UniQure Biopharma B.V.
The European Phase Ib/II clinical trial of AMT-130 for the treatment of HD will explore the safety, proof of concept, and dosing in 15 total patients with with early manifest Huntington’s disease split into a five person manifest Huntington’s disease split into a five person, low-dose open-label(patients know what they are getting) cohort, followed by a nine patient, higher-dose open-label cohort. All patients will be dosed with AMT-130. The therapeutic goal is to inhibit the production of the mHTT. Using AAV vectors to deliver micro-RNAs directly to the brain. A one time injection.
Estimated Study Completion Date: May 2026
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