A Sequential, Two-Part Study to Evaluate the Clinical Benefit, Safety, Pharmacokinetics, and Pharmacodynamics of AT-007 in Pediatric Patients With Classic Galactosemia (CG)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 47
- 试验地点
- 3
- 主要终点
- Sensitivity Analysis of the Primary Endpoint (GST) with Fine Motor Skills
研究概览
简要总结
This study is designed to assess the clinical benefit as well as the safety, pharmacokinetics (PK), and pharmacodynamics (PD) (reduction of galactitol levels) of AT-007 in pediatric subjects with Classic Galactosemia (CG).
详细描述
This is a randomized, double-blind, placebo-controlled study in pediatric subjects with CG. Children with CG ages 2 through 17 years will be enrolled. The study is designed to assess the clinical benefit using validated, standardized Clinical Outcome Assessments (COAs) as well as the safety, PK, and PD (reduction of galactitol levels) of AT-007 in pediatric subjects with CG. The study consists of 2 main parts, Part A and Part B, and an open-label extension (OLE).
Part A is an intra-patient dose escalation evaluating multiple ascending doses (MAD) of AT-007.
Part B is designed to assess the clinical benefits of long-term administration of AT-007 (at the optimum dose identified in Part A) on validated, standardized COAs.
Open-label extension (OLE) is an active treatment extension for patients who received placebo treatment in Part B.
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
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Experimental: AT-007
AT-007 The starting dose in Part A will be 5 mg/kg for all age groups. For each age group, Part B of the study will not start until the optimum dose evaluated in Part A has been identified
干预措施: AT-007 (Drug)
Placebo
Placebo given orally
干预措施: Placebo (Other)
结局指标
主要结局
Sensitivity Analysis of the Primary Endpoint (GST) with Fine Motor Skills
时间窗: Month 6, Month 12 and Month 18
Sensitivity Analysis adding the National Institutes of Health Toolbox Motor Battery (NIH-MB) 9-Hole Pegboard test which measures fine motor skills. NIH-MB is described below in the secondary endpoints. For the GST with fine motor skills, change from baseline using standardized z-scores is calculated for active versus placebo.
Global Statistical Test (GST)
时间窗: Month 6, Month 12 and Month 18
The primary endpoint is a GST of four components: 1) Behavioral Symptoms Index of the Behavioral Assessment Scale for Children 3 (BASC-3); 2) Activities of Daily Living from the BASC-3; 3) Oral Expression from the Oral and Written Language Skills-II (OWLS-II); 4) Listening Comprehension from the OWLS-II. Each individual component is also a secondary endpoint on their own with the test and scoring described below in the secondary endpoint section. For the GST, change from baseline using standardized z-scores is calculated for active versus placebo.
Sensitivity Analysis of the Primary Endpoint (GST) with Cognition
时间窗: Month 6, Month 12 and Month 18
Sensitivity Analysis adding the National Institutes of Health Toolbox Cognition Battery (NIH-CB) test which measures cognition. NIH-CB is described below in the secondary endpoints. For the GST with cognition, change from baseline using standardized z-scores is calculated for active versus placebo.
次要结局
- Ovarian function in female patients determined by hormonal tests.(Month 6, Month 12 and Month 18)
- Galactitol Level(Month 1, Month 2, Month 3, Month 6, Month 12, and Month 18)
- Behavioral Assessment System for Children-3 [Individual Components](Month 6, Month 12 and Month 18)
- National Institute of Health Toolbox Cognition Battery(Month 6, Month 12 and Month 18)
- Archimedes Spiral Drawing Test(Month 6, Month 12 and Month 18)
- National Institute of Health Toolbox Motor Battery(Month 6, Month 12 and Month 18)
- Scale for Assessment & Rating of Ataxia(Month 6, Month 12 and Month 18)
- Oral and Written Language Scales-II(Month 6, Month 12 and Month 18)
- Rate of Lens Opacity(Month 6, Month 12 and Month 18)
- Sexual maturation in female patients as indicated by Tanner Stage(Month 6, Month 12 and Month 18)