MedPath

Enroll -HD: A Prospective Registry Study in a Global Huntington's Disease Cohort

Recruiting
Conditions
Huntington's Disease
Registration Number
NCT01574053
Lead Sponsor
CHDI Foundation, Inc.
Brief Summary

Enroll-HD is a longitudinal, observational, multinational study that integrates two former Huntington's disease (HD) registries-REGISTRY in Europe, and COHORT in North America and Australasia-while also expanding to include sites in Latin America. More than 30,000 participants have now enrolled into the study. With annual assessments and no end date, Enroll-HD has built a large and rich database of longitudinal clinical data and biospecimens that form the basis for studies developing tools and biomarkers for progression and prognosis, identifying clinically-relevant phenotypic characteristics, and establishing clearly defined endpoints for interventional studies. Periodic cuts of the database are now available to any interested researcher to use in their research - visit www.enroll-hd.org/for-researchers/access-data/ to learn more.

Detailed Description

The primary objective of Enroll-HD is to develop a comprehensive repository of prospective and systematically collected clinical research data (demography, clinical features, family history, genetic characteristics) and biological specimens (blood) from individuals with manifest HD, unaffected individuals known to carry the HD mutation or at risk of carrying the HD mutation, and control research participants (e.g., spouses, siblings or offspring of HD mutation carriers known not to carry the HD mutation). Enroll-HD is conceived as a broad-based and long-term project to maximize the efficiencies of non-clinical research and participation in clinical research. With more than 150 active clinical sites in 23 countries, Enroll-HD is now the largest HD database available and is accessible to any interested researcher - visit www.enroll-hd.org/for-researchers/access-data/ to learn more.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
35000
Inclusion Criteria
  • Carriers: This group comprises the primary study population and consists of individuals who carry the HD gene expansion mutation.
  • Controls: This group comprises the comparator study population and consists of individuals who do not carry the HD expansion mutation.

These two major categories can be further subdivided into six different subgroups of eligible individuals:

  • Manifest/Motor-manifest HD: Carriers with clinical features that are regarded in the opinion of the investigator as diagnostic of HD.
  • Pre-Manifest/-Motor-manifest HD: Carriers without clinical features regarded as diagnostic of HD.
  • Genotype Unknown: This group includes a first or second degree relative (i.e., related by blood to a carrier) who has not undergone predictive testing for HD and therefore has an undetermined carrier status.
  • Genotype Negative: This group includes a first or second degree relative (i.e., related by blood to a carrier) who has undergone predictive testing for HD and is known not to carry the HD expansion mutation.
  • Family Control: Family members or individuals not related by blood to carriers (e.g., spouses, partners, caregivers).
  • Community Controls: Individuals unrelated to HD carriers who did not grow up in a family affected by HD. Data collected from community controls will be used for generation of normative data for sub-studies.

Participant status will be captured in the study database using 2 variables: 1) Investigator Determined Status: this will be based on clinical signs and symptoms and genotyping performed as part of medical care, and will be updated at every visit; and 2) Research Genotyping Status: this will be based on genotyping conducted as part of Enroll-HD study procedures. Based on research genotyping, participants will be reclassified under this variable from Genotype Unknown to 'Carriers' or 'Controls'. Investigators and participants will be blinded to this reclassification.

Exclusion Criteria
  • Individuals who do not meet inclusion criteria,
  • Individuals with choreic movement disorders in the context of a negative test for the HD gene mutation.
  • For Community Controls: those individuals with a major central nervous system disorder will be excluded (e.g. stroke, Parkinson's disease, multiple sclerosis, etc.).

Participants under 18 may be eligible to participate (if they have juvenile-onset HD).

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Problem Behaviors Assessment-Short (PBA-s)through study completion, an average of 1 year

The Problem Behavioral Assessment Short Version (PBA-s) will be used to perform behavioral assessments. This instrument measures frequency and severity of symptoms related to altered affect, thought content and coping styles.

