Extracellular Vesicles for HD

NCT ID: NCT06082713

Last Updated: 2024-12-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

100 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-10-25

Study Completion Date

2031-11-30

Brief Summary

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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.

Detailed Description

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Conditions

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Huntington Disease

Keywords

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HD Huntington Disease Healthy

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

RETROSPECTIVE

Study Groups

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Huntington Disease Carriers

No interventions assigned to this group

Non-Huntington Disease Carriers

No interventions assigned to this group

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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University of South Florida

OTHER

Sponsor Role collaborator

University of Central Florida

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Amber Southwell, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Central Florida Burnett School of Biomedical Sciences

Locations

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University of Central Florida

Orlando, Florida, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Amoy Fraser, PhD, CCRP, PMP

Role: CONTACT

Phone: 4072668742

Email: [email protected]

Erica Martin, B.S.

Role: CONTACT

Phone: 4072668742

Email: [email protected]

Facility Contacts

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Amoy Fraser, PhD, CCRP, PMP

Role: primary

Erica Martin, BS

Role: backup

Amber Southwell, PhD

Role: backup

Other Identifiers

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STUDY00004597

Identifier Type: -

Identifier Source: org_study_id