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NCT01574053ClinicalTrials.gov

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

En recrutementAccepte des participants actuellement, selon la fiche du registre.
Contacter cette étude

En bref

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…

Observationnelle35 000 participants recherchés183 sites20 pays

Catégories

Neurologie45%Maladies rares et génétiques45%Psychiatrie et santé mentale10%

Cette fiche n'a pas été confirmée récemment

Le promoteur a confirmé pour la dernière fois le statut de cette fiche en février 2024. Elle n'est peut-être plus exacte.

La fiche indique toujours En recrutement, mais personne ne l'a confirmé depuis 2 ans. Contactez le site de l'étude avant de faire des projets.

Enregistré dans 1 registre

Une même étude peut être enregistrée dans plusieurs registres. Nous l'affichons une seule fois et renvoyons vers chaque fiche que nous détenons.

Les informations de l'essai sont affichées telles que publiées par le registre, dans leur langue d'origine.

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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.

Résumé

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.

Affections

  • Huntington's Disease

Éligibilité

SexeTous
Âges18 Years – Aucun maximum
Volontaires sainsOui
Méthode d'échantillonnageNON_PROBABILITY_SAMPLE

Population de l'étude

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.

Éligibilité telle qu'elle figure dans le registre

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).

Éligibilité en énoncés simples

Les critères de cette fiche n'ont pas encore été décomposés en énoncés distincts. Le texte du registre ci-dessus est complet et fait foi.

Conception de l'étude

Type d'étudeObservationnelle
PhaseNon précisé dans la fiche du registre
RépartitionNon précisé dans la fiche du registre
Modèle d'interventionNon précisé dans la fiche du registre
Objectif principalNon précisé dans la fiche du registre
InsuNon précisé dans la fiche du registre
Recrutement35 000 participants recherchés

Promoteur et collaborateurs

  • CHDI Foundation, Inc. Promoteur

Critères de jugement

  1. Critère de jugement principal

    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.

    Délai through study completion, an average of 1 year

  2. Critère de jugement principal

    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.

    Délai through study completion, an average of 1 year

  3. Critère de jugement principal

    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.

    Délai through study completion, an average of 1 year

  4. Critère de jugement principal

    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.

    Délai through study completion, an average of 1 year

Dates

Date de début1 juillet 2012 (réelle)
Achèvement principal1 janvier 2062 (estimée)
Achèvement1 janvier 2062 (estimée)
Première publication10 avril 2012 (estimée)
Dernière mise à jour28 février 2024
Résultats publiésNon précisé dans la fiche du registre
Statut vérifié pour la dernière foisfévrier 2024

Réelle signifie que l'événement a eu lieu. Estimée signifie que le promoteur s'y attend. Les deux ont un sens différent.

Lieux

160 sites sont en recrutement

Argentina

ÉtablissementVilleÉtat ou régionStatut
Instituto de Neurociencias Buenos Aires (INEBA)Buenos AiresEn recrutement

Australia

ÉtablissementVilleÉtat ou régionStatut
Monash UniversityMelbourneVictoriaEn recrutement
University of Melbourne, Royal Melbourne HospitalParkvilleEn recrutement
The Neurosciences Unit - North Metropolitan HospitalPerthWestern AustraliaEn recrutement
Westmead HospitalWestmeadNew South WalesEn recrutement

Austria

ÉtablissementVilleÉtat ou régionStatut
Universitatsklinik InnsbruckInnsbruckEn recrutement

Belgium

ÉtablissementVilleÉtat ou régionStatut
Bruxelles - ErasmeBrusselsEn recrutement
Institut de Pathologie et de Génétique (IPG)CharleroiEn recrutement
University Hospitals LeuvenLeuvenVlaams-BrabantEn recrutement
Hôpital du Beau Vallon ASBLSaint-ServaisEn recrutement

Canada

ÉtablissementVilleÉtat ou régionStatut
University of Calgary, Movement Disorders ProgramCalgaryAlbertaEn recrutement
University of Alberta (Glenrose)EdmontonAlbertaArrêté prématurément
University of Alberta Hospital in EdmontonEdmontonAlbertaEn recrutement
Nova Scotia Health AuthorityHalifaxNova ScotiaEn recrutement
Centre for Movement DisordersMarkhamOntarioArrêté prématurément
CHUM Hospital of Notre DameMontrealQuebecEn recrutement
The Ottawa HospitalOttawaOntarioEn recrutement
North York General HospitalTorontoOntarioEn recrutement
University of British ColumbiaVancouverBritish ColumbiaEn recrutement

Chile

ÉtablissementVilleÉtat ou régionStatut
CETRAMSantiagoEn recrutement

Colombia

ÉtablissementVilleÉtat ou régionStatut
Servicios Médicos MS, Clínica de MarlyBogotáCundinamarcaEn recrutement
Universidad de Antioquia, Grupo de NeurocienciasMedellínAntioquiaEn recrutement

Denmark

ÉtablissementVilleÉtat ou régionStatut
University Hospital of AarhusAarhusEn recrutement
University Hospital of CopenhagenCopenhagenEn recrutement
University Hospital of OdenseOdenseEn recrutement

France

ÉtablissementVilleÉtat ou régionStatut
CHU Amiens-PicardieAmiensEn recrutement
CHU Angers, Centre de Référence Maladies NeurogénétiquesAngersEn recrutement
Bordeaux - Huntington Disease competencies centerBordeauxEn recrutement
CHU Gabriel-MontpiedClermont-FerrandEn recrutement
Hôpital Henri Mondor, Centre d'Investigation CliniqueCréteilEn recrutement
Lille-Amiens - Huntington Disease Constituent siteLilleEn recrutement
Hôpital La TimoneMarseilleEn recrutement
CHU MontpellierMontpellierEn recrutement
Institut du Cerveau et de la Moelle EpinièreParisEn recrutement
Hôpitaux Universitaires de Strasbourg Hôpital de Hautepierre-Service de NeurologieStrasbourgAlsace LorraineEn recrutement
Hôpital Purpan, Centre d'Investigation CliniqueToulouseEn recrutement

Germany

ÉtablissementVilleÉtat ou régionStatut
University Hospital AachenAachenEn recrutement
University of BerlinBerlinEn recrutement
St. Josef und St. Elisabeth HospitalBochumEn recrutement
University Hospital Carl Gustav Carus DresdenDresdenEn recrutement
University Hospital ErlangenErlangenEn recrutement
University Medical Center FreiburgFreiburg im BreisgauEn recrutement
Paracelsus Elena Klinik KasselKasselEn recrutement
University Hospital Schleswig-HolsteinLübeckEn recrutement
University Hospital Giessen and MarburgMarburgArrêté prématurément
Technical University of MunichMunichEn recrutement
George-Huntington-Institut GmbHMünsterEn recrutement
kbo-Isar-Amper-Klinikum Taufkirchen (Vils)TaufkirchenEn recrutement
University Hopsital of UlmUlmEn recrutement
University Hospital of WuerzburgWürzburgEn recrutement

133 sites supplémentaires sont indiqués dans la fiche du registre.

Documents de l'étude

Aucun document n'est lié dans cette fiche du registre.

Évolution au fil du temps

Aucun changement n'a été enregistré depuis que nous avons intégré cette fiche pour la première fois.

Un changement est enregistré chaque fois que le promoteur met à jour la fiche du registre. Le statut, les dates, le recrutement et les sites apparaissent ici au fur et à mesure de leur évolution.

Registre: ClinicalTrials.gov · NCT########

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