Study Summary
This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study. The first three cohorts of the study have completed enrollment, including the randomized, double-blind, sham-controlled cohorts. Cohort 4 is open-label. Cohort 4 participants will receive high dose AMT-130.
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Request More InfoInterventions
intra-striatal rAAV5-miHTTGENETIC
One time MRI-guided stereotaxic infusion of rAAV5-miHTT into the brain
Imitation (sham) surgeryOTHER
Simulated surgical procedure with skin incisions only; no intrastriatal injections and no burr holes through the skull
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | United States |
| University of Arizona (Surgical Site Only) | Tucson | Arizona | United States |
| University of California, San Francisco | San Francisco | California | United States |
| CenExel Rocky Mountain Clinical Research | Englewood | Colorado | United States |
| Rush University Medical Center | Chicago | Illinois | United States |
| Johns Hopkins University | Baltimore | Maryland | United States |
| University of Michigan Department of Neurology | Ann Arbor | Michigan | United States |
| Ohio State University | Columbus | Ohio | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | United States |
| The University of Texas | Houston | Texas | United States |
| Virginia Commonwealth University VCU School of Medicine, Department of Neurology | Richmond | Virginia | United States |
| University of Washington Medical Center | Seattle | Washington | United States |