PROOF-HD

Завершено

ПОДРОБНЕЕ ОБ ЭТОМ

СПОНСОР

Prilenia Neurotherapeutics & Huntington Study Group

КОЛИЧЕСТВО УЧАСТНИКОВ

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PROOF-HD — это клиническое исследование 3-й фазы, которое оценивает эффективность и безопасность придопидина у пациентов с ранней стадией болезни Гентингтона. Придопидин — это малая молекула, разработанная компанией Prilenia для лечения нейродегенеративных заболеваний, таких как болезнь Гентингтона.

По данным компании Prilenia, придопидин — это пероральный препарат, принимаемый в маленькой капсуле, которую легко проглотить дважды в день, который попадает в головной и спинной мозг, где активирует белок, называемый рецептором сигма-1 (S1R). Активация S1R придопидином улучшает очищение организма от токсичных белков, повышает выработку энергии, снижает клеточный стресс и воспаление. Эти механизмы имеют решающее значение для функционирования и выживания нейрона.

Предполагаемое первичное завершение: 3 марта 2023 года

Вебинар: все, что вам нужно
знать о PROOF HD

Доктор Майкл Хайден представляет
фазу 3 PROOF-HD

Возраст, подходящий для исследования:

25 лет и старше

Пол, допущенный к исследованию:

Все

Принимает здоровых добровольцев:

Нет

Критерии включения

  1. Диагностика БГ на основании клинических признаков и наличия ≥36  ЦАГ (CAG) -повторов в гене гентингтине
  2. Уровень диагностической уверенности (DCL) 4
  3. Взрослая форма БГ с появлением признаков и симптомов в возрасте ≥18 лет
  4. БГ 1 или 2 стадии, определенная по шкале UHDRS-TFC ≥7 баллов, при скрининге

Критерии исключения

  1. Применение придопидина в течение 12 месяцев до базового визита.
  2. Генная терапия
  3. Любое серьезное медицинское состояние или клинически значимое отклонение лабораторных или жизненных показателей, которое препятствует безопасному участию пациента в исследовании и его завершению, например, серьезное заболевание сердца в течение 12 недель до исходного уровня или история некоторых сердечных аритмий
  4. История эпилепсии или судорог в течение последних 5 лет
  5. Беременность или кормление грудью, или намерение забеременеть во время исследования

Страны

Страны

Австрия

TRIAL SITE: Medizinische Universität Innsbruck

Adress: Innsbruck

Contact: Dora Valent, Study Site Coordinator
Phone: 0512 504 83237
Email: dora.valent@tirol-kliniken.at

Канада

TRIAL SITE: University of Calgary

Adress: Calgary

Contact: Lorelei Tainsh, Study Site Coordinator
Phone: (403) 220-8413
Email: derwent@ucalgary.ca

TRIAL SITE:Halifax Infirmary QEII Health Services Center

Adress: Halifax

Contact: Madie El-aghil, Study Site Coordinator
Phone: (902) 431-8783 Ext: 115
Email: madie.el-aghil@truenorthcr.com

TRIAL SITE:University of British Columbia

Adress: Vancouver

Contact: Mike Adurogbangba, Study Site Coordinator
Email: madurogbangba@cmmt.ubc.ca
Phone: (604) 822-4872

TRIAL SITE: CHUM Hospital of Notre Dame

Adress: Montreal

Contact: Vicky Thiffault, Study Site Coordinator
Phone: (514) 890-8000, ext 15921
Email: vicky.thiffault.chum@ssss.gouv.qc.ca

Чешская Республика

TRIAL SITE: Centrum extrapyramidových onemocnění

Adress: Prague

Contact: Jiri Klempir, MD, Study Site Investigator
Tel:   +420  732 273 271
Email:  Jiri.Klempir@seznam.cz