Cognitive Assessments: Symbol Digit Modality Test; Stroop Color Naming; Stroop Word Reading; Categorical Verbal Fluencythrough study completion, an average of 1 year

Cognition will be assessed using the Categorical Verbal Fluency Test, Symbol Digit Modality Test and Stroop Color and Word Reading Test. Verbal fluency is a commonly used neuropsychological test which examines the ability to spontaneously produce words orally within a fixed time span. For category fluency, words must be produced according to semantic constraints. The measure of performance used will be the number of correctly generated words within 60 seconds.

Functional Assessments: UHDRS '99 Total Functional Capacity, UHDRS '99 Functional Assessment Scale, UHDRS '99 Independence Scalethrough study completion, an average of 1 year

The Total Functional Capacity, Functional Assessment and Independence Subscales of the UHDRS '99 will be used to assess participants' functional status. The Total Functional Capacity scale has established psychometric properties including inter-rater reliability and validity, based on radiographic measures of disease progression.

Motor Assessments: Unified Huntington's Disease Rating Scale (UHDRS) 99 Motor, UHDRS '99 Diagnostic Confidence Levelthrough study completion, an average of 1 year

The motor section of the UHDRS assesses motor features of HD with standardized ratings of oculomotor function, dysarthria, chorea, dystonia, gait, and postural stability. UHDRS 99 Motor, UHDRS Diagnostic Confidence Level.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (183)

The Ohio State University

🇺🇸

Columbus, Ohio, United States

University of Pittsburgh

🇺🇸

Pittsburgh, Pennsylvania, United States

North York General Hospital

🇨🇦

Toronto, Ontario, Canada

Northwestern University

🇺🇸

Chicago, Illinois, United States

Baylor College of Medicine

🇺🇸

Houston, Texas, United States

University of Texas Health Science Center

🇺🇸

Houston, Texas, United States

University of Washington Medical Center

🇺🇸

Seattle, Washington, United States

University of Miami

🇺🇸

Miami, Florida, United States

Indiana University

🇺🇸

Indianapolis, Indiana, United States

Cleveland Clinic - Las Vegas

🇺🇸

Las Vegas, Nevada, United States

University of California - San Diego

🇺🇸

San Diego, California, United States

University of Cincinatti

🇺🇸

Cincinnati, Ohio, United States

Duke University

🇺🇸

Durham, North Carolina, United States

Hennepin County Medical Center

🇺🇸

Minneapolis, Minnesota, United States

St. Joseph's Hospital and Medical Center

🇺🇸

Phoenix, Arizona, United States

Wayne State University

🇺🇸

Detroit, Michigan, United States

Vanderbilt University Medical Center

🇺🇸

Nashville, Tennessee, United States

University of Utah

🇺🇸

Salt Lake City, Utah, United States

Oregon Health and Science University

🇺🇸

Portland, Oregon, United States

University of Alabama

🇺🇸

Birmingham, Alabama, United States

University of South Florida, HDSA Center of Excellence at USF

🇺🇸

Tampa, Florida, United States

University of Nebraska Medical Center

🇺🇸

Omaha, Nebraska, United States

Boston University Medical Center

🇺🇸

Boston, Massachusetts, United States

University of California - Davis

🇺🇸

Sacramento, California, United States

University Hospital of Copenhagen

🇩🇰

Copenhagen, Denmark

Beth Israel Deaconess Medical Center

🇺🇸

Boston, Massachusetts, United States

University of Louisville

🇺🇸

Louisville, Kentucky, United States

University of Michigan

🇺🇸

Ann Arbor, Michigan, United States

Wake Forest School of Medicine

🇺🇸

Winston-Salem, North Carolina, United States

Hospital Universitario Ramón y Cajal

🇪🇸

Madrid, Spain

University of South Carolina

🇺🇸

Columbia, South Carolina, United States

University of California - Los Angeles

🇺🇸

Los Angeles, California, United States

University of Maryland School of Medicine

🇺🇸

Baltimore, Maryland, United States

University of California - San Francisco

🇺🇸

San Francisco, California, United States

University of California - Irvine Medical Center

🇺🇸

Irvine, California, United States

University of Connecticut

🇺🇸

Farmington, Connecticut, United States

University of Tennessee Health Science Center

🇺🇸

Memphis, Tennessee, United States

University of Wisconsin - Madison

🇺🇸

Madison, Wisconsin, United States

Cooper University Hospital

🇺🇸

Camden, New Jersey, United States

University of Chicago

🇺🇸

Chicago, Illinois, United States

Rush University Medical Center

🇺🇸

Chicago, Illinois, United States

Instituto de Neurociencias Buenos Aires (INEBA)

