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NCT01574053ClinicalTrials.gov

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

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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…

Beobachtend35.000 Teilnehmende gesucht183 Studienzentren20 Länder

Kategorien

Neurologie45%Seltene und genetische Erkrankungen45%Psychiatrie und psychische Gesundheit10%

Dieser Eintrag wurde kürzlich nicht bestätigt

Der Sponsor hat den Status dieses Eintrags zuletzt in Februar 2024 bestätigt. Er könnte nicht mehr zutreffen.

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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.

Zusammenfassung

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.

Erkrankungen

  • Huntington's Disease

Eignung

GeschlechtAlle
Alter18 Years – Kein Höchstalter
Gesunde FreiwilligeJa
StichprobenverfahrenNON_PROBABILITY_SAMPLE

Studienpopulation

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.

Eignung im Wortlaut des Registers

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).

Eignung in einfachen Aussagen

Die Kriterien dieses Eintrags wurden noch nicht in einzelne Aussagen aufgeschlüsselt. Der Registertext oben ist vollständig und ist die maßgebliche Fassung.

Studiendesign

StudientypBeobachtend
PhaseIm Registereintrag nicht angegeben
ZuteilungIm Registereintrag nicht angegeben
InterventionsmodellIm Registereintrag nicht angegeben
Primäres ZielIm Registereintrag nicht angegeben
VerblindungIm Registereintrag nicht angegeben
Teilnahme35.000 Teilnehmende gesucht

Sponsor und Mitwirkende

  • CHDI Foundation, Inc. Sponsor

Endpunkte

  1. Primärer Endpunkt

    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.

    Zeitrahmen through study completion, an average of 1 year

  2. Primärer Endpunkt

    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.

    Zeitrahmen through study completion, an average of 1 year

  3. Primärer Endpunkt

    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.

    Zeitrahmen through study completion, an average of 1 year

  4. Primärer Endpunkt

    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.

    Zeitrahmen through study completion, an average of 1 year

Daten

Startdatum1. Juli 2012 (tatsächlich)
Primärer Abschluss1. Januar 2062 (geschätzt)
Abschluss1. Januar 2062 (geschätzt)
Erstveröffentlichung10. April 2012 (geschätzt)
Zuletzt aktualisiert28. Februar 2024
Ergebnisse veröffentlichtIm Registereintrag nicht angegeben
Status zuletzt bestätigtFebruar 2024

Tatsächlich bedeutet, dass das Ereignis eingetreten ist. Geschätzt bedeutet, dass der Sponsor es erwartet. Die beiden bedeuten Unterschiedliches.

Standorte

160 Studienzentren rekrutieren

Argentina

EinrichtungStadtBundesland oder RegionStatus
Instituto de Neurociencias Buenos Aires (INEBA)Buenos AiresRekrutiert

Australia

EinrichtungStadtBundesland oder RegionStatus
Monash UniversityMelbourneVictoriaRekrutiert
University of Melbourne, Royal Melbourne HospitalParkvilleRekrutiert
The Neurosciences Unit - North Metropolitan HospitalPerthWestern AustraliaRekrutiert
Westmead HospitalWestmeadNew South WalesRekrutiert

Austria

EinrichtungStadtBundesland oder RegionStatus
Universitatsklinik InnsbruckInnsbruckRekrutiert

Belgium

EinrichtungStadtBundesland oder RegionStatus
Bruxelles - ErasmeBrusselsRekrutiert
Institut de Pathologie et de Génétique (IPG)CharleroiRekrutiert
University Hospitals LeuvenLeuvenVlaams-BrabantRekrutiert
Hôpital du Beau Vallon ASBLSaint-ServaisRekrutiert

Canada

EinrichtungStadtBundesland oder RegionStatus
University of Calgary, Movement Disorders ProgramCalgaryAlbertaRekrutiert
University of Alberta (Glenrose)EdmontonAlbertaVorzeitig beendet
University of Alberta Hospital in EdmontonEdmontonAlbertaRekrutiert
Nova Scotia Health AuthorityHalifaxNova ScotiaRekrutiert
Centre for Movement DisordersMarkhamOntarioVorzeitig beendet
CHUM Hospital of Notre DameMontrealQuebecRekrutiert
The Ottawa HospitalOttawaOntarioRekrutiert
North York General HospitalTorontoOntarioRekrutiert
University of British ColumbiaVancouverBritish ColumbiaRekrutiert

Chile

EinrichtungStadtBundesland oder RegionStatus
CETRAMSantiagoRekrutiert

Colombia

EinrichtungStadtBundesland oder RegionStatus
Servicios Médicos MS, Clínica de MarlyBogotáCundinamarcaRekrutiert
Universidad de Antioquia, Grupo de NeurocienciasMedellínAntioquiaRekrutiert

Denmark

EinrichtungStadtBundesland oder RegionStatus
University Hospital of AarhusAarhusRekrutiert
University Hospital of CopenhagenCopenhagenRekrutiert
University Hospital of OdenseOdenseRekrutiert

France

EinrichtungStadtBundesland oder RegionStatus
CHU Amiens-PicardieAmiensRekrutiert
CHU Angers, Centre de Référence Maladies NeurogénétiquesAngersRekrutiert
Bordeaux - Huntington Disease competencies centerBordeauxRekrutiert
CHU Gabriel-MontpiedClermont-FerrandRekrutiert
Hôpital Henri Mondor, Centre d'Investigation CliniqueCréteilRekrutiert
Lille-Amiens - Huntington Disease Constituent siteLilleRekrutiert
Hôpital La TimoneMarseilleRekrutiert
CHU MontpellierMontpellierRekrutiert
Institut du Cerveau et de la Moelle EpinièreParisRekrutiert
Hôpitaux Universitaires de Strasbourg Hôpital de Hautepierre-Service de NeurologieStrasbourgAlsace LorraineRekrutiert
Hôpital Purpan, Centre d'Investigation CliniqueToulouseRekrutiert

Germany

EinrichtungStadtBundesland oder RegionStatus
University Hospital AachenAachenRekrutiert
University of BerlinBerlinRekrutiert
St. Josef und St. Elisabeth HospitalBochumRekrutiert
University Hospital Carl Gustav Carus DresdenDresdenRekrutiert
University Hospital ErlangenErlangenRekrutiert
University Medical Center FreiburgFreiburg im BreisgauRekrutiert
Paracelsus Elena Klinik KasselKasselRekrutiert
University Hospital Schleswig-HolsteinLübeckRekrutiert
University Hospital Giessen and MarburgMarburgVorzeitig beendet
Technical University of MunichMunichRekrutiert
George-Huntington-Institut GmbHMünsterRekrutiert
kbo-Isar-Amper-Klinikum Taufkirchen (Vils)TaufkirchenRekrutiert
University Hopsital of UlmUlmRekrutiert
University Hospital of WuerzburgWürzburgRekrutiert

133 weitere Studienzentren sind im Registereintrag aufgeführt.

Studiendokumente

In diesem Registereintrag sind keine Dokumente verlinkt.

Änderungen im Zeitverlauf

Seit wir diesen Eintrag erstmals eingelesen haben, wurden keine Änderungen erfasst.

Eine Änderung wird jedes Mal erfasst, wenn der Sponsor den Registereintrag aktualisiert. Status, Daten, Teilnahme und Studienzentren erscheinen hier, sobald sie sich ändern.

Register: ClinicalTrials.gov · NCT########

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