N-acetylcysteine (NAC) for Children With Tourette Syndrome
Information source: Yale University
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Tourettes Syndrome; Tic
Intervention: N-Acetylcysteine (NAC) (Drug); Placebo (Drug)
Phase: Phase 2
Sponsored by: Yale University
Official(s) and/or principal investigator(s):
Michael H. Bloch, MD, MS, Principal Investigator, Affiliation: Yale University
Tourette syndrome is a childhood-onset neuropsychiatric disorder characterized by multiple
motor and vocal tics that last for at least a year in duration. Currently, there exist
several effective pharmacological treatments for childhood tics including alpha-2 agonist
medications (guanfacine and clonidine) and neuroleptics (antipsychotic) medications. These
medications, however, have significant side-effects and are only partially efficacy in
N-acetylcysteine (NAC) is a natural supplement that acts as an antioxidant and glutamate
modulating agent. NAC has been used safely for decades in doses 20-40 times higher than in
this trial as an antidote for acetaminophen overdose. The only side-effect commonly seen
with NAC is nausea and this side-effect is seldom seen in the doses used in this trial.
NAC has recently been demonstrated to be effective in a double-blind, placebo-controlled
trial in adults with trichotillomania (chronic hair pulling). Hairpulling is hypothesized
to be closely related to tics because these conditions (1) have similar clinical
characteristics - - both groups typically experience urges before engaging in pulling or
tics, (2) neuroimaging studies suggest they involve similar brain circuits - - the basal
ganglia, (3) the same pharmacological treatments (neuroleptics) may be effective for both
conditions and (4) they tend to be inherited together in families. In other trials NAC has
evidence of some efficacy in treating diverse psychiatric conditions such as bipolar
depression, schizophrenia and cocaine dependence.
The investigators are conducting this trial to determine if NAC is an effective treatment
Official title: Double-Blind, Placebo-Controlled Trial of N-acetylcysteine (NAC) for the Treatment of Children With Tourette Syndrome
Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: Improvement in Tic Severity
Improvement of Premonitory Urges
Improvement in OCD Severity
Minimum age: 8 Years.
Maximum age: 17 Years.
- Children aged 8-17 years.
- Primary diagnosis of Tourette syndrome or chronic tic disorder.
- Duration of tics greater than 1 year.
- Significant Current tic symptoms: Current YGTSS score greater than or equal to 22
with a TS diagnosis or greater than or equal to 14 with a chronic tic disorder.
- Comorbid bipolar disorder, psychotic disorder, substance use disorder, developmental
disorder or mental retardation (IQ<70).
- Recent change (less than 4 weeks) in medications that have potential effects on tic
severity (such as neuroleptic medications, dopamine agonists, alpha-2 agonists
(guanfacine, clonidine or prazosin), SSRIs, clomipramine, naltrexone, lithium,
psychostimulants, or anxiolytics). Medication change is defined to include dose
changes or medication discontinuation.
- Recent change in behavioral treatment for Tourette syndrome or comorbid conditions
(i. e. OCD) within the last 4 weeks or initiation of behavioral therapy for tics
within the last 12 weeks.
- Asthma requiring medication use within the last 3 months
- Known hypersensitivity or previous anaphylactoid reaction to acetylcysteine or any
components in its preparation
- Positive pregnancy test or drug screening test.
- Previous use of N-acetylcysteine (dose greater than 600mg for more than 2 weeks)
Locations and Contacts
Yale Child Study Center, New Haven, Connecticut 06520, United States
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Starting date: July 2010
Last updated: July 21, 2014