Франция

TRIAL SITE: Centre Hospitalier Universitaire (CHU) Henri Mondor

Adress: Paris

Contact: Tiffany Monnier, Study Site Coordinator
Tel. 01.49.81.37.93

TRIAL SITE:Hôpitaux Universitaires de Marseille Timone

Adress: Marseille

Contact: Email: Laura Laura.MUNDLER@ap-hm.fr

TRIAL SITE: Centre Hospitalier Universitaire (CHU) of Lille – Hôpital Roger Salengro

Adress: Lille

Contact: Eric Decorte, Study Site Coordinator
Email: eric.decorte@chru-lille.fr

Германия

TRIAL SITE: Euregional Huntington Center Aachen (EHZA) 

Adress: Aachen

Contact: Maha Sagar, Study Site Coordinator, Email: NeuroStudien@ukaachen.de Tel: +49 241 80 80697

TRIAL SITE: George Huntington Institut

Adress: Münster

Contact: Tel: +49 251 7887880
Email: info@ghi-muenster.de

TRIAL SITE: Huntington Center North Rhine-Westphalia,
Department of Neurology,
Ruhr-University Bochum,
St. Josef-Hospital Bochum

Adress: Bochum

Contact: Email: d.kaminski@klinikum-bochum.de
Tel.: +49-234-509-2703

TRIAL SITE: Kbo-Isar-Amper-Klinikum Taufkirchen (Vils)

Adress: Taufkirchen (Vils)

Contact: Michael Bachmaier, Study Site Coordinator
Email: Michael.Bachmaier@kbo.de
Tel: 08084-934-417

TRIAL SITE: University of Lübeck

Adress: Lübeck

Contact: Saruhi Surnaschjan, Study Site Coordinator 
Phone:+49 451 50043497
Email: saruhi.surnaschjan@neuro.uni-luebeck.de

TRIAL SITE: Ulm University

Adress: Ulm

Contacts:  
Sonja Trautmann
Tel:  +49 731 50063083
 
Ariane Schneider
Tel: +49 731 50063126 

Италия

TRIAL SITE: IRCCS Istituto delle Scienze Neurologiche di Bologna

Adress: Bologna

Contact: Tel: +393387367104
Email: unitastudiclinici@ausl.bologna.it

TRIAL SITE:Universita’ di Bari

Adress: Bari

TRIAL SITE: Fondazione IRCCS Istituto Neurologico Carlo Besta

Adress: Milano

Contact: Tel: +39 02 23942519
Email: centroHD@istituto-besta.it

TRIAL SITE:Università di Napoli Federico II

Adress: Naples

Contact: Email: centrohd.unina@gmail.com
Tel: +39 081 5455213

TRIAL SITE: CSS-Mendel Institute at IRCCS Casa Sollievo della Sofferenza Research Hospital

Adress: Roma

Contact: Tel: +39 06 44700887
Email: info@lirh.it

Нидерланды

TRIAL SITE: Maastricht University

Adress: Maastricht 

Contact: Dr. Mayke Oosterloo, Study Site Investigator
Tel: 0031-(0)43-387 76 76, Email: expertisecentrumhuntington@mumc.nl

TRIAL SITE:Leiden University Medical Center

Adress: Leiden

Contact: Tel: 0031715265442 
Email: huntington@lumc.nl

Польша

TRIAL SITE: Szpital Specjalistyczny Swietego Wojciecha

Adress: Gdansk

Contact: Agnieszka Konkel, Study Site Coordinator
Tel: +48 609 952 555
Email: konkel1989@gmail.com

TRIAL SITE: Krakowska Akademia Neurologii

Adress: Krakow

Contact: Email: centrum@neurologia.org.pl 
Tel: +48 12 426 92 80

TRIAL SITE:Instytut Psychiatrii i Neurologii

Adress: Warsaw

Contact: Grzegorz Witkowski, Study Site Investigator
Email: gwitkowski@ipin.edu.pl
Phone: +48694904208

Испания

TRIAL SITE:Hospital de la Santa Creu i Sant Pau

Adress: Barcelona

Contact: Dr. Jaime Kulisevsky
Email: JKulisevsky@santpau.cat
Tel: +34 649 14 23 60

