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NCT01574053ClinicalTrials.gov

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

מגייסמקבל משתתפים כעת, לפי רשומת המרשם.
יצירת קשר עם המחקר

בקצרה

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…

תצפיתידרושים 35,000 משתתפים183 אתרים20 מדינות

קטגוריות

נוירולוגיה45%מחלות נדירות וגנטיות45%פסיכיאטריה ובריאות הנפש10%

רשומה זו לא אושרה לאחרונה

נותן החסות אישר את מצב הרשומה הזו לאחרונה בתאריך פברואר 2024. ייתכן שהיא כבר אינה מדויקת.

הרשומה עדיין רשומה כ־מגייס, אך איש לא אישר זאת במשך שנתיים. צרו קשר עם אתר המחקר לפני שאתם מתכננים.

רשום במרשם אחד

מחקר יחיד יכול להיות רשום בכמה מרשמים. אנו מציגים אותו פעם אחת ומקשרים לכל רשומה שברשותנו.

מידע הניסוי מוצג כפי שפורסם על ידי המרשם, בשפת המקור שלו.

מתעניינים במחקר הזה?

התחברות או צרו חשבון כדי לרשום את התעניינותכם ולעקוב אחר המחקר הזה.

How this study is set up

The points below are drawn from the public registry record for this study. Each one cites the field or sentence it came from. A dash (—) means the record does not state something — not that it is missing or wrong. Many well-run studies, especially small ones, leave some of these blank.

  • Registered before enrolment beganFirst posted 2012-04-10; recorded start 2012-07-01
  • Has a defined primary outcomeA primary outcome measure is listed in the record
  • The primary outcome states a time frameThe primary outcome measure records a time frame
  • Whether participants are randomly assigned is not stated in the registry recordNo allocation method is recorded
  • Whether there is a comparison group is not stated in the registry recordThe record does not describe the study arms
  • Whether the study is blinded is not stated in the registry recordNo masking information is recorded
  • An ethics committee is not stated in the registry recordThis registry's ingested record has no ethics-committee field
  • Regulatory oversight (such as an IND or IDE) is not stated in the registry recordThis registry's ingested record has no regulatory-authorisation field
  • A data monitoring committee is not stated in the registry recordThis registry's ingested record has no data-monitoring-committee fieldA data monitoring committee is not required for many smaller studies, and its absence here is not unusual.
  • No cost to participants is mentioned in the recordChecked the summary, description and eligibility text; no cost-to-participant phrase found
  • Sponsor has 3 other studies in this databaseCounted from the lead sponsor named in the record (CHDI Foundation, Inc.)
  • No posted results from this sponsor are in this database yetBased on the sponsor’s studies in this database
  • The record lists 1 condition.
  • Lead sponsor type recorded as: other.

These are facts from the public registry record, not a judgement about the study. Discuss any trial with your own doctor before taking part.

What these facts mean, and what to ask · ruleset v1

Trial stature

Three independent measures of this study, described from its registry record — not a recommendation about it. A rigorous study by investigators nobody has heard of is better evidence than a weak study led by a famous one. How these are scored.

Methodological rigourEXPLORATORY

An exploratory-stage design for a study of this type, judged from its ClinicalTrials.gov record.

How this score is built
  • Randomised allocation0/20

    Allocation not stated in the record

  • Blinding0/20

    Masking not stated in the record

  • Control arm0/15

    No comparator arm stated in the record

  • Primary-outcome specificity10/10

    Named primary outcome with a defined time frame

  • Endpoint type3/10

    Surrogate or intermediate endpoint (conservative default)

  • Multi-centre8/8

    Multi-centre: 183 sites

  • Data monitoring committee7/7

    A data monitoring committee is in place

  • Prospective registration5/5

    Registered before the study start date

  • Protocol / SAP posted0/5

    No protocol or SAP posted to the registry

ScaleMEGA

A very large study, international in scope: 35,000 participants (target), run at 183 sites, across 20 countries.

