jRCT2031240472招募中不适用
A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared with Even-chain Medium-chain Triglycerides (MCT) on Major Clinical Events (MCEs) in Pediatric Patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 4
- 主要终点
- -
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomized Controlled Trial
- 干预模型
- Parallel Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Double Blind
入排标准
- 年龄范围
- No limit 至 18age old not（—）
- 性别
- All
入选标准
- •Confirmed diagnosis of LC-FAOD: carnitine palmitoyl transferase (CPT) I deficiency, CPT II deficiency, carnitine/acylcarnitine translocase (CACT) deficiency, very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency, long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency, or mitochondrial trifunctional protein (TFP) deficiency. Diagnosis must be confirmed by results of acylcarnitine profiles, fatty acid oxidation probe studies in cultured fibroblasts, or mutation analysis obtained from medical records
- •Males and females, from 0 (including newborns) to < 18 years of age
- •Have ANY ONE of the following significant clinical manifestations of LC-FAOD:
- •At least 2 in the prior year, or 3 in the prior 2 years, of severe major episodes of metabolic decompensation (eg, hypoglycemia, rhabdomyolysis, or exacerbation of cardiomyopathy, requiring ER/urgent care unit visits or hospitalizations)
- •Recurrent symptomatic hypoglycemia (clinical symptoms of hypoglycemia) requiring intervention
- •Susceptibility to hypoglycemia after short periods of fasting (less than 4 to 12 hours, depending on age)
- •Evidence of functional cardiomyopathy requiring ongoing medical management or clinical manifestation of heart failure
- •Sibling(s) with the same pathogenic variant who presented with MCEs
- •Subject with pathogenic variants that are known or suspected to be associated with absent or severely reduced enzyme activity or with severe disease manifestations
- •Note: Additional inclusion/exclusion criteria may apply, per protocol.
排除标准
- •Treatment with triheptanoin within 60 days of Screening
- •History of known hypersensitivity to triheptanoin or MCT
- •Have any comorbid conditions, including unstable major organ-system disease(s) that in the opinion of the Investigator places the subject at increased risk of complications, interferes with study participation or compliance, or confounds study objectives or interpretation of results. History of metabolic decompensation(s) with metabolic acidosis, hyperammonemia, and/or liver enzyme elevations does not constitute an exclusion criterion unless in the opinion of the Investigator places the subject at increased risk of complications, interferes with study participation or compliance, or confounds study objectives or interpretation of results.
- •Have a diagnosis of pancreatic insufficiency
- •Note: Additional inclusion/exclusion criteria may apply, per protocol.
结局指标
主要结局
-
- Annualized event rate of MCEs
次要结局
- Change from baseline to 6 months in hepatic PDFF%, assessed by 1H-MRS in subjects enrolled in the Liver Substudy(6 months)
- Change from baseline in scores for Caregiver-reported PedsQL 4.0 Generic Core Scale
- Change from baseline in scores for PedsQL Infant Scale
研究者
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