Antibiotic Treatment Trial for the PANDAS/PANS Phenotype (AZT)
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|ClinicalTrials.gov Identifier: NCT01617083|
Recruitment Status : Completed
First Posted : June 12, 2012
Results First Posted : April 10, 2018
Last Update Posted : April 10, 2018
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 or disease||Intervention/treatment||Phase|
|PANS PANDAS Obsessive Compulsive Disorder (OCD)||Drug: Azithromycin Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||47 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Antibiotic Treatment Trial for the PANDAS/PANS Phenotype|
|Study Start Date :||May 2012|
|Actual Primary Completion Date :||December 2014|
|Actual Study Completion Date :||December 2014|
Active Comparator: Azithromycin
Antibiotic used to treat infections.
Antibiotic used to treat infections
Placebo Comparator: Placebo
Compound thickening agent with sugar and flavor additives.
Thickening compound with sugar and flavoring
- Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) [ Time Frame: Before and after 4 week randomization ]The CYBOCS is a clinician rated, semi-structured interview for rating the severity of OCD (Scahill, et al., 1997). Total scores range from 0 to 50, with higher scores representing greater severity. The total score is comprised of two subscores, obsessions and compulsions, each with a range of 0-25.
- Clinical Global Impressions-Severity OCD [ Time Frame: Before and after 4 week randomization ]The CGI-S scale is a 7-point clinician rating of severity of psychopathology. Severity ratings range from 1 (no illness) to 7 (extremely severe). This instrument has been successfully used in treatment studies (Cook, et al., 2001; Storch, et al., 2007).
- Screen for Childhood Anxiety-Related Emotional Disorders (SCARED) [ Time Frame: Before and after 4 week randomization ]The Screen for Childhood Anxiety-Related Emotional Disorders (SCARED) is a 41-item parent and participant-completed tool used to measure symptoms of anxiety, including the most common symptoms of panic/somatic, generalized anxiety, separation anxiety, social phobia, and school phobia. Scores range from 0-82, with higher scores indicating more severe symptoms.The child and parent versions of the SCARED have moderate parent-child agreement and good internal consistency, test-retest reliability, and discriminant validity; it is also sensitive to treatment response (Birmaher et al. 1999). The target population for this rating is 8-18 years of age (Birmaher et al. 1997). The parent and child version of the SCARED were administered before and after week 4 randomization. This measure is not validated for use in 4-7 year olds.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01617083
|United States, Florida|
|Rothman Center for Neuropsychiatry|
|Saint Petersburg, Florida, United States, 33701|
|Principal Investigator:||Tanya Murphy, MD||University of South Florida|