NCT07640984尚未招募1 期
A Global, Open-Label, Phase I/II Trial, to Evaluate Safety, Tolerability, Pharmacodynamic and Preliminary Efficacy of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 12
- 试验地点
- 5
研究概览
简要总结
This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its pharmacodynamic effects, and explore its potential to demonstrate early clinical effects on disease-relevant outcomes.
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
- — 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with confirmed MPS IIIB with a phenotype of MPS IIIB that is not classified as slowly progressive (i.e., not attenuated).
- •A chronological age <6 years.
- •Ability to complete and achieve a Standard score ≥55, using either Bailey Scale of Infant Development-4 (BSID-4) Cognitive domain or Kaufman Assessment Battery for Children-II (KABC-II) Non-Verbal Index, whichever tool is most appropriate for the participant's chronological age.
排除标准
- •Contraindication for lumbar puncture or MRI.
- •A participant who has a medical condition or extenuating circumstance that, in the opinion of the principal investigator or sub-investigator, might compromise the participant's ability to comply with protocol requirements, the participant's well-being or safety, or the interpretability of the participant's clinical data.
- •A participant who has received any other investigational drug product (including but not limited to, tralesinidase alfa enzyme replacement therapy [TA-ERT], Genistein, KINERET [anakinra], ambroxol, miglustat) within 4 months (or 5 half-lives, whichever is longer) before the time of providing informed consent.
- •A participant who has received gene therapy treatment or hematopoietic stem cell transplantation (HSCT) with successful engraftment.
- •Serious drug allergy or hypersensitivity to any components of JR-446 or medications likely prescribed during the trial.
- •A participant has a history of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture.
- •A patient with recurrent epileptic seizures not adequately controlled with anti-seizure medication, and which, in the clinical judgment of the principal investigator, would preclude safe participation in the trial.
- •Serology consistent with human immunodeficiency virus (HIV) exposure or consistent with active hepatitis B (HepB) or hepatitis C (HepC) infection.
- •A participant/family, who, in the opinion of the investigator, may not be able to comply with protocol requirements and cooperate fully with the trial assessments, procedures, and scheduling for JR-446 IMP dose administrations.
- •Study participants for whom informed consent is unable to be provided by a parent or legal guardian; or when applicable for a study participant who is unable to provide assent with respect to study participation in conjunction with parental or legal guardian consent for participation on study.
- •The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
研究组 & 干预措施
JR-446
Experimental
Arm: 1
干预措施: JR-446 (Drug)
研究者
研究点 (5)
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