NL-OMON47624尚未招募不适用
A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects with Mucopolysaccharidosis type VI (MPS VI) Using Adeno-Associated Viral Vector 8 to Deliver the human ARSB gene to Liver. - Phase I/II Study in MPS VI with viral vector
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 4
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
- 2 至 64（—）
入选标准
- •1. Subjects must have a documented biochemical and molecular diagnosis of MPS VI.
- •2. Subjects must be 4 years old or older.
- •3. Subjects should have received Enzyme Replacement Therapy (ERT) for at least 12 months before enrolment, and should continue to receive treatment until 7-14 days before IMP administration.
- •4. Documented informed consent; willingness to adhere to protocol and required long-term follow-up as evidenced by written informed consent.
排除标准
- •1. Subjects unable or unwilling to meet requirements of the study.
- •2. Participation in a clinical study with an investigational drug in the 6 months prior to enrolment in this trial.
- •3. Subjects who are unable to perform the 6MWT.
- •4. History of severe anaphylactoid reaction to Naglazyme in subjects receiving ERT that could affect the safety (severe reaction is meant to be an event with respiratory impairment that is life-threatening).
- •5. Presence of tracheostomy or need of ventilatory assistance.
- •6. Subjects with evidence of progressive severe myelomalacia that is predicted to require neck surgery in the first six months after enrolment.
- •7. Serum AST or ALT above the upper limit of normal range at the baseline evaluations (Baseline 2, -5 days).
- •8. Co-existence of chronic diseases or clinically relevant abnormal baseline laboratory values; infections with hepatitis B, C, or HIV (Baseline 1).
- •9. Systemic corticosteroid therapy or other immunosuppressive/immunomodulating drugs within 2 weeks prior to IMP administration.
- •10. Female individuals of childbearing age who are pregnant or nursing or unwilling to use effective contraception for at least one year post-IMP administration.
- •11. Fertile male individuals who are unwilling to use male barrier contraceptives such as condom.
- •12. Any other condition that would not allow the subject to complete follow-up examinations during the course of the study and that, in the opinion of the Investigator, would make the subject unsuitable for the study.
- •13. Detectable serum neutralizing antibodies (NAB) against AAV8 vector (Screening)
- •14. Presence of serum antibodies anti-ARSB above the limit of detection of the assay (antibodies anti-ARSB titer >31250 or positive to the value of dilution 1:10 according to the performed assay) at Screening.
研究者
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