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

August 26, 2013
September 4, 2013
August 2013
March 2015   (final data collection date for 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 ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT01937923 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
A Pre-Cellular Therapy Observational Study in Early Huntington's Disease
A Pre-Cellular Therapy Observational Study in Early Huntington's Disease

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.

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).

Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Retention:   Samples Without DNA
Description:

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

Non-Probability Sample

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.

Huntington Disease
Not Provided
No treatment
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
40
March 2015
March 2015   (final data collection date for primary outcome measure)

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
Both
18 Years and older
No
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
United States
 
NCT01937923
455690, DR2A-05415
No
University of California, Davis
University of California, Davis
Not Provided
Principal Investigator: Vicki Wheelock, MD University of California, Davis
University of California, Davis
September 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP