NL-OMON52445招募中3 期
An open-label, single-arm, multicenter study of intracerebral administration of adeno-associated viral vectors serotype rh.10 carrying the human N-sulfoglycosamine sulfohydrolase (SGSH) cDNA for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA) - P4-SAF-302
ysogene SA0 个研究点目标入组 10 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
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 11（—）
入选标准
- •1. Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene
- •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
- •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.
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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