CelluTarget · Multifocal Motor Neuropathy · Clinical Trials
Phase 2
Active (not recruiting)
NCT04120493

AMT-130 (AAV5-miHTT gene therapy)

Sponsored by uniQure ↗
View on ClinicalTrials.gov ↗
Timeline
Study Dates
Start Date
April 2020
Est. Completion
December 2027
Enrollment Target
26 participants
Status
Active (not recruiting)
Study Design
Primary Endpoint
Safety and tolerability of AMT-130 administered via MRI-guided stereotactic neurosurgery; exploratory efficacy on disease progression and CSF neurofilament light
Registry
ClinicalTrials.gov Record
Registry ID
NCT04120493
Direct Link
clinicaltrials.gov/NCT04120493 ↗
Related Treatment
Treatment Being Studied
AMT-130 (AAV5-miHTT gene therapy) →