Terapia genica con AMT-130 (UniQure)

HD Gene TRX1


about the study

It is a dose-escalating, randomized and controlled Phase I/II clinical study to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington’s disease.

about the experimental drug

The therapeutic goal is to inhibit the production of the mutant protein (mHTT).

There are two key components to AMT-130, a vector and a gene encoding a microRNA. The vector acts as a delivery system and is based on a non-disease causing adeno-associated virus (AAV) that has been changed to carry and deliver a gene encoding a microRNA that will recognize, bind and non-selectively lower the human huntingtin protein. 

The U.S. Food and Drug Administration has granted orphan drug designation for AMT-130 in Huntington’s disease and AMT-130 has received an Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency (EMA)

Number of participants: 26

study sites

Six clinical sites in the US are involved :

1 – University of California, San Francisco (San Francisco, California)
2 – University of Michigan Department of Neurology (Ann Arbor, Michigan)
3 – Ohio State University (Columbus, Ohio)
4 – The University of Texas (Houston, Texas)
5 –Virginia Commonwealth University VCU School of Medicine, Department of Neurology (Richmond, Virginia)
6 – University of Washington Medical Center (Seattle, Washington)


5 years: from September 2019 until May 2026


  • Between the ages of 25 to 65 
  • 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
  • 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) stable for 3 months prior to Screening

study design

The study will test two dose levels of AMT-130 (low dose and high dose). The safety of the low dose will be assessed before testing the high dose. A total of 26 patients will be enrolled. Of the 26 patients enrolled, 16 will receive treatment with AMT-130 (“Treated Group’) and 10 will not receive any study treatment (“Imitation Group”). Patients assigned to the Treated Group will receive the dose of AMT-130 during a neurosurgical visit.

drug administration

AMT-130 will be infused into two specific brain regions (caudate and striatum) under general anesthesia. This is done by drilling two to six small holes in the skull and administering AMT-130 by a micro-catheter. For patients assigned to the Imitation Group, small, superficial holes will be drilled into the surface of the skull under general anesthesia, but they will NOT receive a dose of AMT-130. 

estimated closing date

May 2026

the trial will be successful if

If the drug will be considered safe and well to tolerated by all participants to whom it will be administered for the duration of the entire study.


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