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Extracellular Vesicles for HD

Sponsor
University of Central Florida (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT06082713
Collaborator
University of South Florida (Other)
100
Enrollment
96
Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

The primary objective of this study is to discover blood-based biomarker of brain Huntingtin (HTT) protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials.

Secondary objectives of this study include developing more accurate biomarkers of Huntington disease (HD) progression or conversion and to develop standard practices for extracellular vesicle biomarker discovery research.

The investigators hypothesize that brain-derived extracellular vesicles (EVs) isolated from human biofluids contain biological cargo specific to their tissue of origin that could allow their use as brain biomarkers for HD. EVs are lipid bilayer-delimited particles that are naturally released from cells in the brain.

The investigators will investigate if EVs contents reflect the pathological alterations occurring with disease progression when compared with EVs isolated from biofluids of healthy non-HD persons.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    100 participants
    Observational Model:
    Case-Control
    Time Perspective:
    Retrospective
    Official Title:
    Extracellular Vesicles as Non-Invasive Biomarkers for Huntington Disease Progression and Huntingtin Lowering Therapy
    Anticipated Study Start Date :
    Nov 1, 2023
    Anticipated Primary Completion Date :
    Nov 1, 2031
    Anticipated Study Completion Date :
    Nov 1, 2031

    Arms and Interventions

    Arm Intervention/Treatment
    Huntington Disease Carriers
    Non-Huntington Disease Carriers

    Outcome Measures

    Primary Outcome Measures

    1. To discover blood-based biomarker of brain HTT protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials. [1 year]

    Secondary Outcome Measures

    1. To develop more accurate biomarkers of HD progression or conversion [8 years]

    2. to develop standard practices for extracellular vesicle biomarker discovery research [8 years]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 75 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    Yes
    Inclusion Criteria:

    1. 18 - 75 years of age

    2. can provide informed consent

    3. able to read and speak English

    4. agree to comply with study procedures (including overnight fasting, blood collection and lumbar puncture); and

    5. has been diagnosed with HD (HD Carriers) or not been diagnosed with HD (Non-HD Carriers).

    Exclusion Criteria:

    1. younger than 18 or older than 75 years old

    2. known to carry an intermediate CAG repeat between 27 and 39 or a larger expansion of 60 or more CAG repeats

    3. receiving nutrition through a tube

    4. pregnant

    5. participated in a clinical drug trial within 30 days

    6. use prescribed or non-prescribed medications that are not compatible with collection of the study samples (those that may cause excessive bleeding or prevent clotting)

    7. positive for HIV, hepatitis B or C

    8. have a confirmed or suspected immunodeficient condition/state

    9. significant medical, psychiatric, or neurological morbidity is observed by the clinic physician on the day of sample collection that might impair completion of the study procedures

    10. have needle phobia, frequent headache, significant lower spinal deformity or major surgery

    11. received antiplatelet or anticoagulant therapy within 14 days prior to sample collection (including but not limited to: aspirin, clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban and apixaban)

    12. have a blood clotting or bruising disorder

    13. do not comply with or are unwilling to undertake any of the study procedures

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • University of Central Florida
    • University of South Florida

    Investigators

    • Principal Investigator: Amber Southwell, PhD, University of Central Florida Burnett School of Biomedical Sciences

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    University of Central Florida
    ClinicalTrials.gov Identifier:
    NCT06082713
    Other Study ID Numbers:
    • STUDY00004597
    First Posted:
    Oct 13, 2023
    Last Update Posted:
    Oct 19, 2023
    Last Verified:
    Oct 1, 2023
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by University of Central Florida
    HD
    Huntington Disease
    Healthy
    Additional relevant MeSH terms:
    Huntington Disease

    Study Results

    No Results Posted as of Oct 19, 2023