Clinical Trial: Antibiotic Treatment Trial for the PANDAS/PANS Phenotype
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Antibiotic Treatment Trial for the PANDAS/PANS Phenotype
Brief Summary:
The purpose of this research study is to know if the antibiotic azithromycin, an antibiotic approved by the U.S. Food and Drug Administration (FDA) for treating infections, improves symptom severity in children with sudden and severe onset obsessive compulsive symptoms known as PANS, Pediatric Acute Onset Neuropsychiatric Syndrome, and PANDAS, Pediatric Autoimmune Neuropsychiatric Disorder Associated with Streptococcus. This study seeks to compare the effects of placebo vs. azithromycin on Obsessive Compulsive Disorder (OCD) symptom severity as well as to assess immune risk factors in children with PANDAS/PANS. Obsessions are repetitive, unwanted thoughts or worries that may be unpleasant, silly, or embarrassing. Compulsions are repetitive or ritualistic actions that are performed to ease anxiety or worries. Doctors think these symptoms may be caused or exacerbated by certain infections such as Streptococcus pyogenes, Mycoplasma pneumonia, Borrelia burgordfi, etc. These infections commonly cause strep throat, walking pneumonia, and Lyme Disease, among others.
This study will involve a 4 week double-blind, placebo-controlled randomized trial of azithromycin (Double Blind Phase). At the end of this 4 week trial, the child will be assigned to azithromycin for 8 weeks (Open Label Phase). At the end of these 12 weeks, a Naturalistic Observation phase will assess the child's symptom characteristics for up to 40 weeks.
The study hypothesizes that children receiving antibiotic will show significantly greater overall improvement in severity compared with placebo, and that children with sudden onset of OCD and whose subsequent course shows dramatic fluctuations will have evidence of immune risk factors that predisposes to this presentation.
Detailed Summary:
Accumulating evidence suggests that a subset of children with obsessive compulsive disorder (OCD) have a symptom course that is temporally associated with infections (group A Streptococcus, Mycoplasma, Borrelia burgordfi, etc.). The intent of this proposal is to test the hypotheses that: 1) this subset of children with OCD known as Pediatric Acute Onset Neuropsychiatric Syndrome (PANS) and the specific subset associated with Streptococcus, PANDAS (Pediatric Autoimmune Neuropsychiatric Disorder Associated with Streptococcus), experience symptom onset and exacerbations due to autoimmune/inflammatory responses to infectious triggers and, 2) antibiotic treatment will reduce symptom severity. Investigators have reported improvement in neuropsychiatric symptoms of patients presenting with PANDAS following antibiotic treatment (Murphy & Pichichero, 2002; Snider, Lougee, Slattery et al., 2005). Difficulties with study design and small sample size of early antibiotic trials limit the influence of their findings (Kurlan & Kaplan, 2004) although anecdotally, many families report often dramatic improvements in OCD symptoms after antibiotic therapy.
The proposed study is a 4 week double-blind placebo-controlled study of the efficacy of azithromycin once daily on OCD symptom severity in children ages 4-14 years presenting with the PANS phenotype. An 8 week open label phase will be offered to all subjects completing the 1-month trial to assess longer term efficacy and tolerability (12 weeks total antibiotics for those on active, 8 weeks for those on placebo). At the end of week 8, the child can transition to best clinical care practice (cognitive behavioral therapy, additional medication trials, etc) if not fully remitted. The rate and timing of any relapse will be assessed but are not the primary outcome variables. Evaluations will consist of: 1) semi-structured psychiatric dia
Sponsor: University of South Florida
Current Primary Outcome: Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) [ Time Frame: Every 2 weeks for 12 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Clinical Global Impressions [ Time Frame: Every 2 weeks for 12 weeks ]
- Screen for Child Anxiety Related Disorders [ Time Frame: Every week for 12 weeks ]
Original Secondary Outcome: Same as current
Information By: University of South Florida
Dates:
Date Received: May 23, 2012
Date Started: May 2012
Date Completion:
Last Updated: February 3, 2015
Last Verified: December 2014
