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Antibiotic Treatment Trial for the PANDAS/PANS Phenotype (AZT)

This study is currently recruiting participants.
Verified March 2013 by University of South Florida
Sponsor:
Collaborator:
Massachusetts General Hospital
Information provided by (Responsible Party):
University of South Florida
ClinicalTrials.gov Identifier:
NCT01617083
First received: May 23, 2012
Last updated: March 29, 2013
Last verified: March 2013
History of Changes
  Purpose

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.


Condition Intervention Phase
Pediatric Acute Onset Neuropsychiatric Syndrome (PANS)
Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections
Obsessive Compulsive Disorder (OCD)
 Drug: Azithromycin
Drug: Placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Antibiotic Treatment Trial for the PANDAS/PANS Phenotype

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by University of South Florida:

Primary Outcome Measures:
  • Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) [ Time Frame: Every 2 weeks for 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Clinical Global Impressions [ Time Frame: Every 2 weeks for 12 weeks ] [ Designated as safety issue: No ]
  • Screen for Child Anxiety Related Disorders [ Time Frame: Every week for 12 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 100
Study Start Date: May 2012
Estimated Study Completion Date: May 2013
Estimated Primary Completion Date: May 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Azithromycin
Antibiotic used to treat infections.
 Drug: Azithromycin
Antibiotic used to treat infections
Other Names:
  • Zithromax
  • Zmax
Placebo Comparator: Sugar Compound
Compound thickening agent with sugar and flavor additives.
 Drug: Placebo
Thickening compound with sugar and flavoring
Other Name: Placebo

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   4 Years to 14 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Ages 4 -14 years.

  • Presence of OCD and at least two of the following:

    1. Anxieties e.g. new onset separation anxiety
    2. Sensory abnormalities (tactile/auditory/visual defensiveness or visual misperceptions)
    3. Behavioral Regression (e.g. new onset impulsivity, hyperactivity, meltdowns)
    4. Deterioration in school performance or in handwriting
    5. Emotional lability and/or depression
    6. Urinary symptoms (frequent urination or enuresis)
    7. 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).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01617083

Contacts
Contact: Tanya Murphy, MD 727-767-8230 Rothmanctr@health.usf.edu
Contact: Laura L Ramirez, BS 727-767-8230 Rothmanctr@health.usf.edu

Locations
United States, Florida
Rothman Center for Neuropsychiatry Recruiting
Saint Petersburg, Florida, United States, 33701
Contact: Tanya Murphy, MD     727-767-8230     Rothmanctr@health.usf.edu    
Contact: Laura Ramirez, BS     727-767-8230     Rothmanctr@health.usf.edu    
Principal Investigator: Tanya Murphy, MD            
Sponsors and Collaborators
University of South Florida
Massachusetts General Hospital
Investigators
Principal Investigator: Tanya Murphy, MD University of South Florida
  More Information

Publications:

Responsible Party: University of South Florida
ClinicalTrials.gov Identifier: NCT01617083     History of Changes
Other Study ID Numbers: 6119-128500, 270332
Study First Received: May 23, 2012
Last Updated: March 29, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by University of South Florida:
PANS
PANDAS
OCD
 Tics
Azithromycin
Antibiotic Treatment

Additional relevant MeSH terms:
Obsessive-Compulsive Disorder
Streptococcal Infections
Autoimmune Diseases
Anxiety Disorders
Mental Disorders
Gram-Positive Bacterial Infections
Bacterial Infections
 Immune System Diseases
Anti-Bacterial Agents
Azithromycin
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 21, 2013