Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03771898 | A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy | PHASE2 | ACTIVE NOT_RECRUITING | 36 | — | — | May 13, 2019 | May 1, 2026 | Apr 15, 2026 | 30 | United States, Argentina +12 |
Loss of locomotion was estimated using interval censoring survival analysis. Survival probability free of loss of locomotion based on GMFC-MLD was estimated up to Week 106 (or two years), with associated 2-sided 95 percent (%) confidence interval (CI). GMFC-MLD scale consists of 7 categories, scores ranging from 0 (walking without support with quality of performance normal for age) to 6 (loss of any locomotion as well as loss of any head and trunk control). Higher scores mean a worse outcome. The data was reported in terms of Mean as survival function was quantified using a weighted average of percentage of participants not reaching the event of interest, with weights derived from the relative size of treated and control units in the strata used for the stratified log-rank test in the primary analysis.
| Arm | Type | Description |
|---|---|---|
| SHP611 | EXPERIMENTAL | Participants will receive 150 milligrams (mg) of SHP611 intrathecally (IT) via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once weekly for 106 weeks in six groups (Group A, B, C, D, E, and F) based on participant's age and motor dysfunction. |
| Name | Type | Description |
|---|---|---|
| SHP611 | DRUG | Participants will receive 150 mg of SHP611 IT via IDDD or LP once weekly for 106 weeks. |
Inclusion Criteria: * The participant must have a documented diagnosis of MLD (Groups A-F): 1. Low ASA activity in leukocytes (compared to laboratory normal range). 2. Elevated sulfatides in urine. * The participant must have a gait disorder due to spastic ataxia or weakness attributable to ML...