EUCTR2018-000195-15-GB进行中（未招募）1 期
An Open-Label, Single-Arm, Multicenter Study of Intracerebral Administration of Adeno-Associated Viral Vectors Serotype rh10 Carrying the Human N-sulfoglucosamine sulfohydrolase (SGSH) cDNA for the Treatment of Mucopolysaccharidosis Type IIIA
ysogene SA0 个研究点目标入组 20 人开始时间: 2018年9月19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
- 20
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. Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene mutations
- •2. Age = 30 months at screening (main cohort) or =6 months and < 30 months (ancillary cohort)
- •3. Cognitive DQ score on BSID-III = 50%
- •4. Signed written informed consent before any study related procedure is performed
- •5. Medical status sufficiently stable, in the opinion of the investigator, to adhere to the study visit schedule and other protocol procedures
- •6. Confirmation by the study neurosurgeon and anesthesiologist of the feasibility of the neurosurgical procedure.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 20
- •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. Homozygous for the S298P mutation or non-severe form of MPS IIIA, based on investigator’s judgement
- •2. Past participation in another gene or cell therapy clinical trial
- •3. Past use of SGSH enzyme replacement therapy for a cumulative period exceeding 3 months. In addition, a washout period of at least 2 months is required prior to screening
- •4. Current participation in a clinical trial of another investigational medicinal product.
- •NOTE: Nutritional supplements, including Genistein are permitted if they are taken outside the context of an investigational trial
- •5. History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following surgery
- •6. Presence of concomitant medical condition precluding lumbar puncture
- •7. Presence of any item (e.g., metal braces) precluding undergoing MRI
- •8. Any condition that would contraindicate treatment with immunosuppressants such as tacrolimus, mycophenolate mofetil or steroids
- •9. History of significant non-MPS IIIA related CNS impairment or behavioral disturbances that would confound scientific rigor or interpretation of results
- •10. Rare and unrelated serious comorbidities (e.g. Down syndrome), intraventricular hemorrhage in the new-born period, or extreme low birth weight (<1500 grams)
- •11. History of poorly controlled seizure disorder
- •12. Any vaccination 1 month prior to the planned surgery
- •13. Serology consistent with HIV exposure or consistent with active hepatitis B or C infection
- •14. Grade 2 or higher lab abnormalities for LFT, bilirubin, creatinine, hemoglobin, WBC count, platelet count, PT, and a PTT
- •15. Visual or hearing impairment sufficient, in the clinical judgment of the investigator, to preclude cooperation with neurodevelopmental testing. Use of hearing aids is permitted.
研究者
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