Antibiotic Treatment Trial for the PANDAS/PANS Phenotype
Status:
Completed
Trial end date:
2014-12-01
Target enrollment:
Participant gender:
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.