for people ages 25-65 (full criteria)
at San Francisco, California and other locations
study started
estimated completion
Principal Investigator
Michael Geschwind, MD, Ph.D.
Photo of Michael Geschwind
Michael Geschwind



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.

Official Title

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 Disease


AMT-130 is an investigational, single administration gene therapy intended to modify the disease course for HD. Preclinical studies have shown that AMT-130 lowers huntingtin protein and improved Huntington disease signs in animal models.

This 5-year trial consists of a blinded 18-month Core Study Period to evaluate the safety and potential impact of AMT-130 on disease progression and an unblinded 3.5-year Long-Term Period with annual follow-up visits to evaluate the safety of AMT-130 and disease progression.


Huntington Disease Gene therapy AAV (adeno-associated virus) serotype 5 AAV (adeno-associated virus) serotype 5 Viral vector miHTT muHTT Huntington Disease (HD) rAAV5-miHTT Imitation surgery


You can join if…

Open to people ages 25-65

  • First 4 subjects in Cohort 1: early manifest HD (Stage 2). All remaining subjects in

Cohort 1 and all subjects in Cohort 2: early manifest HD (Stages 1 and 2).

  • HTT gene expansion testing with the presence of ≥44 CAG repeats.
  • Striatal MRI volume requirements per hemisphere: Putamen ≥2.5 cm3 (per side); Caudate ≥2.0 cm3 (per side)
  • All HD concomitant medications stable for 3 months prior to Screening.

You CAN'T join if...

  • Receipt of an experimental agent within 60 days or five half-lives prior to Screening or anytime over the duration of this study.
  • Participation 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.
  • Presence of an implanted deep brain stimulation device.
  • 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.
  • Any contraindication to lumbar puncture or 3.0 Tesla MRI as per local guidelines.
  • Brain and spinal pathology that may interfere with CSF homeostasis and circulation, increased intracranial pressure (including presence of a shunt for the drainage of CSF or an implanted CNS catheter), malformations and/or tumors.
  • Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study.
  • Current use of medications that could improve or worsen chorea or other HD movements including typical neuroleptics, tetrabenazine and deutetrabenazine.


  • University of California, San Francisco accepting new patients
    San Francisco California 94158 United States
  • University of Washington Medical Center accepting new patients
    Seattle Washington 98195 United States

Lead Scientist at UCSF

  • Michael Geschwind, MD, Ph.D.
    Dr. Geschwind received his M.D.and Ph.D. in neuroscience through the National Institutes of Health (NIH)-sponsored Medical Scientist Training Program (MSTP) at the Albert Einstein College of Medicine in New York.


accepting new patients
Start Date
Completion Date
UniQure Biopharma B.V.
Phase 1/2
Study Type
Last Updated