A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants with Hunter Syndrome - DNLI-E-0002

Denali Therapeutics Inc.0 个研究点目标入组 45 人开始时间: 2024年5月10日最近更新: 2024年7月22日

适应症

试验速览

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•Confirmed diagnosis of MPS II, Cohort A: Participants aged =5 to =10 years with neuronopathic MPS II, Cohort B: Participants aged = 1 to = 18 years with non-neuronopathic, neuronopathic MPSII or unknown phenotype, Cohort C:Participants aged < 4 years with neuronopathic MPS II (this cohort can include participants =4 to =18 years of age if the participant is a blood relative with the same genetic mutation as a participant aged < 4 years 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, non-neuronopathic MPS II participants <6 or =17 years at screening, and neuronopathic MPS II participants =1 to =18 years at screening with a history of prior haematopoietic stem cell transplantation or gene therapy 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.

•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 (except for participants in Cohort E), 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

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