EUCTR2014-001411-39-FR进行中（未招募）1 期
Phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB
Abeona Therapeutics Europe SL.0 个研究点目标入组 9 人开始时间: 2018年10月29日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 9
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 IIIB by both of the following methods:
- •a. No detectable or significantly reduced NAGLU enzyme activity by plasma, serum, or leukocyte assay.
- •b. Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the NAGLU gene
- •3. Clinical history or examination features of neurologic dysfunction
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 9
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •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 NAGLU gene, as judged by the principal investigator
- •3. Prior treatment with NAGLU enzyme replacement therapy (ERT)
- •4. Has evidence of an attenuated phenotype of MPS IIIB, as judged by the principal investigator
- •5. Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
- •6. Inability to be safely sedated in the opinion of the clinical anesthesiologist
- •7. Active viral infection based on clinical observations
- •8. Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer
- •9. Subjects with anti-AAV9 antibody titers = 1:100 as determined by ELISA binding immunoassay
- •10. Serology consistent with exposure to HIV, or serology consistent with active hepatitis B or C infection
- •11. Bleeding disorder or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated according to local institutional policy
- •12. Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing
- •13. Uncontrolled seizure disorder, due to the requirement for multiple MRI examinations as part of the study protocol. Subjects who are stable on anticonvulsive medications may be included
- •14. Any item (braces, etc.) which would exclude the patient from being able to undergo MRI according to local institutional policy
- •15. Any other situation that would exclude the patient from undergoing any other procedure required in this study
- •16. Subjects with cardiomyopathy or significant congenital heart abnormalities
- •17. The presence of significant non-MPS IIIB related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study
- •18. 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 aPTT
- •19. Female participant who is pregnant or demonstrates a positive urine or ß-HCG result at screening assessment (if applicable).
研究者
相似试验
进行中（未招募）
1 期
Gene transfer clinical trial for Mucopolysaccharidosis IIIBEUCTR2014-001411-39-DEAbeona Therapeutics Europe SL.20
进行中（未招募）
1 期
Gene transfer clinical trial for Mucopolysaccharidosis IIIBMPS IIIB is a devastating lysosomal storage disease, caused by a N-a-acetylglucosaminidase (NAGLU) gene defect. Infants with MPS IIIB appear normal at birth, but the disease is relentlessly progressive, with deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatment currently available for the disease.MedDRA version: 20.1 Level: PT Classification code 10056890 Term: Mucopolysaccharidosis III System Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2014-001411-39-GBAbeona Therapeutics Europe SL.9
进行中（未招募）
1 期
Gene transfer clinical trial for Mucopolysaccharidosis IIIBEUCTR2014-001411-39-ESAbeona Therapeutics Inc9
进行中（未招募）
1 期
Gene transfer clinical trial for Mucopolysaccharidosis IIIAMPS IIIA is a devastating lysosomal storage disease, caused by a N-sulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, with deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatment currently available for the disease.MedDRA version: 20.1 Level: PT Classification code 10056890 Term: Mucopolysaccharidosis III System Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2015-003904-21-FRAbeona Therapeutics Inc22
进行中（未招募）
1 期
Gene transfer clinical trial for Mucopolysaccharidosis IIIAMPS IIIA is a devastating lysosomal storage disease, caused by a N-sulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, with deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatment currently available for the disease.EUCTR2015-003904-21-ESAbeona Therapeutics Inc18