A Pre-Cellular Therapy Observational Study in Early Huntington's Disease
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 4/21/2016 |
| Start Date: | August 2013 |
| End Date: | July 2016 |
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.
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).
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).
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
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