How this score is built
  • Enrolment40/40

    35,000 participants (target)

  • Site count25/25

    183 sites

  • Country count15/15

    20 countries

  • Planned duration10/10

    Planned over about 603 months

  • Sponsor scale2/10

    CHDI Foundation, Inc. has led 4 trials in our corpus

Investigator standingUNKNOWN

We have no verifiable track record for the investigator named on this trial. That is common for early-career investigators and for records held outside ClinicalTrials.gov — it is not a negative signal.

How this score is built
  • Investigator standing0/100

    No investigator recorded in the registry for this trial

These describe the registry record only, and today we hold ClinicalTrials.gov data. Absent fields lower a score, and absence often reflects registration practice rather than study quality. A high-stature trial is not necessarily safer or a better choice for you — enrolling in a large definitive trial can mean a higher chance of receiving placebo, while a small early-phase study may be the only route to a new therapy.

תקציר

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.

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.

מצבים רפואיים

  • Huntington's Disease

זכאות

מיןהכול
גילאים18 Years – ללא מקסימום
מתנדבים בריאיםכן
שיטת דגימהNON_PROBABILITY_SAMPLE

אוכלוסיית המחקר

Patients with HD and their family members are recruited from specialty clinics (Human Genetics, Neurology, Psychiatry) that advise and treat people affected by HD. In addition, in some areas community clinics and neurologists who see HD patients recruit participants for this study. Participants also receive information about the study through websites, clinical practices, support groups, advocacy newsletters, etc. and place a direct request to be considered for participation in the study. Community controls are identified by study site staff (using advertisements, flyers and newsletters) with the support of the Enroll-HD operational staff.

זכאות כפי שנכתבה במרשם

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).

זכאות במשפטים פשוטים

הקריטריונים של רשומה זו טרם פורקו למשפטים נפרדים. טקסט המרשם שלמעלה שלם והוא הגרסה המחייבת.

תכנון המחקר

סוג המחקרתצפיתי
שלבלא צוין ברשומת המרשם
הקצאהלא צוין ברשומת המרשם
מודל ההתערבותלא צוין ברשומת המרשם
מטרה ראשיתלא צוין ברשומת המרשם
הסתרהלא צוין ברשומת המרשם
גיוסדרושים 35,000 משתתפים

נותן חסות ושותפים

  • CHDI Foundation, Inc. נותן חסות

מדדי תוצאה

  1. מדד תוצאה ראשוני

    Motor Assessments: Unified Huntington's Disease Rating Scale (UHDRS) 99 Motor, UHDRS '99 Diagnostic Confidence Level

    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.

    מסגרת זמן through study completion, an average of 1 year

  2. מדד תוצאה ראשוני

    Functional Assessments: UHDRS '99 Total Functional Capacity, UHDRS '99 Functional Assessment Scale, UHDRS '99 Independence Scale

    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.

    מסגרת זמן through study completion, an average of 1 year

  3. מדד תוצאה ראשוני

    Problem Behaviors Assessment-Short (PBA-s)

    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.

    מסגרת זמן through study completion, an average of 1 year

  4. מדד תוצאה ראשוני

    Cognitive Assessments: Symbol Digit Modality Test; Stroop Color Naming; Stroop Word Reading; Categorical Verbal Fluency

    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.

    מסגרת זמן through study completion, an average of 1 year

תאריכים

תאריך התחלה1 ביולי 2012 (בפועל)
סיום ראשוני1 בינואר 2062 (משוער)
סיום1 בינואר 2062 (משוער)
פורסם לראשונה10 באפריל 2012 (משוער)
עודכן לאחרונה28 בפברואר 2024
התוצאות פורסמולא צוין ברשומת המרשם
המצב אומת לאחרונהפברואר 2024

בפועל פירושו שהאירוע התרחש. משוער פירושו שנותן החסות צופה אותו. שתי המשמעויות שונות זו מזו.

