- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01937923
A Pre-Cellular Therapy Observational Study in Early Huntington's Disease (PRE-CELL)
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.
Study Overview
Status
Conditions
Detailed Description
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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California
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Sacramento, California, United States, 95817
- UC Davis Medical Center, Clinical Research Center
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
- 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
Study Plan
How is the study designed?
Design Details
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Rate of change from baseline in white matter volume on magnetic resonance imaging (MRI) brain scan.
Time Frame: Baseline and 12 or 18 months
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Baseline and 12 or 18 months
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Rate of change from baseline on the UHDRS total motor score
Time Frame: Baseline and 12 or 18 months
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Baseline and 12 or 18 months
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Rate of change from baseline on the Total Functional Capacity score
Time Frame: Baseline and 12 or 18 months
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Baseline and 12 or 18 months
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Vicki Wheelock, MD, University of California, Davis
Publications and helpful links
General 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 record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Mental Disorders
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neurocognitive Disorders
- Genetic Diseases, Inborn
- Basal Ganglia Diseases
- Movement Disorders
- Neurodegenerative Diseases
- Dyskinesias
- Heredodegenerative Disorders, Nervous System
- Dementia
- Cognition Disorders
- Chorea
- Huntington Disease
Other Study ID Numbers
- 455690
- DR2A-05415 (Other Grant/Funding Number: CIRM)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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