A Multi-site, Open-Label, Pilot Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)
试验速览
- 阶段
- 4 期
- 入组人数
- 21
- 试验地点
- 6
- 主要终点
- Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS)
研究概览
简要总结
This study will evaluate the use of intravenous immunoglobulins (IVIG) at a dose of 1g/Kg/body weight given every three weeks for 6 infusions in pediatric subjects ages 4 - 16 years with moderate to severe PANS. The study will compare biomarkers and behavioral scales before treatment, after the last infusion, 2 months, and at a minimum 6 months post-treatment.
详细描述
IVIG at an immunomodulatory dose of 1 g/Kg body weight has been known to induce suppression of systemic inflammation and has been used in the treatment of autoimmune diseases. It has been proven beneficial in inflammatory conditions affecting the nervous system.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female children, 4-16 years of age
- •Diagnosis of moderate to severe PANS based on accepted criteria
- •Must be willing to follow study procedures and comply with wash-out period
- •If using prophylactic antibiotics, must be on stable dose for 3 months
排除标准
- •History of rheumatic fever, including Sydenham chorea (the neurologic manifestation)
- •Previous IVIG therapy within the last 6 months
- •Allergic reactions to blood products
- •Patients who, in the investigator's opinion, might not be suitable for the trial.
- •Steroid use
结局指标
主要结局
Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS)
时间窗: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
ADIS Child and Parent Interviews are designed to diagnose children with emotional disorder, where anxiety is a prominent component. Problem behaviors and diagnoses include school refusal behavior, separation anxiety, social phobia, specific phobia, panic disorder, agoraphobia, OCD and PTSD. Assessment of ADHD allows for differentiation of inattentive type, hyperactive-type, and combined type.
Change in Yale Global Tic Severity Scale (YGTSS)
时间窗: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
The CY-BOCS rating scale is designed to rate the severity and type of symptoms in patients with obsessive compulsive disorder (OCD). Ratings of the characteristics of obsessive or compulsive ideation and actions on a scale of 0 - 4, with 0 as none and 4 as extreme.
Change in Clinical Global Impressions (CGI)
时间窗: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
CGI is a 3-item observer-rated scale used to measure symptom severity, global improvement, and therapeutic response. Items 1 and 2 are rated on a 7-point scale; item 3 is rated from 0 to 4
Change in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
时间窗: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
The scale is a clinician-rated, 10-item scale, each item rated from 0 (no symptoms) to 4 (extreme symptoms) (total range, 0 to 40), with separate subtotals for severity of obsessions and compulsions.
Change in biomarkers levels
时间窗: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Neuro-immune biomarkers
次要结局
未报告次要终点