PIVOT-HD
Not yet recruiting
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
- Address: London, United Kingdom, WC1N 3BG
- Principal Investigator: Sarah Tabrizi