Extracellular Vesicles for HD

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

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06082713

Enrollment

100

Registered

2023-10-13

Start date

2023-10-25

Completion date

2031-11-30

Last updated

2024-12-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Huntington Disease

Keywords

HD, Huntington Disease, Healthy

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.

Interventions

None listed

Study design

Observational model

CASE_CONTROL

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to 75 Years

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

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
To develop more accurate biomarkers of HD progression or conversion	8 years
to develop standard practices for extracellular vesicle biomarker discovery research	8 years

Countries

United States

Contacts

Primary ContactAmoy Fraser, PhD, CCRP, PMP

amoy.fraser@ucf.edu4072668742

Backup ContactErica Martin, B.S.

erica.martin@ucf.edu4072668742

Outcome results