NCT04120493View on ClinicalTrials.gov

Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

43

Participants

Timeline

Start Date

September 6, 2019

Primary Completion Date

June 30, 2029

Study Completion Date

December 31, 2029

Conditions
Huntington's Disease
Interventions
GENETIC

intra-striatal rAAV5-miHTT

One time MRI-guided stereotaxic infusion of rAAV5-miHTT into the brain

OTHER

Imitation (sham) surgery

Simulated surgical procedure with skin incisions only; no intrastriatal injections and no burr holes through the skull

Trial Locations (12)

21287

Johns Hopkins University, Baltimore

23298

Virginia Commonwealth University VCU School of Medicine, Department of Neurology, Richmond

37232

Vanderbilt University Medical Center, Nashville

43210

Ohio State University, Columbus

48105

University of Michigan Department of Neurology, Ann Arbor

60612

Rush University Medical Center, Chicago

77030

The University of Texas, Houston

80113

CenExel Rocky Mountain Clinical Research, Englewood

85724

University of Arizona (Surgical Site Only), Tucson

94158

University of California, San Francisco, San Francisco

98195

University of Washington Medical Center, Seattle

35294-0111

University of Alabama at Birmingham, Birmingham

All Listed Sponsors
lead

UniQure Biopharma B.V.

INDUSTRY