מיקומים

160 אתרים מגייסים

Argentina

מתקןעירמדינה או אזורמצב
Instituto de Neurociencias Buenos Aires (INEBA)Buenos Airesמגייס

Australia

מתקןעירמדינה או אזורמצב
Monash UniversityMelbourneVictoriaמגייס
University of Melbourne, Royal Melbourne HospitalParkvilleמגייס
The Neurosciences Unit - North Metropolitan HospitalPerthWestern Australiaמגייס
Westmead HospitalWestmeadNew South Walesמגייס

Austria

מתקןעירמדינה או אזורמצב
Universitatsklinik InnsbruckInnsbruckמגייס

Belgium

מתקןעירמדינה או אזורמצב
Bruxelles - ErasmeBrusselsמגייס
Institut de Pathologie et de Génétique (IPG)Charleroiמגייס
University Hospitals LeuvenLeuvenVlaams-Brabantמגייס
Hôpital du Beau Vallon ASBLSaint-Servaisמגייס

Canada

מתקןעירמדינה או אזורמצב
University of Calgary, Movement Disorders ProgramCalgaryAlbertaמגייס
University of Alberta (Glenrose)EdmontonAlbertaהופסק
University of Alberta Hospital in EdmontonEdmontonAlbertaמגייס
Nova Scotia Health AuthorityHalifaxNova Scotiaמגייס
Centre for Movement DisordersMarkhamOntarioהופסק
CHUM Hospital of Notre DameMontrealQuebecמגייס
The Ottawa HospitalOttawaOntarioמגייס
North York General HospitalTorontoOntarioמגייס
University of British ColumbiaVancouverBritish Columbiaמגייס

Chile

מתקןעירמדינה או אזורמצב
CETRAMSantiagoמגייס

Colombia

מתקןעירמדינה או אזורמצב
Servicios Médicos MS, Clínica de MarlyBogotáCundinamarcaמגייס
Universidad de Antioquia, Grupo de NeurocienciasMedellínAntioquiaמגייס

Denmark

מתקןעירמדינה או אזורמצב
University Hospital of AarhusAarhusמגייס
University Hospital of CopenhagenCopenhagenמגייס
University Hospital of OdenseOdenseמגייס

France

מתקןעירמדינה או אזורמצב
CHU Amiens-PicardieAmiensמגייס
CHU Angers, Centre de Référence Maladies NeurogénétiquesAngersמגייס
Bordeaux - Huntington Disease competencies centerBordeauxמגייס
CHU Gabriel-MontpiedClermont-Ferrandמגייס
Hôpital Henri Mondor, Centre d'Investigation CliniqueCréteilמגייס
Lille-Amiens - Huntington Disease Constituent siteLilleמגייס
Hôpital La TimoneMarseilleמגייס
CHU MontpellierMontpellierמגייס
Institut du Cerveau et de la Moelle EpinièreParisמגייס
Hôpitaux Universitaires de Strasbourg Hôpital de Hautepierre-Service de NeurologieStrasbourgAlsace Lorraineמגייס
Hôpital Purpan, Centre d'Investigation CliniqueToulouseמגייס

Germany

מתקןעירמדינה או אזורמצב
University Hospital AachenAachenמגייס
University of BerlinBerlinמגייס
St. Josef und St. Elisabeth HospitalBochumמגייס
University Hospital Carl Gustav Carus DresdenDresdenמגייס
University Hospital ErlangenErlangenמגייס
University Medical Center FreiburgFreiburg im Breisgauמגייס
Paracelsus Elena Klinik KasselKasselמגייס
University Hospital Schleswig-HolsteinLübeckמגייס
University Hospital Giessen and MarburgMarburgהופסק
Technical University of MunichMunichמגייס
George-Huntington-Institut GmbHMünsterמגייס
kbo-Isar-Amper-Klinikum Taufkirchen (Vils)Taufkirchenמגייס
University Hopsital of UlmUlmמגייס
University Hospital of WuerzburgWürzburgמגייס

133 אתרים נוספים מצוינים ברשומת המרשם.

מסמכי המחקר

לא צורפו מסמכים ברשומת מרשם זו.

שינויים לאורך זמן

לא נרשמו שינויים מאז שקלטנו רשומה זו לראשונה.

שינוי נרשם בכל פעם שנותן החסות מעדכן את רשומת המרשם. מצב, תאריכים, גיוס ואתרים מופיעים כאן ככל שהם משתנים.

מרשם: ClinicalTrials.gov · NCT########

https://xmedica.io/trials/ctgov/NCT01574053