TRIAL SITE: Burgos Foundation for Health Research

Adress: Burgos

Contact: Tel:  +34 947 28 18 00 ext 35380
Email: jessicajrp@hubu.es

mcubo@saludcastillayleon.es

TRIAL SITE: Fundacion Hospital Universitario La Fé

Adress: Valencia

Contact: Francisco Castera, Study Site Coordinator
Email: franciscocasteraenroll@gmail.com

Carmen PEIRO, Study Site Investigator. Email: cpeirov@gmail.com
Tel: Phone: +34 682893185

TRIAL SITE:Hospital Ramón y Cajal

Adress: Madrid

Contact: Email: joselopezsendon@hotmail.com
Tel: +34  638 158 849

Великобритания

TRIAL SITE: University of Aberdeen, Institute of Medical Sciences

Adress: Aberdeen (Scotland)

Contact: Stella Sihlabela, Study Site Coordinator
Tel: +44 1224 552120
Email: stella.sihlabela@nhs.scot

Resifina Seyara, Study Site Coordinator
Tel: +44 1224 552120
Email: resifina.seyara@nhs.scot

TRIAL SITE: Barberry National Centre for Mental Health

Adress: Birmingham (England)

TRIAL SITE: Cardiff University

Adress: Cardiff (Wales)

Contact:  Alison Johnson, Study Site Coordinator
Email: Alison.Johnson@wales.nhs.uk

TRIAL SITE: Newcastle upon Tyne Hospitals NHS Foundation

Adress:Newcastle (England)

Соединенные Штаты

TRIAL SITE: University of California, Davis (UC Davis) Medical Center

Adress: Sacramento

Contact: Becky Craig, Study Site Coordinator
Phone: (916) 734-3541
Email: rscraig@ucdavis.edu

TRIAL SITE:Georgetown University

Adress: Washington

Contact: Robin Kuprewicz, Study Site Coordinator
Phone: (202) 893-1115
Email: rk1028@georgetown.edu

TRIAL SITE: University of California, San Diego (UCSD)

Adress: San Diego

Contact: Email: jcoreybloom@ucsd.edu
Phone: (858) 249-0569

TRIAL SITE: University of Florida

Adress: Gainesville

Contact: Kyle Rizer, Study Site Coordinator
Phone: (352) 733-2426
Email: kyle.rizer@neurology.ufl.edu

TRIAL SITE: Rocky Mountain Movement Disorder Center

Adress: Englewood

Contact: Jessica Jaynes, Study Site Coordinator
Phone: (303) 357-5456

TRIAL SITE:University of South Florida

Adress: Tampa

Contact: Kelly (Kolleen) Elliott, Study Site Coordinator
Email: kelliot@usf.edu  

TRIAL SITE: Emory University

Adress: Atlanta

Contact: Elaine Sperin, Study Site Coordinator
Email: esperin@emory.edu

TRIAL SITE: Hereditary Neurological Disease Centre

Adress: Wichita

Contact:Gregory Suter, Study Site Coordinator
Phone: (888) 232-4632

TRIAL SITE: Northwestern University

Adress: Chicago

Contact:ZsaZsa Brown, Study Site Coordinator
Phone: (312) 503-4121
Email: zsazsa.brown@northwestern.edu

TRIAL SITE: University of Louisville

Adress: Louisville

Contact: Annette Robinson, Study Site Coordinator
Email: annette.robinson@louisville.edu

TRIAL SITE: University of Kansas Medical Center

Adress: Kansas City

Contact: Carolyn Gray, Study Site Coordinator
Phone: (913) 588-6983
Email: cgray1@kumc.edu

TRIAL SITE: Johns Hopkins University

Adress: Baltimore

Contact: Kia Ultz, Study Site Coordinator
Phone: (410) 955-1349
Email: kcarte23@jhmi.edu

TRIAL SITE:Beth Israel Deaconess Medical Center

Adress: Boston

Contact: Sophie Antonioli, Study Site Coordinator
Phone: (617) 667-2355
Email: hdresearch@bidmc.harvard.edu

