Phase 2
N=47
Antibiotic Treatment Trial for the PANDAS/PANS Phenotype
PANS · PANDAS · Obsessive Compulsive Disorder (OCD)
Bottom Line
View on ClinicalTrials.gov: NCT01617083 ↗Enrolled (actual)
47
Serious AEs
0.0%
Results posted
Apr 2018
Primary outcome: Primary: Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) — 29.49; 28.43; 20.53; 23.45 units on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Azithromycin (Drug); Placebo (Drug)
- Age
- Pediatric · 4+ yrs
- Sex
- All
- Sponsor
- University of South Florida
- Primary completion
- Dec 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) |
29.49; 28.43; 20.53; 23.45 | — |
| SECONDARY Clinical Global Impressions-Severity OCD |
5.24; 5.00; 4.06; 4.93 | — |
| SECONDARY Screen for Childhood Anxiety-Related Emotional Disorders (SCARED) |
31.82; 20.43; 30.88; 21.97 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Ages 4 -14 years.
- Presence of OCD and at least two of the following:
- Anxieties e.g. new onset separation anxiety
- Sensory abnormalities (tactile/auditory/visual defensiveness or visual misperceptions)
- Behavioral Regression (e.g. new onset impulsivity, hyperactivity, meltdowns)
- Deterioration in school performance or in handwriting
- Emotional lability and/or depression
- Urinary symptoms (frequent urination or enuresis)
- Sleep disturbances Anorexia
- Current episode OCD of recent onset (less or equal to 6 months) associated with infection
- Symptom onset appears temporally related to infection or exposure
- Symptoms are of moderate severity with significant impairment (CGI of moderate or worse) and CY-BOCS of more or equal to 16.
- Parental willingness to accompany their child for multiple study visits and be responsible for medication compliance.
Exclusion Criteria
- History of Rheumatic Fever including Sydenham's Chorea (heart murmur, frank chorea, EKG PR or QTc prolongation, abnormal reflexes (Gordon-Hey reflex)).
- Diagnosis of autism (moderate - severe), schizophrenia, mental retardation or chronic degenerative neurological disease.
- Any illness for which antibiotic treatment may be contraindicated (e.g. Liver disease).
- Personal history of adverse reaction or allergy to azithromycin.
- Recent or planned psychopharmacologic (4 weeks for most medications or 8 weeks for SSRIs) treatment changes.
- Antibiotic prophylaxis therapy or history of neuropsychiatric non-response to prior antibiotic trial.
- Currently participating in cognitive behavioral therapy or habit reversal therapy for OCD and/or tics.
- Weight less than 15 kilograms
- Concurrent therapy with medications that may increase adverse effects (eg. pimozide, citalopram, tricyclic antidepressants, etc).
Data sourced from ClinicalTrials.gov (NCT01617083). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.