Buspirone Treatment of Anxiety in Williams Syndrome
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|ClinicalTrials.gov Identifier: NCT04807517|
Recruitment Status : Recruiting
First Posted : March 19, 2021
Last Update Posted : July 28, 2022
|Condition or disease||Intervention/treatment||Phase|
|Williams Syndrome Anxiety||Drug: Buspirone||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Buspirone for the Treatment of Anxiety in Williams Syndrome|
|Actual Study Start Date :||August 1, 2021|
|Estimated Primary Completion Date :||May 2025|
|Estimated Study Completion Date :||May 2025|
Subjects will receive buspirone 2.5 mg each morning at the start of the trial. The dose will be increased by 2.5 mg per week in two divided doses daily depending on effectiveness and tolerability. The optimal dose will be reached by week 12 of treatment. The minimum starting dose will be 2.5 mg and the maximum total daily dose will be 30 mg. Medication will be dosed twice daily due to the short half-life (2-3 hours) of this medication.
All participants in the study will receive open-label treatment with orally administered buspirone for the full duration of the 16-week trial. Buspirone has high affinity for serotonin 5-HT1A and 5-HT2 receptors and moderate affinity for dopamine D2 receptors. It is approved for the management of generalized anxiety disorder in adults.
- Mean 16-Week Change in Pediatric Anxiety Rating Scale 5-Item Total Score [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]The Pediatric Anxiety Rating Scale (PARS) is a clinician-rated instrument that assesses anxiety symptoms that are commonly associated with social anxiety, separation anxiety, and generalized anxiety disorders. Scaled score ranges from 0-25 with higher scores indicating more severe anxiety symptoms.
- Proportion of Participants Who Responded to Treatment at 16 Weeks According to the Improvement Item of the Clinical Global Impression-Scale (Response Defined as CGI-I=1 or CGI-I=2) [ Time Frame: Weeks 4, 8, 12, 16 ]The Clinical Global Impressions Global Improvement (CGI-I) is designed to take into account all factors to arrive at an assessment of response to treatment. The CGI-I scale ranges from 1 to 7 (1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse), with lower scales indicating improvement (1=very much improved; 2=much improved). In this study, the CGI-I will be focused on the target symptom of anxiety. Participants with a CGI-I score of 1 or 2 will be classified as responders. The CGI-I will be administered at weeks 4, 8, 12, and 16.
- Mean 16-Week Change in Child and Adolescent Symptom Inventory Anxiety-Modified Score [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]The Child and Adolescent Symptom Inventory (CASI) is a caregiver completed questionnaire with items that map directly onto Diagnostic and Statistical Manual of Mental Disorders diagnostic criteria for anxiety disorders in children and adolescents. The CASI-Modified which includes 20 specific items that have been used to assess anxiety in subjects with developmental disabilities will be administered. Total score ranges from 0-20 with higher scores indicating more severe anxiety symptoms.
- Mean 16-Week Change in Screen for Childhood Anxiety Related Emotional Disorders Total Score [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]The Screen for Childhood Anxiety Related Emotional Disorders (SCARED) includes both a child/self-report and parent-report form, each containing 41-items. It is used to screen for symptoms of panic disorder, separation anxiety disorder, social phobia, generalized anxiety disorder, and school phobia. Total score ranges from 0-82 and a total score of 25 or greater may indicate the presence of an anxiety disorder.
- Mean 16-Week Change in Each Subscale of the Aberrant Behavior Checklist [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]The Aberrant Behavior Checklist (ABC-2) is a caregiver rated instrument that measures psychiatric symptoms and behavioral disturbance in subjects with developmental disability with 5 subscales: Irritability, Social Withdrawal/Lethargy, Stereotypy, Hyperactivity, and Inappropriate Speech. Each item of the 58-item scale is scored on a 4-point scale (0=never a problem to 3=severe problem). The interpretation of the tool and its sub-scales is that a greater number of items indicates greater severity. The range of scores per subscale are: Irritability 0-45; Social Withdrawal/Lethargy 0-48; Stereotypy 0-21; Hyperactivity 0-48; Inappropriate Speech 0-12.
- Mean 16-Week Change in Pittsburgh Sleep Quality Index Global Score [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]The Pittsburgh Sleep Quality Index (PSQI) questionnaire that will be completed by the subject's caregiver to assess sleep quality. The global score ranges from 0-21, where a higher score indicates greater sleep difficulty.
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): NCT04807517
|Contact: Jennifer Mullett||781-860-1711||LurieCenterResearch@partners.org|
|United States, Massachusetts|
|Lurie Center for Autism||Recruiting|
|Lexington, Massachusetts, United States, 02421|
|Principal Investigator:||Robyn P Thom, MD||Massachusetts General Hospital|