TRIAL SITE: Albany Medical College

Adress: Albany

Contact: Email: evanss@amc.edu

TRIAL SITE:Massachusetts General Hospital

Adress: Boston

Contact:Dr. Diana Rosas, Study Site Investigator
Phone: (617) 726-9045

TRIAL SITE:Columbia University

Adress: New York City

Contact: Miles DeGrazia, Study Site Coordinator
Email: MQD2103@cumc.columbia.edu

TRIAL SITE:Washington University, St. Louis

Adress: St. Louis

Contact: Matthew Lewis, Study Recruitment
Phone: (314) 747-5916
Email: lmatthew@wustl.edu

TRIAL SITE: Duke University

Adress: Durham

Contact: McKenzie Luxmore, Study Site Coordinator
Email: mckenzie.luxmore@duke.edu
Phone: (919) 684-0865

TRIAL SITE: University of Cincinnati

Adress: Cincinnati

Contact:Christina Gruenwald, Study Site Coordinator
Phone: (513) 558-0112
Email: gruenwcm@ucmail.uc.edu

TRIAL SITE: University of Pennsylvania

Adress: Philadelphia

Contact: Jennifer Klapper, Study Site Coordinator
Phone: (215) 829-5176

TRIAL SITE: Ohio State University

Adress: Columbus

Contact: Casey Mitchell, Study Site Coordinator
Email: casey.mitchell@osumc.edu
Phone:  (614) 685-9906

Katherine Ambrogi, Study Site Coordinator
Email: katherine.ambrogi@osumc.edu
Phone: (614) 688-6685

TRIAL SITE: Vanderbilt University Medical Center

Adress: Nashville

Contact: Dr. Daniel Claassen, Study Site Investigator
Danielle Buchanan, Study Site Coordinator
Email: danielle.a.buchanan@vumc.org
Phone: (615) 875-3274

TRIAL SITE: Oregon Health & Sciences University

Adress: Portland

Contact: Keenan Ashby, Study Site Coordinator
Phone: (503) 494-7245
Email: ashbyk@ohsu.edu

TRIAL SITE: University of Texas, Houston

Adress: Houston

Contact: Jamie Sims, Study Site Coordinator
Phone: 713-500-7763
Email: Jamie.Sims@uth.tmc.edu

TRIAL SITE:Virginia Commonwealth University

Adress: Richmond

Contact:Kelly Huckstep, Study Site Coordinator
Phone: (804) 965-4283
Email: kelly.huckstep@vcuhealth.org

TRIAL SITE: University of Washington

Adress:Seattle

Contact:Debra Del Castillo, Study Site Coordinator
Phone: (206) 543-3647
Email: debradel@uw.edu

TRIAL SITE: University of Texas, Houston

Adress: Houston

Contact: Jamie Sims, Study Site Coordinator
Phone: 713-500-7763
Email: Jamie.Sims@uth.tmc.edu

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phenoptype

Phenotype refers to an individual’s observable traits, such as height, eye color and blood type. A person’s phenotype is determined by both their genomic makeup (genotype) and environmental factors.

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oxidative seres

an imbalance between unstable molecules called «free radicals» and protective «antioxidants» in your body

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Metabolism & bioenergetics

describe how your body turns food into fuel and uses that energy to live. 

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Small Molecule

a tiny chemical compound, much smaller than big biological structures like proteins, that can easily travel inside our cells to act as medicine (like aspirin or ibuprofen), a building block (like glucose), or a signaling tool in the body, often taken as pills because they’re easy to absorb and distribute

 

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Nucleic acid

(DNA and RNA) are the essential information-carrying molecules in all life, acting like blueprints that store and transmit genetic instructions for building and operating cells, directing everything from growth to protein production, and passing traits from parents to offspring.

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SNP-single nucleotide polymorphisms

a single-letter spelling difference in a gene. SNPs, pronounced ‘snips’, are common and most don’t change the function of the gene.

