A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
2期
已完成
NCT03771898
| 适应症 | Metachromatic Leukodystrophy |
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| 分期 | 2期 |
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| 申办方 | Shire |
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| 状态 | 已完成 |
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| 受试者人数 | 36 人 |
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| 干预（药物） | SHP611 |
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| 开始日期 | 2019年5月13日 |
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| 完成日期 | 2023年3月8日 |
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| 结果公布日 | 2024年6月6日 |
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摘要
The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Other aims of the study are to determine the effects of intrathecal administration of SHP611 on movement and speech functions and to learn how well SHP611 injected in the spinal fluid that surrounds the brain and spinal cord is tolerated. Study participants will receive SHP611 for about 2 years with the possibility of an extended treatment period.
Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control
Baseline up to Week 106 · percent probability
| Group A, SHP611 | 7.1 percent probability |
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| GLIA-MLD Matched External Control for Group A | 9.4 percent probability |
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| p-value | =0.585 |
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| Difference in survival probability (%) | -2.2 (95% CI -22.2–17.7) |
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