🇦🇷

Buenos Aires, Argentina

Bordeaux - Huntington Disease competencies center

🇫🇷

Bordeaux, France

Hôpital La Timone

🇫🇷

Marseille, France

Rutgers, the State University of New Jersey

🇺🇸

New Brunswick, New Jersey, United States

Hereditary Neurological Disease Center

🇺🇸

Wichita, Kansas, United States

University of Iowa

🇺🇸

Iowa City, Iowa, United States

Monash University

🇦🇺

Melbourne, Victoria, Australia

Georgetown University

🇺🇸

Washington, District of Columbia, United States

Johns Hopkins University

🇺🇸

Baltimore, Maryland, United States

University of Illinois College of Medicine at Rockford

🇺🇸

Rockford, Illinois, United States

Loma Linda Medical Center

🇺🇸

Loma Linda, California, United States

Cenexel Rocky Mountain Clinical Research, LLC

🇺🇸

Englewood, Colorado, United States

Emory University, Wesley Woods Health Center

🇺🇸

Atlanta, Georgia, United States

Columbia University

🇺🇸

New York, New York, United States

Virginia Commonwealth University Parkinson's and Movement Disorders Center

🇺🇸

Henrico, Virginia, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

Albany Medical College

🇺🇸

Albany, New York, United States

University of Rochester

🇺🇸

Rochester, New York, United States

University of Virginia

🇺🇸

Charlottesville, Virginia, United States

Washington University in St. Louis

🇺🇸

Saint Louis, Missouri, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

CHUM Hospital of Notre Dame

🇨🇦

Montreal, Quebec, Canada

CHU Angers, Centre de Référence Maladies Neurogénétiques

🇫🇷

Angers, France

Hôpital Henri Mondor, Centre d'Investigation Clinique

🇫🇷

Créteil, France

Sanford Health

🇺🇸

Fargo, North Dakota, United States

University of Vermont

🇺🇸

Burlington, Vermont, United States

Georgia Health Sciences University

🇺🇸

Augusta, Georgia, United States

Institut de Pathologie et de Génétique (IPG)

🇧🇪

Charleroi, Belgium

Hôpital du Beau Vallon ASBL

🇧🇪

Saint-Servais, Belgium

Servicios Médicos MS, Clínica de Marly

🇨🇴

Bogotá, Cundinamarca, Colombia

Hôpitaux Universitaires de Strasbourg Hôpital de Hautepierre-Service de Neurologie

🇫🇷

Strasbourg, Alsace Lorraine, France

CHU Gabriel-Montpied

🇫🇷

Clermont-Ferrand, France

University Hospital Aachen

🇩🇪

Aachen, Germany

University Medical Center Freiburg

🇩🇪

Freiburg, Germany

George-Huntington-Institut GmbH

🇩🇪

Münster, Germany

IRCCS Istituto delle Scienze Neurologiche di Bologna

🇮🇹

Bologna, Italy

Università Cattolica del Sacro Cuore

🇮🇹

Rome, Italy

Azienda Ospedaliera Sant'Andrea

🇮🇹

Rome, Italy

Clinical Trial Center Maastricht B.V.