 
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at risk

You do not know if you carry the genetic mutation for HD gene 

 
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TFC-total functional capacity

A standardized rating scale for function in HD, used to assess capacity to work, handle finances, perform domestic chores and self-care tasks.
Scores range from 0 to 13, with higher scores indicating better functional capacity. 

 
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Double-blinded

 means that neither the participant nor the clinical trial doctor can choose or know the group the participant is in until the trial is over. This approach helps to prevent bias.

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Open label

A trial in which the patient and doctor know what drug is being used. Open label trials are susceptible to bias through placebo effects.

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Gene therapy

a technique that aims to treat or prevent diseases by modifying a person’s genes. It involves introducing, removing, or changing genetic material (DNA or RNA) within a patient’s cells.

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UHDRS- Unified Huntington Disease Rating Scale

A standardized neurological examination that aims to provide a uniform assessment of the clinical features of HD

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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

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Wild-type

the opposite of ‘mutant’. Wild-type huntingtin, for example, is the ‘normal’, ‘healthy’ protein

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Tolerabilty

How well a person can handle a treatment without having serious or uncomfortable side effects.

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Striatum

Part of the brain that  coordinates multiple aspects of cognition, including both motor and action planning, decision-making, motivation, reinforcement, and reward system.

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Randomized allocation

A type of allocation strategy in which participants are assigned to the arms of a clinical trial by chance.

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Radioligand

a radioactive substance that binds to a specific target in the body, allowing visualization of that target’s distribution and activity

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Protein

Protein builds, maintains, and replaces the tissues in your body. The building blocks of life.

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Premanifest / Prodromal

Prior to onset or diagnosis of movement symptoms.

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Placebo

A placebo is a dummy medicine containing no active ingredients. The placebo effect is a psychological effect that causes people to feel better even if they’re taking a pill that doesn’t work.

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PK - Pharmacokinetics

The movement of drugs through the body

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PD - Pharmacodynamics

The body’s biological response to drugs

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PET scan

Positron emission tomography which produces detailed 3-dimensional images of the inside of the body.

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Neuron

Brain cells that store and transmit information

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MRI

Magentic resonance imaging: A technique using powerful magnetic fields to produce detailed images and visualizes the structure of organs, tissues, and bones 

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mHTT

Mutant huntingtin protein. The protein produced by the faulty HD gene.

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Manifest

after HD diagnosis, or when symptoms are already showing

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Longitudinal study

A study where each participant is looked at several times over a time period – unlike a cross-sectional study, where each participant is looked at only once

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HTT

one abbreviation for the gene that causes Huntington’s disease. The same gene is also called HD and IT-15

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fMRI

functional MRI:As with MRI, a technique using powerful magnetic fields  but focusing on brain function by measuring and mapping changes in blood flow, revealing which areas of the brain are active during specific tasks or cognitive processes

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CSF - cerebrospinal fluid

A clear fluid produced by the brain, which surrounds and supports the brain and spinal cord.

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Efficacy

A measure of whether a treatment works or not

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ASO(Antisense oligonucleotides)

A type of gene silencing treatment in which specially designed DNA molecules are used to switch off a gene

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Biomarker

a test of any kind – including blood tests, thinking tests and brain scans – that can measure or predict the progression of a disease like HD. Biomarkers may make clinical trials of new drugs quicker and more reliable

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BDNF

Brain-derived neurotrophic factor: a growth factor that may be able to protect neurons in HD.

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Allele

one of the two copies of a gene

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Plasma

Liquid component of the blood.

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Gene

The basic unit of heredity passed from parent to child. Genes are made up of sequences of DNA and are arranged, one after another, at specific locations on chromosomes in the nucleus of cells.

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Phase

Clinical trial phases are different stages of research that assess the safety and effectiveness of a new medical treatment or intervention in humans.

Each phase has a specific goal and involves a different number of participants. Generally, there are 4 phases (I-IV), with Phase I focusing on safety and dosage, Phase II on efficacy and side effects, Phase III on comparing the new treatment with standard treatments, and Phase IV on long-term safety monitoring.