EUCTR2015-003904-21-ES进行中（未招募）1 期
Phase I/II gene transfer clinical trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1.Age 6 months old or greater
- •2.Confirmed diagnosis of MPS IIIA by both of the following methods:
- •a.No detectable or significantly reduced SGSH enzyme activity by leukocyte assay
- •b.Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene
- •3.Clinical history or examination features of neurologic dysfunction
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 18
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 1
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Inability to participate in the clinical evaluation as determined by principal investigator
- •2.Identification of two nonsense or null variants on genetic testing of the SGSH gene, as judged by the principal investigator
- •3.Has evidence of an attenuated phenotype of MPS IIIA, as judged by the principal investigator
- •4.Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
- •5.Inability to be safely sedated in the opinion of the clinical anesthesiologist
- •6.Active viral infection based on clinical observations
- •7.Concomitant illness or requirement for chronic drug treatment that in the opinion of the Principal Investigatior (PI) creates unnecessary risks for gene transfer
- •8.Subjects with total anti-AAV9 antibody titers > or = 1:100 as determined by ELISA binding immunoassay
- •9.Serology consistent with exposure to HIV, or serology consistent with active hepatitis B or C infection
- •10.Bleeding disorder or any other medical condition or circumstance in which a lumbar puncture (for collection of cerebrospinal fluid (CSF)) is contraindicated according to local institutional policy
- •11.Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing
- •12.Uncontrolled seizure disorder, due to the requirement for multiple Magnetic Resonance Imagine (MRI) examinations as part of the study protocol. Subjects who are stable on anticonvulsive medications may be included
- •13.Any item (braces, etc.) which would exclude the subject from being able to undergo MRI according to local institutional policy
- •14.Any other situation that would exclude the subject from undergoing any other procedure required in this study
- •15.Subjects with cardiomyopathy or significant congenital heart abnormalities
- •16.The presence of significant non-MPS IIIA related Central Nervous System (CNS) impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study
- •17.Abnormal laboratory values Grade 2 or higher as defined in CTCAE v4.0, for GGT, total bilirubin, creatinine, hemoglobin, WBC count, platelet count, PT and a PTT
- •18.Female participant who is pregnant or demonstrates a positive urine or serum ß-HCG result at screening assessment (if applicable).
研究者
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