Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
SHP611 · 1 trial · 1 indication
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...
SHP611 is an investigational small molecule being studied for the treatment of Metachromatic Leukodystrophy (MLD), a rare genetic disorder. It is administered intrathecally and is currently in clinical development, though it is not approved by regulatory authorities.
SHP611 is being developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company listed on the stock exchange under the ticker symbol TAK. The drug is in Phase 2 clinical development for Metachromatic Leukodystrophy.
SHP611 is in Phase 2 clinical development. A Phase 2 study of intrathecal SHP611 in children with Metachromatic Leukodystrophy has been completed. The drug remains investigational and has not received regulatory approval.
SHP611 has been studied in one clinical trial, identified as NCT03771898, titled "A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy." This Phase 2 study enrolled 36 participants and has been completed. The trial was conducted across multiple countries including the United States, Argentina, Belgium, Brazil, Canada, France, Germany, Greece, Israel, Italy, Japan, Netherlands, Spain, and the United Kingdom.
SHP611 is not FDA approved. It is an investigational drug currently in Phase 2 clinical development for Metachromatic Leukodystrophy. The completed Phase 2 trial, NCT03771898, evaluated the drug in children, but regulatory approval has not been granted.