Estimated Study Completion Date: December 31, 2022
Ages Eligible for Study
25 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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
LOCATIONS
COUNTRIES
FRANCE
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
GERMANY
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
NETHERLANDS
Facility: Leiden University Medical Center
Address: Leiden, Netherlands, 2333 ZA
Principal Investigator: Susanne de Bot
UK
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