NCT01574053ClinicalTrials.gov

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

RecruitingTaking participants now, according to the registry record.

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In brief

Observational35,000 participants sought183 sites20 countries

This record has not been confirmed recently

The sponsor last confirmed this record's status in February 2024. It may no longer be accurate.

The record still reads Recruiting, but nobody has confirmed that in 2 years. Contact the study site before making plans.

Registered in 1 registry

ClinicalTrials.govNCT01574053Open this record at ClinicalTrials.govSynced 2 months ago

One study can be registered in several registries. We show it once and link every record we hold.

Trial information is shown as published by the registry, in its original language.

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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.

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

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.

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.

Conditions

Huntington's Disease

Eligibility

Eligibility
Sex	All
Ages	18 Years – No maximum
Healthy volunteers	Yes
Sampling method	NON_PROBABILITY_SAMPLE

Study population

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.

Eligibility as written in the registry

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).

Eligibility in plain statements

This record's criteria have not been broken into separate statements yet. The registry text above is complete and is the authoritative version.

Study design

Study design
Study type	Observational
Phase	Not stated in the registry record
Allocation	Not stated in the registry record
Intervention model	Not stated in the registry record
Primary purpose	Not stated in the registry record
Masking	Not stated in the registry record
Enrolment	35,000 participants sought

Sponsor and collaborators

CHDI Foundation, Inc. Sponsor

Outcome measures

Primary outcome
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.
Time frame through study completion, an average of 1 year
Primary outcome
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.
Time frame through study completion, an average of 1 year
Primary outcome
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.
Time frame through study completion, an average of 1 year
Primary outcome
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.
Time frame through study completion, an average of 1 year

Dates

Dates
Start date	July 1, 2012 (actual)
Primary completion	January 1, 2062 (estimated)
Completion	January 1, 2062 (estimated)
First posted	April 10, 2012 (estimated)
Last updated	February 28, 2024
Results posted	Not stated in the registry record
Status last verified	February 2024

Actual means the event happened. Estimated means the sponsor expects it. The two mean different things.

Locations

160 sites are recruiting

Argentina

Argentina
Facility	City	State or region	Status
Instituto de Neurociencias Buenos Aires (INEBA)	Buenos Aires		Recruiting

Australia

Australia
Facility	City	State or region	Status
Monash University	Melbourne	Victoria	Recruiting
University of Melbourne, Royal Melbourne Hospital	Parkville		Recruiting
The Neurosciences Unit - North Metropolitan Hospital	Perth	Western Australia	Recruiting
Westmead Hospital	Westmead	New South Wales	Recruiting

Austria

Austria
Facility	City	State or region	Status
Universitatsklinik Innsbruck	Innsbruck		Recruiting

Belgium

Belgium
Facility	City	State or region	Status
Bruxelles - Erasme	Brussels		Recruiting
Institut de Pathologie et de Génétique (IPG)	Charleroi		Recruiting
University Hospitals Leuven	Leuven	Vlaams-Brabant	Recruiting
Hôpital du Beau Vallon ASBL	Saint-Servais		Recruiting

Canada

Canada
Facility	City	State or region	Status
University of Calgary, Movement Disorders Program	Calgary	Alberta	Recruiting
University of Alberta (Glenrose)	Edmonton	Alberta	Terminated
University of Alberta Hospital in Edmonton	Edmonton	Alberta	Recruiting
Nova Scotia Health Authority	Halifax	Nova Scotia	Recruiting
Centre for Movement Disorders	Markham	Ontario	Terminated
CHUM Hospital of Notre Dame	Montreal	Quebec	Recruiting
The Ottawa Hospital	Ottawa	Ontario	Recruiting
North York General Hospital	Toronto	Ontario	Recruiting
University of British Columbia	Vancouver	British Columbia	Recruiting

Chile

Chile
Facility	City	State or region	Status
CETRAM	Santiago		Recruiting

Colombia

Colombia
Facility	City	State or region	Status
Servicios Médicos MS, Clínica de Marly	Bogotá	Cundinamarca	Recruiting
Universidad de Antioquia, Grupo de Neurociencias	Medellín	Antioquia	Recruiting

Denmark

Denmark
Facility	City	Status
University Hospital of Aarhus	Aarhus	Recruiting
University Hospital of Copenhagen	Copenhagen	Recruiting
University Hospital of Odense	Odense	Recruiting

France

France
Facility	City	State or region	Status
CHU Amiens-Picardie	Amiens		Recruiting
CHU Angers, Centre de Référence Maladies Neurogénétiques	Angers		Recruiting
Bordeaux - Huntington Disease competencies center	Bordeaux		Recruiting
CHU Gabriel-Montpied	Clermont-Ferrand		Recruiting
Hôpital Henri Mondor, Centre d'Investigation Clinique	Créteil		Recruiting
Lille-Amiens - Huntington Disease Constituent site	Lille		Recruiting
Hôpital La Timone	Marseille		Recruiting
CHU Montpellier	Montpellier		Recruiting
Institut du Cerveau et de la Moelle Epinière	Paris		Recruiting
Hôpitaux Universitaires de Strasbourg Hôpital de Hautepierre-Service de Neurologie	Strasbourg	Alsace Lorraine	Recruiting
Hôpital Purpan, Centre d'Investigation Clinique	Toulouse		Recruiting

Germany

Germany
Facility	City	Status
University Hospital Aachen	Aachen	Recruiting
University of Berlin	Berlin	Recruiting
St. Josef und St. Elisabeth Hospital	Bochum	Recruiting
University Hospital Carl Gustav Carus Dresden	Dresden	Recruiting
University Hospital Erlangen	Erlangen	Recruiting
University Medical Center Freiburg	Freiburg im Breisgau	Recruiting
Paracelsus Elena Klinik Kassel	Kassel	Recruiting
University Hospital Schleswig-Holstein	Lübeck	Recruiting
University Hospital Giessen and Marburg	Marburg	Terminated
Technical University of Munich	Munich	Recruiting
George-Huntington-Institut GmbH	Münster	Recruiting
kbo-Isar-Amper-Klinikum Taufkirchen (Vils)	Taufkirchen	Recruiting
University Hopsital of Ulm	Ulm	Recruiting
University Hospital of Wuerzburg	Würzburg	Recruiting

133 further sites are listed in the registry record.

Study documents

No documents are linked in this registry record.

Changes over time

No changes have been recorded since we first ingested this record.

A change is recorded each time the sponsor updates the registry record. Status, dates, enrolment and sites appear here as they move.

Registry: ClinicalTrials.gov · NCT########

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

Categories

This record has not been confirmed recently

Registered in 1 registry

Interested in this study?

How this study is set up

Summary

Conditions

Eligibility

Study population

Eligibility as written in the registry

Eligibility in plain statements

Study design

Sponsor and collaborators

Outcome measures

Dates

Locations

Argentina

Australia

Austria

Belgium

Canada

Chile

Colombia

Denmark

France

Germany

Study documents

Changes over time