A Phase I/II, Randomized, Double-Blind, Sham Control Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington's Disease

Who is this study for? Patients with Huntington Disease
What treatments are being studied? Intra-Striatal rAAV5-miHTT
Status: Recruiting
Location: See all (12) locations...
Intervention Type: Other, Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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 I/II, randomized, multicenter, dose escalation, double-blind, imitation surgery, first-in-human (FIH) study.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 25
Maximum Age: 65
Healthy Volunteers: No
View:

• Able and willing to provide written informed consent prior to the study and study-related procedure

• Subjects 25 to 65 years of age of both sexes

• Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and EITHER a diagnostic classification level (DCL) of 4 OR a DCL of 3 if the subject either meets the definition of multidimensional manifest HD (UHDRS question 80) or has cognitive symptoms

• HTT gene expansion testing with the presence of ≥40 CAG repeats

• Striatal MRI volume requirements per hemisphere: Putamen ≥2.5 cm3 (per side); Caudate ≥2.0 cm3 (per side)

• All HD concomitant medications (addressing motor, behavioral, and cognitive symptoms) must be stable for 3 months prior to Screening with no change in clinical symptoms requiring change in medication prior to anticipated administration procedure

• Able and willing to comply with all procedures and the study visit schedule as outlined in the protocol

• All female subjects of childbearing potential (FOCP) must have a negative serum pregnancy test at Screening, (and Visit 1A, as appropriate), a negative pregnancy urine dipstick at Baseline, and not be breastfeeding. All FOCPs and sexually mature males must be compliant with a highly effective birth control method.

Locations
United States
Alabama
University of Alabama at Birmingham
Recruiting
Birmingham
California
University of California, San Francisco
Recruiting
San Francisco
Florida
University of Florida College of Medicine
Recruiting
Gainesville
Illinois
Rush University Medical Center
Recruiting
Chicago
Massachusetts
Beth Israel Deaconess Medical Center
Recruiting
Boston
Maryland
Johns Hopkins University
Recruiting
Baltimore
Michigan
University of Michigan Department of Neurology
Recruiting
Ann Arbor
Ohio
Ohio State University
Recruiting
Columbus
Tennessee
Vanderbilt University Medical Center
Recruiting
Nashville
Texas
The University of Texas
Recruiting
Houston
Virginia
Virginia Commonwealth University VCU School of Medicine, Department of Neurology
Recruiting
Richmond
Washington
University of Washington Medical Center
Recruiting
Seattle
Contact Information
Primary
Diane Lopez, MS
amt130_clinical_trials@uniqure.com
781-777-3697
Backup
Lee Rushton
amt130_clinical_trials@uniqure.com
781-528-7716
Time Frame
Start Date: September 6, 2019
Estimated Completion Date: June 2029
Participants
Target number of participants: 44
Treatments
Experimental: Cohort 1
Low dose rAAV5-miHTT (6x10^12 gc/subject).
Experimental: Cohort 2
High dose rAAV5-miHTT (6x10^13 gc/subject).
Sham Comparator: Cohorts 1, 2, 3A & 3B
Imitation (sham) surgery
Experimental: Cohort 3A
High dose rAAV5-miHTT (6x10^13 gc/subject).
Experimental: Cohort 3B
High dose rAAV5-miHTT (6x10^13 gc/subject).
Sponsors
Leads: UniQure Biopharma B.V.

This content was sourced from clinicaltrials.gov