Huntington DiseasePRE-CELLNCT01937923
A Pre-Cellular Therapy Observational Study in Early Huntington's Disease
An observational study of Huntington Disease, run by University of California, Davis at 1 site in United States.
Trial summary
This observational study will establish a clinical baseline and measure changes over time in movement, thinking, behavior, brain imaging, blood and spinal fluid markers in subjects with early stage Huntington's disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Huntington Disease
- How many people
- 29 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2013to Jul 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Subjects will be primarily recruited from the Huntington's disease clinic in the University of California, Davis (UC Davis) department of neurology. We also plan to recruit participants from among northern California regional support groups, from letters to neurologists and psychiatrists who are treating patients with HD, from listing our study on clinical trials.gov, and at regional and national HD meeting presentations, and via print and video recordings on our website.
Who may be able to join
- Men or women age 18 and older, English speaking, able to give informed consent and comply with study procedures.
- HD diagnosis confirmed with genetic testing demonstrating CAG trinucleotide repeat length (CAGn) greater than 37
- Early stage HD with Total Functional Capacity (TFC) score of 9-13
- Demonstrable motor signs with a Unified Huntington's Disease Rating Scale (UHDRS) diagnostic confidence level of 4
- Must have a caregiver or informant able to give feedback about the participant and willing to report observations about subject on standardized forms.
- Subjects of child bearing potential must agree to adequate birth control measures including intrauterine device, hormone therapy, hormone rings or barrier methods including foams/gels AND condoms.
Who cannot join
- Very early disease without demonstrable motor signs (diagnostic confidence level < 4)
- Significant cognitive impairment or dementia as defined by Montreal Cognitive Assessment (MoCA) score < 12.
- Moderate or advanced disease with TFC < 9
- Concurrent active unstable psychiatric disease including history of suicide attempts within the last year, major personality or psychiatric disorders.
- History of concurrent serious medical illness such as HIV or current anti-retroviral treatment, cancer, major cardiac, pulmonary, immunological or other organ disease.
- History of coagulopathy, bleeding disorder, or concurrent use of blood thinners.
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- History of brain tumor, serious traumatic brain injury with coma, or history of brain surgery.
- Any comorbid condition that presents an unacceptable health risk to the patient in the investigator's view
- Clinically significant laboratory test abnormalities, including full blood count, chemistry panel, liver function tests, Prothrombin time/international normalized ratio (PT/INR), lipid panel, electrocardiogram (EKG), or chest x-ray as judged by the investigator.
- Active drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with safety or adherence to study requirements.
- History or documentation of contraindication for MRI brain scan, including the presence of pacemaker, neurostimulator, aneurysm clips, artificial heart valves, cochlear implants, metal fragments in the eyes, orbits or skin or any other known contraindication to MRI.
- Any significant MRI brain scan findings other than those characteristic of HD.
- Any contraindications to surgery or to the use of general anesthesia, including allergy.
- History of use of any investigational agent within 60 days prior to enrollment
- History of current or previous gene therapy or stem cell therapy.
- History of previous or current treatment with cytokines
- History of sensitivity to ganciclovir.
- Pregnant and/or lactating women
Trial locations and contacts
1 site in 1 country.
United States1 site
UC Davis Medical Center, Clinical Research Center
Sacramento, California, 95817
About this study
This observational study will establish a clinical baseline and measure changes over time in movement, thinking, behavior, brain imaging, blood and spinal fluid markers in subjects with early stage Huntington's disease. Participants enrolled in this study may be eligible to participate in a future planned study of stem cell therapy for Huntington's Disease (HD).
In-person study visits occur at screening, baseline, and every 6 months thereafter for a minimum of 12 months, with interim phone call assessments.
Read the detailed description
In PRE-CELL the investigators propose to enroll a cohort of early-stage HD patients in a prospective observational study designed to characterize clinical, neuro-imaging, laboratory and biomarker correlates of disease progression over 12-18 months. Subjects who complete a minimum of 12 months' participation in this trial will be candidates for enrollment in the future planned Phase 1 trial of intrastriatal delivery of mesenchymal stem cell (MSC)/Brain-derived neurotrophic factor (BDNF).
- Conditions
- Huntington Disease
- Medical subject headings
- Huntington Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of change from baseline in white matter volume on magnetic resonance imaging (MRI) brain scan.
Measured: Baseline and 12 or 18 months
Secondary outcomes (2)
Rate of change from baseline on the UHDRS total motor score
Measured: Baseline and 12 or 18 months
Rate of change from baseline on the Total Functional Capacity score
Measured: Baseline and 12 or 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2013 (estimated)
- Study startAug 2013
- Primary completionJul 2016
- Study completionJul 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- More trials run by University of California, DavisThe sponsor's other registered studies.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Davis
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Vicki Wheelock, MD, Principal investigator, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01937923
- Sponsor's protocol ID
- 455690
- Other IDs
- DR2A-05415
- Acronym
- PRE-CELL
- Status last verified
- Oct 2016
- First submitted
- 26 Aug 2013
- Last updated
- 1 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Tempkin T, DeCarli C, Scher L, Farias S, Duffy A., Fink K, Annett G, Brunberg J, Yarborough M, Hersch S, Stout J, Alyward E, Martin A, Kjer L, Swadell D, Nolta J, Wheelock V. PRE-CELL: A Pre-Cellular Observational Study in Early Huntington's Disease. Eight Annual Huntington's disease Clinical Research Symposium, Minneapolis MN, November 2014; Neurotherapeutics 2015;12(1): 263-284
Wheelock V, Tempkin T, Duffy A, Martin A, Mooney L, Scher L, Farias S, Swadell D, DeCarli C, Brunberg J, Li C-S, Yarborough M, Dayananthan A, Stout J, Hersch S, Aylward E, Fink KD, Annett G and Nolta J. "PRE-CELL: Preparing for a future planned Phase 1 trial of genetically-modified stem cells over-expressing BDNF in patients with Huntington's disease." Ninth Annual Huntington's Disease Clinical Research Symposium (October 2015), Tampa FL
Moscovitch-Lopatin M, DiFiglia M, Kegel-Gleason K, Ritch JJ, Rosenthal SJ, Sapp E, Wheelock V, Duffy A, Chopra V, Rosas HD, Hersch SM. "A Novel Translational Bioassay for Conformers of Mutant Huntingtin." Ninth Annual Huntington's Disease Clinical Research Symposium (October 2015), Tampa FL
Study at a glance
From the registry record, last updated by the sponsor on 1 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pre-Cellular Therapy Observational Study in Early Huntington's Disease
- ClinicalTrials.gov ID
- NCT01937923
- Acronym / sponsor ID
- PRE-CELL / 455690
- Conditions
- Huntington Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- University of California, DavisUniversity, hospital or other
- Study start
- Aug 2013
- Primary completion
- Jul 2016 (actual)
- Study completion
- Jul 2016 (actual)
- First posted
- 10 Sep 2013 (estimated)
- 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.
