Enroll -HD: A Prospective Registry Study in a Global Huntington's Disease Cohort
ConditionHuntington's Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorCHDI Foundation, Inc.
About this trial
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.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
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.
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.
Disqualifiers
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.).
Trial 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.
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
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 year2
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 year3
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 year4
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 yearSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov