NCT04348890撤回1 期
A Phase I/II Open-Label, Proof-of-Concept Study of Vamorolone in Children and Adolescents With Mild-Moderately Active Ulcerative Colitis
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 主要终点
- Clinical Remission
研究概览
简要总结
This is a Phase I/II, multi-center, open-label proof-of-concept study of vamorolone. Twenty participants with a flare of mild or moderately active Ulcerative Colitis (defined as a Pediatric Ulcerative Colitis Activity Index [PUCAI] 10-60) will be enrolled and receive vamorolone 6 mg/kg/day orally once daily for 8 weeks.
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 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has provided written informed consent/Health Insurance Portability and Accountability Act (HIPAA) authorization prior to any study-related procedures;
- •Subject has current mild to moderately active ulcerative colitis, defined as a PUCAI score of 10-
- •Subject is ≥ 4 years old and <18 years old at the time of enrollment.
- •Subject has had 1) a colonoscopy demonstrating endoscopic and histologic inflammation, and/or; 2) a fecal calprotectin > 250 mcg/g, in the preceding 1 month.
- •Subject is willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
- •Subject has not started a new immunomodulator or biologic in the preceding 2 months.
- •If subject is taking an immunomodulator and/or biologic, the dose has not been changed in the last 2 months
- •Subject has a positive varicella IgG titer, or history of at least 2 documented varicella vaccines
排除标准
- •Subject is currently being treated or has received previous treatment with oral or rectal glucocorticoids (including budesonide) in the past month
- •Subject has an allergy or hypersensitivity to the study medication or to any of its constituents
- •Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator
- •Subject is taking any other investigational drug currently or has taken any other investigational drug within 3 months prior to the start of study treatment
- •Clinically significant abnormal biochemical and hematological parameters, including:
- •Neutrophil count < 1000 cells/mm3
- •Platelet count ≤ 130 cells/mm3
- •Creatinine ≥ 1.2 x the upper limit of normal
- •Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) ≥ 2x the upper limit of normal
- •Conjugated bilirubin greater than 1.
- •Has active infection with enteric pathogens (including C. difficile)
- •Has a positive PPD, Quantiferon Gold, or Interferon-gamma assay
- •Is pregnant or breast-feeding
研究组 & 干预措施
Treatment Arm
Experimental
干预措施: Vamorolone 4% suspension for oral dosing (Drug)
结局指标
主要结局
Clinical Remission
时间窗: 8 weeks
Pediatric Ulcerative Colitis Activity Index score \<10 and no additional therapy or colectomy). The PUCAI is scored from 0-85, lower numbers indicate less severe disease activity.
次要结局
- Safety (adverse events)(From day 1 through 30 days after last dose)
- Change in osteocalcin, P1NP, CTX(From baseline to week 8 and week 12)
- Change in serum CCL22(Baseline to Week 8 and Week 12)
- Change in serum trefoil factor 3(Baseline to Week 8 and Week 12)
- Cushingoid appearance(Week 8 and Week 12)
- Week 8 response(Baseline to 8 Weeks and 12 Weeks)
- Change in serum C-reactive protein(Baseline to Week 8 and Week 12)
- Change in Mayo Score Stool Frequency subscore(Baseline to 8 Weeks and 12 Weeks)
- Change in Mayo rectal bleeding score(Baseline to 8 Weeks and 12 Weeks)
- Mayo Score Stool Frequency subscore of 0 or 1(Week 12)
- Mayo Score Rectal Bleeding Score of 0(Week 12)
- Change in fecal calprotectin(Baseline to Week 8 and Week 12)
- Change in serum miRNA 146b(Baseline to Week 8 and Week 12)
研究者
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