Intranasal Vasopressin Treatment in Children With Autism
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 157
- 试验地点
- 2
- 主要终点
- Change From Baseline in Parent Rated Social Responsiveness Scale, Second Edition (SRS-2) Total Scores During Treatment.
研究概览
简要总结
The purpose of this clinical trial is to investigate the effectiveness of vasopressin nasal spray for treating symptoms associated with autism. Vasopressin is a hormone that is produced naturally within the body and has been implicated in regulating social behaviors. It has been proposed that administration of the hormone may also help improve social functioning in individuals with autism.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Medically healthy outpatients between 6 and 17 years of age;
- •Diagnostic and Statistical Manual 5th edition (DSM-5) criteria for Autism Spectrum Disorder (ASD) on the basis of clinical evaluation, confirmed with the Autism Diagnostic Interview Revised (ADI-R) and Autism Diagnostic Observation Schedule, Second Edition (ADOS-2) or Childhood Autism Rating Scale, Second Edition (CARS-2);
- •males and females;
- •intelligence quotient (IQ) of 40 and above;
- •rating of 4 or higher on the Social Communication domain of the Clinical Global Impressions Severity (CGI-S);
- •Social Responsiveness Scale-2 Total Score of 70 and above;
- •care provider who can reliably bring participant to clinic visits, provide trustworthy ratings, and interacts with participant on a regular basis;
- •stable concomitant psychotropic medications or medications potentially affecting vasopressin for at least 4 weeks (with the exception of fluoxetine, 6 weeks);
- •no planned changes in psychosocial and biomedical interventions during the trial;
- •willingness to provide blood samples and ability to participate in key study procedures (i.e., diagnostic assessments and laboratory safety measurements).
排除标准
- •DSM-5 diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder;
- •regular nasal obstruction or nosebleeds;
- •unstable medical conditions such as migraine, asthma attacks, or seizures, and significant physical illness (e.g. serious liver disease, renal dysfunction, or cardiac pathology);
- •clinically significant abnormal electrocardiogram reading;
- •history of hypersensitivity to vasopressin, its analogs, or compounding preservatives (e.g., chlorobutanol);
- •evidence of a genetic mutation known to cause ASD or intellectual disability (e.g., Fragile X Syndrome); or metabolic, or infectious etiology for ASD on the basis of medical history, neurologic history, and available tests for inborn errors of metabolism and chromosomal analysis;
- •significant hearing or vision impairments;
- •habitually drinks large volumes of water;
- •pregnant or sexually active females not using a reliable method of contraception;
- •current use of any medications known to interact with vasopressin including: 1) carbamazepine (i.e., Tegretol); chlorpropamide; clofibrate; urea; fludrocortisone; tricyclic antidepressants (all of which may potentiate the antidiuretic effect of vasopressin when used concurrently); 2) demeclocycline; norepinephrine; lithium; heparin; alcohol (all of which may decrease the antidiuretic effect of vasopressin when used concurrently); 3) ganglionic blocking agents including benzohexonium, chlorisondamine, pentamine (all of which may produce a marked increase in sensitivity to the pressor effects of vasopressin);
- •previous participation in a vasopressin clinical trial or current use of vasopressin;
- •current use of desmopressin (DDAVP) or oxytocin.
研究组 & 干预措施
Placebo-Vasopressin
4 weeks of placebo nasal spray followed by 4 weeks of vasopressin nasal spray (16 international units twice daily)
干预措施: Placebo (Drug)
Placebo-Placebo
8 weeks of placebo nasal spray; followed by a 4 week open-label extension of vasopressin nasal spray (16 international units twice daily)
干预措施: Placebo (Drug)
Placebo-Vasopressin
4 weeks of placebo nasal spray followed by 4 weeks of vasopressin nasal spray (16 international units twice daily)
干预措施: Vasopressin (USP) Injectable Solution [Vasostrict] (Drug)
Placebo-Placebo
8 weeks of placebo nasal spray; followed by a 4 week open-label extension of vasopressin nasal spray (16 international units twice daily)
干预措施: Vasopressin (USP) Injectable Solution [Vasostrict] (Drug)
Vasopressin-Vasopressin
8 weeks of vasopressin nasal spray (16 international units twice daily)
干预措施: Vasopressin (USP) Injectable Solution [Vasostrict] (Drug)
结局指标
主要结局
Change From Baseline in Parent Rated Social Responsiveness Scale, Second Edition (SRS-2) Total Scores During Treatment.
时间窗: baseline, 4-week, 8-week
Social Responsiveness Scale, 2nd Edition (SRS) scores measure social abilities with lower scores meaning better social abilities. The SRS-2 is reported as a total score (T-Score Range: 37 to above 90), and the Diagnostic and Statistical Manual of Mental Disorders (DSM)-5-compatible Social Communication and Interaction (SCI) score (T-Score Range: 36 to above 90) and Restricted Interests and Repetitive Behavior (RRB) score (T-Score range: 41 to above 90). A T-score of 50 indicates the population mean with a standard deviation of 10. Higher scores correspond to greater symptom levels (≤ 59: within normal limits; 60-65: mild range; 66-75: moderate range; ≥ 76: severe range). Change is reported as 4-week minus the baseline score, and 8-week minus the 4-week score. For this outcome, the baseline score is the average of the screening visit and baseline visit (average approximately 2 to 3 weeks after the screening visit).
次要结局
- Change From Baseline on Weight During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Baseline Vasopressin Concentration Predicting Primary and Secondary Behavioral Outcome Measures.(4-week; 8-week)
- Change From Baseline on Height During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline in Clinical Global Impression (CGI) Scores During Treatment.(baseline; 4-week; 8-week)
- Change From Baseline on Reading the Mind in the Eyes Test (RMET) During Treatment.(baseline; 4-week; 8-week)
- Change From Baseline on the Facial Emotion Recognition Test During Treatment.(baseline; 4-week; 8-week)
- Change From Baseline in Parent Rated Repetitive Behavior Scale Revised (RBS-R) Scores During Treatment.(baseline; 4-week; 8-week)
- Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment.(baseline; 4-week; 8-week)
- Change From Baseline on Electrocardiogram (EKG) P Duration During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Electrocardiogram (EKG) PR Interval During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Adverse Event Severity(Up to 12 weeks)
- Change From Baseline on Electrocardiogram (EKG) QRS Interval During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Electrocardiogram (EKG) QT Interval During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (Sodium) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (Potassium) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (Chloride) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (CO2) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (Anion Gap) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (Glucose) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (Creatinine) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (Urea Nitrogen) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (Calcium) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Blood Clinical Labs (Osmolality) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Urine Clinical Labs (Osmolality) During Treatment.(baseline to 4-week, 8-week, and 12-week)
- Change From Baseline on Vital Signs (Systolic Blood Pressure) During Treatment.(Up to 12 weeks)
- Change From Baseline on Vital Signs (Diastolic Blood Pressure) During Treatment.(Up to 12 weeks)
- Change From Baseline on Vital Signs (Pulse) During Treatment.(Up to 12 weeks)
- Change From Baseline on the Dosage Record Treatment Emergent Symptom Scale (DOTES) During Treatment.(Up to 12 weeks)
- Change From Baseline in Overt Aggression Scale (OAS) During Treatment.(2-week, 4-week; 6-week, 8-week)
研究者
Antonio Hardan
Principal Investigator
Stanford University