Trial Outcomes & Findings for Antibiotic Treatment Trial for the PANDAS/PANS Phenotype (NCT NCT01617083)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

47 participants

Primary outcome timeframe

Before and after 4 week randomization

Results posted on

2018-04-10

Participant Flow

Participant milestones

Participant milestones
Measure	Azithromycin Antibiotic used to treat infections. Azithromycin: Antibiotic used to treat infections	Placebo Compound thickening agent with sugar and flavor additives. Placebo: Thickening compound with sugar and flavoring	Not Randomized Participants consented to participate in the study. They completed baseline assessments but did not randomize to treatment.
Overall Study STARTED	18	14	15
Overall Study COMPLETED	18	14	15
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Antibiotic Treatment Trial for the PANDAS/PANS Phenotype

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Azithromycin n=18 Participants Antibiotic used to treat infections. Azithromycin: Antibiotic used to treat infections	Placebo n=14 Participants Compound thickening agent with sugar and flavor additives. Placebo: Thickening compound with sugar and flavoring	Not Randomized n=15 Participants Participants only completed the baseline assessments but did not receive treatment.	Total n=47 Participants Total of all reporting groups
Age, Categorical <=18 years	18 Participants n=5 Participants	14 Participants n=7 Participants	15 Participants n=5 Participants	47 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	7.78 years STANDARD_DEVIATION 2.39 • n=5 Participants	8.86 years STANDARD_DEVIATION 3.11 • n=7 Participants	7.21 years STANDARD_DEVIATION 2.58 • n=5 Participants	8.25 years STANDARD_DEVIATION 2.74 • n=4 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	5 Participants n=7 Participants	9 Participants n=5 Participants	21 Participants n=4 Participants
Sex: Female, Male Male	11 Participants n=5 Participants	9 Participants n=7 Participants	6 Participants n=5 Participants	26 Participants n=4 Participants
Region of Enrollment United States	18 participants n=5 Participants	14 participants n=7 Participants	15 participants n=5 Participants	47 participants n=4 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Azithromycin n=18 Participants Antibiotic used to treat infections. Azithromycin: Antibiotic used to treat infections	Placebo n=14 Participants Compound thickening agent with sugar and flavor additives. Placebo: Thickening compound with sugar and flavoring	Not Randomized Participants consented to participate in the study. They completed baseline assessments but did not randomize to treatment.
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) Baseline	29.49 units on a scale Standard Error 1.85	28.43 units on a scale Standard Error 2.04	—
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) After 4 week randomization	20.53 units on a scale Standard Error 1.85	23.45 units on a scale Standard Error 2.09	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Azithromycin n=18 Participants Antibiotic used to treat infections. Azithromycin: Antibiotic used to treat infections	Placebo n=14 Participants Compound thickening agent with sugar and flavor additives. Placebo: Thickening compound with sugar and flavoring	Not Randomized Participants consented to participate in the study. They completed baseline assessments but did not randomize to treatment.
Clinical Global Impressions-Severity OCD Baseline	5.24 units on a scale Standard Error 0.23	5.00 units on a scale Standard Error 0.25	—
Clinical Global Impressions-Severity OCD After 4 week randomization	4.06 units on a scale Standard Error .23	4.93 units on a scale Standard Error .25	—

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Azithromycin n=18 Participants Antibiotic used to treat infections. Azithromycin: Antibiotic used to treat infections	Placebo n=14 Participants Compound thickening agent with sugar and flavor additives. Placebo: Thickening compound with sugar and flavoring	Not Randomized Participants consented to participate in the study. They completed baseline assessments but did not randomize to treatment.
Screen for Childhood Anxiety-Related Emotional Disorders (SCARED) Baseline	31.82 units on a scale Standard Error 3.75	20.43 units on a scale Standard Error 4.13	—
Screen for Childhood Anxiety-Related Emotional Disorders (SCARED) After 4 week randomization	30.88 units on a scale Standard Error 3.75	21.97 units on a scale Standard Error 4.17	—

Adverse Events

Azithromycin

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Azithromycin n=18 participants at risk This group was assigned to receive azithromycin (antibiotic).	Placebo n=14 participants at risk This group was assigned to receive placebo (compound thickening agent with sugar and flavor additives - not active treatment).
Gastrointestinal disorders Loose stools	50.0% 9/18 • Before and after 4 week randomization	7.1% 1/14 • Before and after 4 week randomization
Gastrointestinal disorders Constipation	0.00% 0/18 • Before and after 4 week randomization	35.7% 5/14 • Before and after 4 week randomization

Additional Information

Study Coordinator

University of South Florida Rothman Center

Phone: 7277678393

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place