This observational study will establish a clinical baseline and measure changes over time in movement, thinking, behavior, brain imaging, blood and spinal fluid markers in subjects with early stage Huntington's disease. Participants enrolled in this study may be eligible to participate in a future planned study of stem cell therapy for Huntington's Disease (HD).
In-person study visits occur at screening, baseline, and every 6 months thereafter for a minimum of 12 months, with interim phone call assessments.
Detailed description
In PRE-CELL the investigators propose to enroll a cohort of early-stage HD patients in a prospective observational study designed to characterize clinical, neuro-imaging, laboratory and biomarker correlates of disease progression over 12-18 months. Subjects who complete a minimum of 12 months' participation in this trial will be candidates for enrollment in the future planned Phase 1 trial of intrastriatal delivery of mesenchymal stem cell (MSC)/Brain-derived neurotrophic factor (BDNF).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Subjects will be primarily recruited from the Huntington's disease clinic in the University of California, Davis (UC Davis) department of neurology. We also plan to recruit participants from among northern California regional support groups, from letters to neurologists and psychiatrists who are treating patients with HD, from listing our study on clinical trials.gov, and at regional and national HD meeting presentations, and via print and video recordings on our website.
Key inclusion criteria (6 listed)
- Men or women age 18 and older, English speaking, able to give informed consent and comply with study procedures.
- HD diagnosis confirmed with genetic testing demonstrating CAG trinucleotide repeat length (CAGn) greater than 37
- Early stage HD with Total Functional Capacity (TFC) score of 9-13
- Demonstrable motor signs with a Unified Huntington's Disease Rating Scale (UHDRS) diagnostic confidence level of 4
- Must have a caregiver or informant able to give feedback about the participant and willing to report observations about subject on standardized forms.
Key exclusion criteria (18 listed)
- Very early disease without demonstrable motor signs (diagnostic confidence level < 4)
- Significant cognitive impairment or dementia as defined by Montreal Cognitive Assessment (MoCA) score < 12.
- Moderate or advanced disease with TFC < 9
- Concurrent active unstable psychiatric disease including history of suicide attempts within the last year, major personality or psychiatric disorders.
- History of concurrent serious medical illness such as HIV or current anti-retroviral treatment, cancer, major cardiac, pulmonary, immunological or other organ disease.
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
Rate of change from baseline in white matter volume on magnetic resonance imaging (MRI) brain scan.
Time frame: Baseline and 12 or 18 months
2 secondary outcomes
Rate of change from baseline on the UHDRS total motor score
Time frame: Baseline and 12 or 18 months
Rate of change from baseline on the Total Functional Capacity score
Time frame: Baseline and 12 or 18 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2016.
- BackgroundTempkin T, DeCarli C, Scher L, Farias S, Duffy A., Fink K, Annett G, Brunberg J, Yarborough M, Hersch S, Stout J, Alyward E, Martin A, Kjer L, Swadell D, Nolta J, Wheelock V. PRE-CELL: A Pre-Cellular Observational Study in Early Huntington's Disease. Eight Annual Huntington's disease Clinical Research Symposium, Minneapolis MN, November 2014; Neurotherapeutics 2015;12(1): 263-284
- BackgroundWheelock V, Tempkin T, Duffy A, Martin A, Mooney L, Scher L, Farias S, Swadell D, DeCarli C, Brunberg J, Li C-S, Yarborough M, Dayananthan A, Stout J, Hersch S, Aylward E, Fink KD, Annett G and Nolta J. "PRE-CELL: Preparing for a future planned Phase 1 trial of genetically-modified stem cells over-expressing BDNF in patients with Huntington's disease." Ninth Annual Huntington's Disease Clinical Research Symposium (October 2015), Tampa FL
- BackgroundMoscovitch-Lopatin M, DiFiglia M, Kegel-Gleason K, Ritch JJ, Rosenthal SJ, Sapp E, Wheelock V, Duffy A, Chopra V, Rosas HD, Hersch SM. "A Novel Translational Bioassay for Conformers of Mutant Huntingtin." Ninth Annual Huntington's Disease Clinical Research Symposium (October 2015), Tampa FL
- 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. This study is not recruiting at the moment.
Vicki Wheelock, MD principal investigator
University of California, Davis
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 California, Davis 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 6 inclusion and 18 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: finished, as of the sponsor's last update on 1 Nov 2016. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of change from baseline in white matter volume on magnetic resonance imaging (MRI) brain scan. (time frame: Baseline and 12 or 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01937923, which also lists 3 linked publications.
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 1 Nov 2016.