NL-OMON54074招募中不适用
A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants with Hunter Syndrome - DENA211102_DNL310 in Pediatric Participants with Hunter Syndrome
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 2
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 17（—）
入选标准
- •- Confirmed diagnosis of MPS II
- •- Cohort A: Participants aged >= 5 to <=10 years with neuronopathic MPS II (nMPS
- •- Cohort B: Participants aged >= 1 to <=18 years with non-neuronopathic MPS
- •II (nnMP II), neuronopathic MPS II, or unknown phenotype
- •- Cohort C: Participants aged <4 years with neuronopathic MPS II (this cohort
- •can include participants >=4 years of age if participant is a blood relative
- •with the same genetic mutation as participant >= 4 to <=18 years of age who will
- •be enrolled in the study)
- •- Cohort D: Participants aged <=18 years with nMPS II and nnMPS II and
- •preexisting hepatomegaly who have never taken standard - of - care ERT
- •- Cohort E: Neuronopathic MPS II Participants aged >= 6 years at screening,
- •nnMPS II participants < 6 or >=17 years at screening, and nMPS II participants
- •>= 1 to <=18 years at screening who have completed at least 48 weeks in Study
- •DNLI-E-0001
- •- For participants receiving intravenous iduronate 2-sulfatase (IDS) ERT,
- •tolerated a minimum of 4 months of therapy during the period immediately prior
- •to screening.
排除标准
- •- Unstable or poorly controlled medical condition(s) or significant medical or
- •psychological comorbidity or comorbidities that, in the opinion of the
- •investigator, would interfere with safe participation in the trial or
- •interpretation of study assessments
- •- Use of any CNS-targeted MPS II ERT within 3 months before study start for
- •participants aged >=5 years, and within 6 months before study start for
- •participants aged <5 years.
- •- Use of IDS gene therapy or stem cell therapy at any time
- •- Clinically significant thrombocytopenia, other clinically significant
- •coagulation abnormality, or significant active bleeding, or required treatment
- •with an anticoagulant or more than two antiplatelet agents
- •- Contraindication for lumbar punctures
- •- Have a clinically significant history of stroke, status epilepticus, head
- •trauma with loss of consciousness, or any CNS disease that is not MPS
- •II-related within 1 year of screening
- •- Have had a ventriculoperitoneal (VP) shunt placed, or any other brain
- •surgery, or have a clinically significant VP shunt malfunction within 30 days
- •of screening
- •- Have any clinically significant CNS trauma or disorder that, in the opinion
- •of the investigator, may interfere with assessment of study endpoints or make
- •participation in the study unsafe
- •- Have clinically significant anemia
研究者
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