CTIS2023-510032-37-00进行中（未招募）1 期
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA - ABT-001
ltragenyx Pharmaceutical Inc.0 个研究点目标入组 32 人开始时间: 2024年2月1日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 32
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（—）
- 性别
- All
入选标准
- •Diagnosis of MPS IIIA confirmed by the following methods: o No detectable or significantly reduced SGSH enzyme activity by leukocyte assay, and o Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene, Age: From 6 months to 2 years or children older than 2 years with a minimum Cognitive DQ of 60 or above (calculated by BSID-III)
排除标准
- •Inability to participate in the clinical evaluation as determined by Principal Investigator, Serology consistent with exposure to human immunodeficient virus, or serology consistent with active hepatitis B or C infection, Bleeding disorder or any other medical condition or circumstance in which a LP (for collection of CSF) is contraindicated according to local institutional policy, Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing, Uncontrolled seizure disorder, Any item (braces, etc.) which would exclude the subject from being able to undergo MRI according to local institutional policy, Any other situation that precludes the subject from undergoing procedures required in this study, Subjects with cardiomyopathy or significant congenital heart abnormalities, Yhe presence of significant non-MPS IIIA related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study, Abnormal laboratory values Grade 2 or higher as defined in Common Terminology Criteria for Adverse Events (CTCAE) v4.03 for gamma glutamyl transferase (GGT), total bilirubin, creatinine, hemoglobin, white blood cell (WBC) count, platelet count, prothrombin time (PT) and aPTT, Female participant who is pregnant or demonstrates a positive urine or beta hCG result at screening assessment (if applicable), Identification of two nonsense or null variants on genetic testing of the SGSH gene, Any vaccination with viral attenuated vaccines less than 30 days prior to the scheduled date of treatment (and use of prednisolone), Previous treatment by Hematopoietic Stem Cell transplantation, Previous participation in a gene/cell therapy or enzyme replacement therapy clinical trial., At least one S298P mutation in the SGSH gene, Has evidence of an attenuated phenotype of MPS IIIA, Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics, Active viral infection based on clinical observations, Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer, or precludes the child from participating in the protocol assessments and follow up, • Subjects with total anti-AAV9 antibody titers > or = 1:100 equivalent to a positive screen as determined by ELISA in serum, Subjects with a positive response for the enzyme-linked immunosorbent spot (ELISpot) for T-cell responses to AAV9
研究者
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