A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
2상
완료
NCT03771898
| 대상 질환 | Metachromatic Leukodystrophy |
|---|
| 임상 단계 | 2상 |
|---|
| 스폰서 | Shire |
|---|
| 상태 | 완료 |
|---|
| 참여자 수 | 36 명 |
|---|
| 중재(약물) | SHP611 |
|---|
| 시작일 | 2019년 5월 13일 |
|---|
| 완료일 | 2023년 3월 8일 |
|---|
| 결과 공개일 | 2024년 6월 6일 |
|---|
요약
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 |
|---|
| GLIA-MLD Matched External Control for Group A | 9.4 percent probability |
|---|
| p-value | =0.585 |
|---|
| Difference in survival probability (%) | -2.2 (95% CI -22.2–17.7) |
|---|
BENITIONS에서 보기
ClinicalTrials.gov 원문 보기
전체 임상
이 페이지는 ClinicalTrials.gov의 공개 데이터를 정리한 것입니다. 의학적 조언이 아니며 투자 권유나 자문도 아닙니다. 반드시 전문의와 상담하세요.