Not yet recruiting


PTC Therapeutics



Estimated Study Completion Date: December 31, 2022

Ages Eligible for Study

25 Years and older (Adult, Older Adult)

Sexes Eligible for Study:


Accepts Healthy Volunteers:


Inclusion Criteria:

  • Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length from 42 to 50, inclusive
  • A Unified Huntington’s Disease Rating Scale (UHDRS)-Independence Scale (IS) score of 100
  • A UHDRS Total Functional Capacity (TFC) score of 13
  • A score between 0.18 and 4.93 inclusive on the normed version of the HD prognostic index (PINHD)

Exclusion Criteria:

  • Receipt of an experimental agent within 90 days or 5 half-lives prior to Screening or anytime over the duration of this study, ribonucleic acid (RNA)- or deoxyribonucleic acid (DNA)-targeted HD-specific investigational agents such as antisense oligonucleotides, cell transplantation, or any other experimental brain surgery
  • Any history of gene therapy exposure for the treatment of HD
  • Participation in an investigational study or investigational paradigm (such as exercise/physical activity, cognitive therapy, brain stimulation, etc) within 90 days prior to Screening or anytime over the duration of this study
  • Any medical history of brain or spinal disease that would interfere with the lumbar puncture process safety assessments
  • Any medical history or condition that would interfere with the ability to complete the protocol-specified assessments (for example, implanted shunt, conditions precluding magnetic resonance imaging [MRI] scans)
  • Pregnancy, planning on becoming pregnant during the course of the study or within 6 months of end of treatment, or currently breastfeeding




  • Facility: Centre Hospitalier Universitaire d’Angers
  • Address: Angers, France, 49100
  • Principal Investigator: Christophe Verny   
  • Facility: Hôpital Universitaire de Marseille Hôpital de la Timone
  • Address: Marseille, France, 13385
  • Principal Investigator: Jean-Philippe Azulay
  • Facility: Brain and Spine Institute Paris
  • Address: Paris, France, 75013
  • Principal Investigator: Alexandra Durr 


  • Facility: Ruhr-Univ. Bochum St. Joseph-Hospital
  • Address: Bochum, Germany, 44791
  • Principal Investigator: Carsten Saft    
  • Facility: George-Huntington-Institut
  • Address: Münster, Germany, 48149
  • Principal Investigator: Ralf Reilmann  
  • Facility: Ulm University, UKU, Dep. of Neurology
  • Address: Ulm, Germany, 89081
  • Principal Investigator: Bernhard Landwehrmeyer 


  • Facility: Leiden University Medical Center
  • Address: Leiden, Netherlands, 2333 ZA
  • Principal Investigator: Susanne de Bot


  • Facility: Cardiff University Schools of Medicine and Biosciences
  • Address: Cardiff, United Kingdom, CF10 3AX
  • Principal Investigator: Anne Rosser
  • Facility: UCL Queen Square Institute of Neurology National Hospital for Neurology and Neurosurgery
  • Address: London, United Kingdom, WC1N 3BG
  • Principal Investigator: Sarah Tabrizi