Huntington DiseaseNCT06082713
Extracellular Vesicles for HD
An observational study of Huntington Disease, run by University of Central Florida at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Huntington Disease
- How many people
- 100 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Oct 2023to Nov 2031
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: The study will enroll HD carriers (HD Carriers) and non-carriers (Non-HD Carriers). 100 participants will be enrolled. 25 from Central Florida Center for Huntington Disease at the University of Central Florida (Orlando) and 75 from Huntington's Disease Center of Excellence at the University of South Florida (Tampa).
Who may be able to join
- 18 - 75 years of age
- can provide informed consent
- able to read and speak English
- agree to comply with study procedures (including overnight fasting, blood collection and lumbar puncture); and
- has been diagnosed with HD (HD Carriers) or not been diagnosed with HD (Non-HD Carriers).
Who cannot join
- younger than 18 or older than 75 years old
- known to carry an intermediate CAG repeat between 27 and 39 or a larger expansion of 60 or more CAG repeats
- receiving nutrition through a tube
- pregnant
- participated in a clinical drug trial within 30 days
- 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)
Show all 13Show fewer
- positive for HIV, hepatitis B or C
- have a confirmed or suspected immunodeficient condition/state
- 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
- have needle phobia, frequent headache, significant lower spinal deformity or major surgery
- 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)
- have a blood clotting or bruising disorder
- do not comply with or are unwilling to undertake any of the study procedures
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Central Florida
Orlando, Florida, 32816
Amoy Fraser, PhD, CCRP, PMP, 4072668742, amoy.fraser@ucf.edu
Erica Martin, BS, 4072668742, erica.martin@ucf.edu
About this study
Official title: Extracellular Vesicles As Non-Invasive Biomarkers for Huntington Disease Progression and Huntingtin Lowering Therapy
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.
Read more
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
- Keywords
- HD, Huntington Disease, Healthy
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Huntington Disease Carriers | Not described | |
| Non-Huntington Disease Carriers | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
To discover blood-based biomarker of brain HTT protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials.
Measured: 1 year
Secondary outcomes (2)
To develop more accurate biomarkers of HD progression or conversion
Measured: 8 years
to develop standard practices for extracellular vesicle biomarker discovery research
Measured: 8 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2023
- Study start25 Oct 2023
- Primary completionNov 2031 (estimated)
- Study completionNov 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Retrospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Central Florida
- Sponsor type
- University, hospital or other
- Collaborators
- University of South Florida
- Responsible party
- Sponsor
- Investigators
- Amber Southwell, PhD, Principal investigator, University of Central Florida Burnett School of Biomedical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06082713
- Sponsor's protocol ID
- STUDY00004597
- Status last verified
- Dec 2024
- First submitted
- 8 Oct 2023
- Last updated
- 6 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Extracellular Vesicles As Non-Invasive Biomarkers for Huntington Disease Progression and Huntingtin Lowering Therapy
- ClinicalTrials.gov ID
- NCT06082713
- Acronym / sponsor ID
- STUDY00004597
- Conditions
- Huntington Disease
- Keywords
- HD, Huntington Disease, Healthy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- University of Central FloridaUniversity, hospital or other
- Collaborators
- University of South Florida
- Study start
- 25 Oct 2023 (actual)
- Primary completion
- Nov 2031 (estimated)
- Study completion
- Nov 2031 (estimated)
- First posted
- 13 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case-control
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Huntington Disease Carriers | – | – |
| Non-Huntington Disease Carriers | – | – |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- The study will enroll HD carriers (HD Carriers) and non-carriers (Non-HD Carriers). 100 participants will be enrolled. 25 from Central Florida Center for Huntington Disease at the University of Central Florida (Orlando) and 75 from Huntington's Disease Center of Excellence at the University of South Florida (Tampa).
Key inclusion criteria (5 listed)
- 18 - 75 years of age
- can provide informed consent
- able to read and speak English
- agree to comply with study procedures (including overnight fasting, blood collection and lumbar puncture); and
- has been diagnosed with HD (HD Carriers) or not been diagnosed with HD (Non-HD Carriers).
Key exclusion criteria (13 listed)
- younger than 18 or older than 75 years old
- known to carry an intermediate CAG repeat between 27 and 39 or a larger expansion of 60 or more CAG repeats
- receiving nutrition through a tube
- pregnant
- participated in a clinical drug trial within 30 days
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To discover blood-based biomarker of brain HTT protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials.
Time frame: 1 year
2 secondary outcomes
To develop more accurate biomarkers of HD progression or conversion
Time frame: 8 years
to develop standard practices for extracellular vesicle biomarker discovery research
Time frame: 8 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Dec 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Amber Southwell, PhD principal investigator
University of Central Florida Burnett School of Biomedical Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Huntington Disease, run by University of Central Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Amoy Fraser, PhD, CCRP, PMP (4072668742, amoy.fraser@ucf.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To discover blood-based biomarker of brain HTT protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials. (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Dec 2024; AzTrials last refreshed its copy on 28 Oct 2026.