UniQure Biopharma B.V.



The European Phase Ib/II clinical trial of AMT-130 for the treatment of HD will explore the safety and proof of concept (evidence from pilot project) in 15 patients with early manifest Huntington’s disease. The gene therapy product candidate AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene.

The vector is inactive and just a vehicle  to deliver the drug. This a one-time injection directly into the brain. The trial is called, open-label, so the participants know what they are receiving. There are 2 doses, one low, one high.

Ricardo Dolmetsch presents AMT-130 updates

Ages Eligible
for Study:

25 Years to 65 Years

(Adult, Older Adult)

Sexes Eligible
for Study:


Accepts Healthy Volunteers:


  • 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) stable for 3 months prior to Screening.
  • Able and willing to provide written informed consent
  • Able and willing to comply with all procedures and study visits
  • Evidence of suicide risk
  • 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, ventriculoperitoneal or other CSF shunt, or other implanted catheter
  • 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 the surgical delivery of AMT-130 or represents a significant neurologic comorbid disorder.
  • Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study.
  • Current or recurrent disease, infection, or other significant concurrent medical condition or medications that could confound clinical and laboratory evaluations or could affect a subject’s safety or their ability to undergo the neurosurgical procedure or comply with the procedures and study visit schedule



  • TRIAL SITE: Instytut Psychiatrii I Neurologii
  • Address: Warsaw, Poland, 02-957
  • Principal Investigator: Grzegorz Witkowski, MD, PhD


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The chemical, similar to DNA, that makes up the ‘message’ molecules that cells use as working copies of genes, when manufacturing proteins.

(from HDbuzz.org)

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Inclusion Criteria

A type of eligibility criteria. These are the reasons that a person is allowed to participate in a clinical study.

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Exclusion Criteria

A type of eligibility criteria. These are reasons that a person is not allowed to participate in a clinical study.

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CAG repeat

The stretch of DNA at the beginning of the HD gene, which contains the sequence CAG repeated many times, and is abnormally long in people who will develop HD. (from HDbuzz.org)

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The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study’s objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.

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Antisense Oligonucleotide(ASO)

A type of gene silencing treatment in which specially designed DNA molecules are used to switch off a gene. (from HDbuzz.org)

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Mutant huntingtin protein. The protein produced by the faulty HD gene.