Huntington's Disease Clinical Trial
Official title:
Stimulation of Tyrosine Kinase and ERK Signaling Pathways in Huntington's Disease
This study will examine whether lithium carbonate, given alone or with divalproex, increases
the amount of brain-derived neurotrophic factor (BDNF) in the spinal fluid of patients with
Huntington's disease (HD), a hereditary disorder of the central nervous system. Patients
with this fatal degenerative disease have lower amounts of substances in the brain and
spinal fluid called trophic or growth factors. One of these factors is BDNF. A possible
treatment for HD may be to increase the levels of BDNF. Lithium carbonate, a drug used to
treat bipolar disorder, and divalproex, a drug used to treat mood disorders and seizure
disorders, have both been shown to increase the amount of BDNF protein in laboratory
studies.
Patients 18 to 70 years old with a DNA-confirmed diagnosis of Huntington's disease may be
eligible for this study. Candidates are screened with a medical history and physical
examination, neurological evaluation, blood and urine tests, and electrocardiogram (EKG).
Participants take lithium carbonate with and without divalproex. They also receive placebo
(an inactive substance) for portions of the study. On the first day of the study, patients
are given a supply of pills with instructions on how to take them. Blood pressure and pulse
are measured, and blood and urine tests may be done. Patients are evaluated with
standardized tests and scales for assessment of various aspects of HD.
Patients return to the clinic once a week for follow-up evaluations, including blood and
urine tests, physical examinations, disease assessments, and a review of medication side
effects. Each week, they receive a new supply of medications and instructions on how to take
them. At the end of the sixth week, they finish taking the medications.
During the study, patients undergo three lumbar punctures (spinal taps) - at weeks 2, 4, and
6 - to measure BDNF and various other brain chemicals. For this test, a local anesthetic is
given and a needle is inserted in the space between the bones in the lower back where the
CSF circulates below the spinal cord. A small amount of fluid is collected through the
needle. The procedure generally takes from 5 to 20 minutes.
Patients return to the clinic 2 weeks after completing the study medication for a final
evaluation, including a physical examination and blood and urine tests.
Objective: The overall objective of this study is to examine the acute effects of lithium
alone and/ or in combination with divalproex on markers of neuroprotective activity in
patients with Huntington's disease (HD). It is hypothesized that at safe and tolerable doses
these drugs will enhance depleted levels of brain-derived neurotrophic factor (BDNF), a
neurotrophin which is necessary for the survival of striatal neurons.
Study population: Patients suffering from Huntington's disease will be study participants.
Design: The acute effects of lithium and divalproex on surrogate measures of neuroprotective
activity will be evaluated in up to 24 HD patients. The study, lasting approximately 6
weeks, will be conducted on an outpatient basis.
Outcome measures: In this proof-of-principle study, efficacy in restoring BDNF
concentrations will be assessed through cerebrospinal fluid (CSF) measurements. Safety will
be monitored by means of frequent clinical evaluations and laboratory test.
;
Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
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