Extracellular Vesicles for HD

Description

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.

Conditions

Huntington Disease

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Study Overview

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Study Details

Study overview

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.

Extracellular Vesicles As Non-Invasive Biomarkers for Huntington Disease Progression and Huntingtin Lowering Therapy

Extracellular Vesicles for HD

Condition
Huntington Disease
Intervention / Treatment

-

Contacts and Locations

Orlando

University of Central Florida, Orlando, Florida, United States, 32816

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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).
  • 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

Ages Eligible for Study

18 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

Yes

Collaborators and Investigators

University of Central Florida,

Amber Southwell, PhD, PRINCIPAL_INVESTIGATOR, University of Central Florida Burnett School of Biomedical Sciences

Study Record Dates

2031-11