NCT04227600已完成1 期
Phase I/II Study of JR-171 ㏌ Patients With Mucopolysaccharidosis Type I
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 6
- 主要终点
- Number of Participants With Adverse Events
研究概览
简要总结
Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis type I (MPS I).
详细描述
In the JR-171-102 study, subjects will receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously at the same dosages received at Week 12 of the JR-171-101 study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A patient aged 18 years or older in Part 1 or any age in Part 2, at the time of informed consent
- •A patient from whom written informed consent can be obtained. If the patient is aged under 18 years (20 years in case of Japan) at the time of assent or willingness to participate in the study cannot be confirmed due to MPS I-related intellectual disability, informed permission from the patient's legally acceptable representative (e.g. his/her parents or guardians) need to be obtained instead of his/her consent. Even in this case, written informed consent or assent should be obtained from the patient, wherever possible
- •A patient diagnosed with MPS I based on any one of the following criteria:
- •Activity of IDUA enzyme below 10% of lower reference level in leucocytes or cultured skin fibroblasts, AND increased age-related urinary levels of GAGs (before enzyme replacement therapy)
- •Activity of IDUA enzyme below 10% of lower reference level in leucocytes or cultured skin fibroblasts, AND presence of one pathogenic mutation in each of the alleles of the IDUA gene
- •Increased age-related urinary levels of GAGs (before enzyme replacement therapy), AND presence of one pathogenic mutation in each of the alleles of the IDUA gene
- •A patient diagnosed as having no or mild MPS I-related intellectual disability (able to report their own subjective symptoms) by the principal investigator or subinvestigator (Part 1 only)
- •A patient who has received laronidase continuously for at least 12 weeks and has received laronidase on a stable dosage for 2 weeks immediately before the initial administration of JR-171, except for a laronidase naïve patient or a patient who has previously been treated by HSCT)
- •Female patient or male patient whose co-partner is of child-bearing potential agrees to use a medically accepted, highly effective method of contraception, such as spermatocidal gel plus condom, an intrauterine device or oral contraceptives until one month after the final administration
排除标准
- •A patient who received gene therapy treatment
- •A patient who, in the opinion of the principal investigator or subinvestigator, cannot undergo lumbar puncture, including those who have a difficulty in taking a position for lumbar puncture due to joint contracture and those who are likely to develop dyspnea during lumbar puncture
- •A patient who is pregnant or lactating
- •A patient who has developed serious drug allergy or hypersensitivity to any drugs, in the opinion of the principal investigator or subinvestigator, is inappropriate for participation in the study
- •A patient who has received another investigational product within 12 months before enrollment in the study
- •A patient who, in the opinion of the principal investigator or subinvestigator, is ineligible to participate in the study out of consideration for the participant safety.
研究组 & 干预措施
Part1 JR-171
Experimental
Drug: JR-171 IV infusion, dose escalation
干预措施: JR-171 (lepunafusp alfa) (Drug)
Part2 JR-171
Experimental
Drug: JR-171 IV infusion, dose escalation, low dose, high dose
干预措施: JR-171 (lepunafusp alfa) (Drug)
结局指标
主要结局
Number of Participants With Adverse Events
时间窗: Time of the first dose of JR-171 until the last visit (Week 5 for Part 1/ Week 13 for Part 2).
次要结局
- Pharmacokinetics of Each Dose in Part 2 (AUC0-t)(Week 1 to Week 12)
- Heparan Sulfate Concentrations in Cerebrospinal Fluid in Part 2(Baseline to Week 12)
- Pharmacokinetics of Each Dose in Part 1 (AUC0-t)(Week 1 to Week 4)
- Heparan Sulfate Concentrations in Serum in Part 1(Baseline to Week 5)
- Pharmacokinetics of Each Dose in Part 1 (Cmax)(Week 1 to Week 4)
- Dermatan Sulfate Concentrations in Cerebrospinal Fluid in Part 2(Baseline to Week 12)
- Pharmacokinetics of Each Dose in Part 2 (Cmax)(Week 1 to Week 12)
- Heparan Sulfate Concentrations in Cerebrospinal Fluid in Part 1(Baseline to Week 4)
- Dermatan Sulfate Concentrations in Cerebrospinal Fluid in Part 1(Baseline to Week 4)
- Dermatan Sulfate Concentrations in Serum in Part 1(Baseline to Week 5)
- Heparan Sulfate Concentrations in Serum in Part 2(Baseline to Week 13)
- Dermatan Sulfate Concentrations in Serum in Part 2(Baseline to Week 13)
- Liver Volumes (Body Weight Adjusted) in Part 1(Baseline to Week 5)
- Heparan Sulfate Concentrations in Urine in Part 1(Baseline to Week 5)
- Dermatan Sulfate Concentrations in Urine in Part 1(Baseline to Week 5)
- Heparan Sulfate Concentrations in Urine in Part 2(Baseline to Week 13)
- Dermatan Sulfate Concentrations in Urine in Part 2(Baseline to Week 13)
- Spleen Volumes (Body Weight Adjusted) in Part 1(Baseline to Week 5)
- Liver Volumes (Body Weight Adjusted) in Part 2(Baseline to Week 13)
- Spleen Volumes (Body Weight Adjusted) in Part 2(Baseline to Week 13)
研究者
研究点 (6)
Loading locations...
相似试验
已完成
2 期
A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)Mucopolysaccharidosis IINCT03359213JCR Pharmaceuticals Co., Ltd.18
进行中（未招募）
3 期
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)Mucopolysaccharidosis IINCT04573023JCR Pharmaceuticals Co., Ltd.86
已完成
1 期
A Study of JR-171 in Patients With Mucopolysaccharidosis Type IJPRN-jRCT2080225015JCR Pharmaceuticals Co., Ltd,19
已完成
2 期
A Study of JR-141 in Patients With Mucopolysaccharidosis IIMucopolysaccharidosis IINCT03568175JCR Pharmaceuticals Co., Ltd.28
已完成
1 期
A Study of JR-141 in Patients With Mucopolysaccharidosis Type IIMucopolysaccharidosis IINCT03128593JCR Pharmaceuticals Co., Ltd.14