2022-503142-41-00招募中1 期
An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Male Subjects - JR-141-GS32
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 71
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
- 0 至 65+（—）
- 性别
- All
入选标准
- •A subject who participated in the Parent Study (JR-141-GS31) and completed the assessments at Week 105 in Cohort A or Week 53 in Cohort B, and in the opinion of the principal investigator has no safety concerns to enter this study., A subject from whom an IRB or IEC-approved written informed consent form (ICF) can be obtained, which is voluntarily signed. If the subject is aged under 18 years (aged under 16 years in the United Kingdom [UK]) at the time of enrollment or willingness to participate in the study cannot be confirmed due to MPS II related intellectual disability, the subject’s legally acceptable representative (e.g., his parents or guardians) may sign the ICF on behalf of the subject. Written informed assent should be obtained from the subject, wherever possible. The principal investigator or his/her designee will retain the original copy of each subject’s signed consent and assent (if applicable) document., Female subject of child bearing potential or male subject whose female partner is of child-bearing potential , i.e., fertile, following menarche and until becoming post- menopausal unless permanently sterile, agrees to use a medically accepted, highly effective method of contraception, from the time of signing the ICF., For subjects with hearing impairment requiring hearing aid(s), every effort will be made to encourage compliance with the use of functioning hearing aid(s). Subject or/and parent/legally acceptable representative agrees to wearing them during the study and on neurocognitive testing days.
排除标准
- •A subject who has received gene therapy treatment at any point., Refusal to sign the ICF., A subject who is judged by the principal investigator or sub-investigator as being unable to undergo lumbar puncture, including those who have difficulties in attaining the required position for lumbar puncture due to joint contracture or those who are likely to experience breathing difficulties during the lumbar puncture process., A subject who changed treatment from JR-141 to idursulfase during the treatment period in the Parent Study (JR-141-GS31)., A subject who is unable to comply with the protocol (e.g., is unable to return for safety evaluations or is otherwise unlikely to complete the study) as determined by the principal investigator or sub-investigator., A subject who is judged by the principal investigator or sub-investigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity including to anesthesia or hypersensitivity to any component of JR-141., A subject who has a known or suspected local or general chronic infection or is at risk of abnormal bleeding due to medical conditions* or therapies the investigator classifies as causing the patient to be ineligible to participate in the study. * Medical Conditions: - Clinically significant multiple or severe drug allergies, intolerance to topical corticosteroids, or severe post-treatment hypersensitivity reactions (including, but not limited to, erythema multiforme major, linear immunoglobulin A [IgA], toxic epidermal necrolysis, and exfoliative dermatitis) - Evidence or history of significant active bleeding or coagulation disorder or use of non-steroidal anti-inflammatory drugs or other drugs that affect coagulation or platelet function within 14 days prior to lumbar catheter insertion - Allergy to lidocaine (Xylocaine®) or its derivatives, A subject who otherwise is judged by the principal investigator to be ineligible to participate in the study., [Only in France] (9) Persons deprived of their liberty by a judicial or administrative decision, according to article L. 1121-6 of the Public Health Code (Code dela santé publique, CSP) adults who are the subject of a measure of legal protection or unable to express their consent according to article L. 1121-8 of the CSP.
研究者
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