🇳🇱

Maastricht, Netherlands

Universidad de Antioquia, Grupo de Neurociencias

🇨🇴

Medellín, Antioquia, Colombia

University of Melbourne, Royal Melbourne Hospital

🇦🇺

Parkville, Australia

Universitatsklinik Innsbruck

🇦🇹

Innsbruck, Austria

CHU Amiens-Picardie

🇫🇷

Amiens, France

CHU Montpellier

🇫🇷

Montpellier, France

University Hospital Carl Gustav Carus Dresden

🇩🇪

Dresden, Germany

Paracelsus Elena Klinik Kassel

🇩🇪

Kassel, Germany

University of Calgary, Movement Disorders Program

🇨🇦

Calgary, Alberta, Canada

University of Alberta (Glenrose)

🇨🇦

Edmonton, Alberta, Canada

University of Alberta Hospital in Edmonton

🇨🇦

Edmonton, Alberta, Canada

University of British Columbia

🇨🇦

Vancouver, British Columbia, Canada

The Neurosciences Unit - North Metropolitan Hospital

🇦🇺

Perth, Western Australia, Australia

Booth Gardner Parkinson's Care Center (Evergreen Health)

🇺🇸

Kirkland, Washington, United States

Institut du Cerveau et de la Moelle Epinière

🇫🇷

Paris, France

The Ottawa Hospital

🇨🇦

Ottawa, Ontario, Canada

University Hospitals Leuven

🇧🇪

Leuven, Vlaams-Brabant, Belgium

Bruxelles - Erasme

🇧🇪

Bruxelles, Belgium

Centre for Movement Disorders

🇨🇦

Markham, Ontario, Canada

CETRAM

🇨🇱

Santiago, Chile

University Hospital of Aarhus

🇩🇰

Aarhus, Denmark

University Hospital of Odense

🇩🇰

Odense, Denmark

Lille-Amiens - Huntington Disease Constituent site

🇫🇷

LILLE Cedex, France

St. Josef und St. Elisabeth Hospital

🇩🇪

Bochum, Germany

University Hospital Erlangen

🇩🇪

Erlangen, Germany

University Hospital of Wuerzburg

🇩🇪

Würzburg, Germany

Beaumont Hospital

🇮🇪

Dublin 9, Republic Of Ireland, Ireland

Università degli Studi di Genova (DINOGMI)

🇮🇹

Genoa, Italy

Fondazione IRCCS Istituto Neurologico Carlo Besta - Neurology

🇮🇹

Milan, Italy

Lega Italiana Ricerca Huntington (LIRH)

🇮🇹

Rome, Italy

Nova Scotia Health Authority

🇨🇦

Halifax, Nova Scotia, Canada

Westmead Hospital

🇦🇺

Westmead, New South Wales, Australia

Hôpital Purpan, Centre d'Investigation Clinique

🇫🇷

Toulouse, France

Technical University of Munich

🇩🇪

Munich, Germany

kbo-Isar-Amper-Klinikum Taufkirchen (Vils)

