A Pre-Cellular Therapy Observational Study in Early Huntington's Disease (PRE-CELL)

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2013 by University of California, Davis
Sponsor:
Information provided by (Responsible Party):
University of California, Davis
ClinicalTrials.gov Identifier:
NCT01937923
First received: August 26, 2013
Last updated: September 4, 2013
Last verified: September 2013
  Purpose

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.


Condition
Huntington Disease

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Pre-Cellular Therapy Observational Study in Early Huntington's Disease

Resource links provided by NLM:


Further study details as provided by University of California, Davis:

Primary Outcome Measures:
  • Rate of change from baseline in white matter volume on magnetic resonance imaging (MRI) brain scan. [ Time Frame: Baseline and 12 or 18 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Rate of change from baseline on the UHDRS total motor score [ Time Frame: Baseline and 12 or 18 months ] [ Designated as safety issue: No ]
  • Rate of change from baseline on the Total Functional Capacity score [ Time Frame: Baseline and 12 or 18 months ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples Without DNA

Safety labs: Complete blood count, comprehensive metabolic panel, international normalized ration, partial thromboplastic time, thyroid stimulating hormone, urinalysis, HIV screen.

Biomarkers: Brain-derived neurotrophic factor (BDNF) in cerebrospinal fluid (CSF) and Plasma, BDNF related and Huntington's Disease (HD) specific gene transcription markers in CSF and blood, Small molecule markers of HD in CSF and plasma


Estimated Enrollment: 40
Study Start Date: August 2013
Estimated Study Completion Date: March 2015
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Groups/Cohorts
No treatment

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).

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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.

Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01937923

Contacts
Contact: Teresa Tempkin, RNC MSN ANP 916-734-6278 teresa.tempkin@ucdmc.ucdavis.edu
Contact: Amanda Martin, BA 916-734-3541 amanda.martin@ucdmc.ucdavis.edu

Locations
United States, California
UC Davis Medical Center, Clinical and Translational Science Center Recruiting
Mather, California, United States, 95655
Sponsors and Collaborators
University of California, Davis
Investigators
Principal Investigator: Vicki Wheelock, MD University of California, Davis
  More Information

No publications provided

Responsible Party: University of California, Davis
ClinicalTrials.gov Identifier: NCT01937923     History of Changes
Other Study ID Numbers: 455690, DR2A-05415
Study First Received: August 26, 2013
Last Updated: September 4, 2013
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Delirium, Dementia, Amnestic, Cognitive Disorders
Mental Disorders

ClinicalTrials.gov processed this record on July 29, 2014