Clinical Trial TitleStudy of a recombinant DNA gene therapy treatment for safety and proof-of-concept (POC) of AMT-130 in adults with early manifest Huntington disease (HD).
National Clinical Trial Number:NCT04120493
Clinical Trial Protocol Description:
The purpose of this study is to find a safe dose of AMT-130 in adults who have tested positive for the HD gene and who have early stage HD compared to sham control for patients with HD. Nationwide, 26 patients will be recruited for this study. At Rush University Medical Center we expect to recruit approximately 5 patients. The treatment will be assigned randomly.
Clinical Trial Eligibility Criteria:
In order to participate you must meet the following criteria:
- First 4 subjects in Cohort 1: Have early manifest HD (Stage 2). All remaining subjects in Cohort 1 and all subjects in Cohort 2: Have early manifest HD (Stages 1 and 2).
- Have HTT gene expansion testing with the presence of ≥44 CAG repeats.
- Have Striatal MRI volume requirements per brain hemisphere
- Have all HD concomitant medications stable for 3 months prior to Screening.
You will be excluded from the study if any of the following criteria apply to you:
- Have eceived an experimental agent within 60 days or five half-lives prior to Screening or anytime over the duration of this study.
- Have participated in an investigational trial or investigational paradigm (such as exercise/physical activity, cognitive therapy, brain stimulation, etc.) within 60 days prior to Screening or anytime over the duration of this study.
- Have an implanted deep brain stimulation device.
- Have any history of gene therapy, RNA or DNA targeted HD specific investigational agents, such as antisense oligonucleotides (ASO), cell transplantation or any other experimental brain surgery.
This is a partial list of eligibility requirements. To inquire about your eligibility, please call the contact number provided. If you wish to inquire via email, please include the title of the study in your message.
Clinical Trial Investigator
RUSH University Medical Center
1620 W Harrison St
Chicago, IL 60612