🇩🇪

Taufkirchen, Germany

University Hopsital of Ulm

🇩🇪

Ulm, Germany

Bari Policlinico General Hospital

🇮🇹

Bari, Italy

Medical University of Warsaw

🇵🇱

Warsaw, Poland

Hospital Prof Doutor Fernando Fonseca

🇵🇹

Amadora, Portugal

Hospital Universitário de São João

🇵🇹

Porto, Portugal

Hospital Universitari de Bellvitge

🇪🇸

Barcelona,, Spain

Hospital Son Espases

🇪🇸

Palma De Mallorca, Spain

North Bristol NHS Trust

🇬🇧

Bristol, UK, United Kingdom

University of Aberdeen

🇬🇧

Aberdeen, United Kingdom

Leeds Teaching Hospitals

🇬🇧

Leeds, United Kingdom

The Walton Centre

🇬🇧

Liverpool, United Kingdom

Oxford Radcliffe Hospitals

🇬🇧

Oxford, United Kingdom

University of Berlin

🇩🇪

Berlin, Germany

Fundacion Hospital Universitario La Fe

🇪🇸

Valencia, Spain

University Hospital Giessen and Marburg

🇩🇪

Marburg, Germany

Leiden University Medical Centre

🇳🇱

Leiden, Netherlands

Royal Devon and Exeter NHS Foundation Trust

🇬🇧

Exeter, Devon, United Kingdom

Greater Glasgow Health Board

🇬🇧

Glasgow, United Kingdom

NHS Forth Valley

🇬🇧

Larbert, United Kingdom

Northumberland, Tyne & Wear NHS Foundation Trust of St. Nicholas Hospital

🇬🇧

Newcastle-upon-Tyne, United Kingdom

St. Andrews Healthcare

🇬🇧

Northampton, United Kingdom

Poole Hospital

🇬🇧

Poole, United Kingdom

Sheffield Children's NHS Foundation Trust

🇬🇧

Sheffield, United Kingdom

Fundación Jiménez Díaz

🇪🇸

Madrid, Spain

Guy's and St. Thomas's NHS Foundation Trust

🇬🇧

London, United Kingdom

University Hospital Schleswig-Holstein

🇩🇪

Lübeck, Germany

Fondazione IRCCS Istituto Neurologico Carlo Besta - Genetics

🇮🇹

Milan, MI, Italy

University of Naples

🇮🇹

Napoli, Italy

University Medical Center Groningen

🇳🇱

Groningen, Netherlands

Copernicus Podmiotem Leczniczym sp. z o.o.

🇵🇱

Gdansk, Poland

Institute of Psychiatry and Neurology, Warsaw

🇵🇱

Warsaw, Poland

Hospital Infanta Cristina, Badajoz

🇪🇸

Badajoz, Spain

Hospital de Cruces

🇪🇸

Barakaldo, Spain

Siloah AG

🇨🇭

Gümligen, Switzerland

University Hospital Zurich

🇨🇭

Zürich, Switzerland

Leicestershire Partnership

🇬🇧

Narborough, Leicestershire, United Kingdom

Fife Health Board

🇬🇧

Kirkcaldy, UK, United Kingdom

Betsi Cadwaladr University Health Board

🇬🇧

Wrexham, UK, United Kingdom

Hospital de la Santa Creu i Sant Pau

🇪🇸

Barcelona, Spain

Auckland City Hospital

🇳🇿

Auckland, New Zealand

New Zealand Brain Research Institute at University of Otago

🇳🇿

Christchurch, New Zealand

Krakow - Krakowska Akademia Neurologii

🇵🇱

Kraków, Poland

Hospital Universitário de Coimbra

🇵🇹

Coimbra, Portugal

Hospital Universitario de Burgos

🇪🇸

Burgos, Spain

Complejo Hospitalario de Navarra

🇪🇸

Pamplona, Spain

Plymouth Hospitals NHS Trust

🇬🇧

Plymouth, United Kingdom

North Staffordshire Combined Healthcare NHS Trust

🇬🇧

Stoke-on-Trent, United Kingdom

Avon and Wiltshire Mental Health Partnership

🇬🇧

Swindon, United Kingdom

Central Manchester University Hospitals NHS Foundation Trust

🇬🇧

Manchester, United Kingdom

Wellington Hospital

🇳🇿

Wellington, New Zealand

Krakowska Akademia Neurologii Sp. z o.o

🇵🇱

Katowice, Poland

Solumed Centrum Medyczne

🇵🇱

Poznan, Poland

Central Lisbon Hospital

🇵🇹

Lisbon, Portugal

Hospital de Santa Maria / Instituto de Medicina Molecular

🇵🇹

Lisbon, Portugal

Cardiff University

🇬🇧

Cardiff, United Kingdom

Tayside Health Board

🇬🇧

Dundee, United Kingdom

Lothian Health Board

🇬🇧

Edinburgh, United Kingdom

Royal Berkshire NHS Foundation Trust

🇬🇧

Reading, United Kingdom

St. George's Hospital

🇬🇧

London, United Kingdom

Hospital Mare de Déu de la Mercè

🇪🇸

Barcelona, Spain

University of Cambridge

🇬🇧

Cambridge, United Kingdom

Ayrshire Central Hospital

🇬🇧

Kilmarnock, United Kingdom

University Hospital Southampton

🇬🇧

Southampton, United Kingdom

University College of London

🇬🇧

London, United Kingdom

University of Florida Board of Trustees

🇺🇸

Gainesville, Florida, United States

Virginia Commonwealth University

🇺🇸

Richmond, Virginia, United States

Fundacion Clinic per a La Recerca Biomèdica

🇪🇸

Barcelona, Spain

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

Birmingham and Solihull Mental Health NHS Foundation Trust

🇬🇧

Birmingham, United Kingdom

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