The PIVOT-HD study is a PhaseII trial looking at how safety is PTC518, a new Huntington-Lowering drug, in patients with HD. PTC518 is an oral tablet (taken by mouth every day) that not only tries to lower huntingtin (mHTT) in the brain but also treat HD everywhere in the body (brain, muscle, immune system…).
This Phase 2 is hoping to enrol 162 participants. 2 doses will be given and also a placebogroup over 12 weeks and a follow-up of 12 months.
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
AUSTRIA
Facility: Medizinische Universität Innsbruck
Address: Christoph-Probst-Platz 1, Innrain 52 A, Fritz-Pregl-Straße 3, 6020 Innsbruck, Austria
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
ITALY
Facility: IRCCS Istituto delle Scienze Neurologiche di Bologna
Address: Padiglione G, Via Altura, 3, 40139 Bologna BO, Italy
Facility: IRCCS Carlo Besta Neurological Institutte
Address: Via Giovanni Celoria, 11, 20133 Milano MI, Italy
Facility: IRCCS Casa Sollievo della Sofferenza Research Hospital
Address: Viale Cappuccini, sn, 71013 San Giovanni Rotondo FG, Italy
NETHERLANDS
Facility: Leiden University Medical Center
Address: Leiden, Netherlands, 2333 ZA
Principal Investigator: Susanne de Bot
SPAIN
Facility: Hospital Universitario Cruces
Address: Cruces Plaza, s/n, 48903 Barakaldo, Bizkaia
Facility: Hospital de la Santa Creu i Sant Pau
Address: C/ de Sant Quintí, 89, Horta-Guinardó, 08025 Barcelona
Facility: Hospital Universitario de Burgos
Address: Av. Islas Baleares, 3, 09006 Burgos
Facility: Hospital Universitario Ramón y Cajal
Address: M-607, 9, 100, Fuencarral-El Pardo, 28034 Madrid
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
Facility: The Barberry National Centre For Mental Health
Address: 6 Bullace Croft, Birmingham B15, United Kingdom
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)
dictionary:
Inclusion Criteria
A type of eligibility criteria. These are the reasons that a person is allowed to participate in a clinical study.
dictionary:
Exclusion Criteria
A type of eligibility criteria. These are reasons that a person is not allowed to participate in a clinical study.
dictionary:
Placebo
An inactive substance or treatment that looks the same as, and is given in the same way as, an active drug or intervention/treatment being studied.
The placebo effect is a psychological effect that causes people to feel better even if they’re taking a pill that doesn’t work. (from HDbuzz.org)
dictionary:
mHTT
Mutant huntingtin protein. The protein produced by the faulty HD gene.
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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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Phase
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.