2023-509809-76-00招募中3 期
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)
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 7
- 主要终点
- Annualized event rate of MCEs
研究概览
简要总结
Evaluate the effect of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of major clinical events (MCE)
研究设计
- 研究类型
- Interventional
- 分配方式
- Not Applicable
- 主要目的
- Extension period
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
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 at time of randomination
- •Have a caregiver(s) willing and able to assist in all applicable study requirements
- •Have a legally authorized representative willing and able to provide written informed consent after the nature of the study has been explained and prior to any research-related procedures, and the study subject to be able to provide age-appropriate written assent
- •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.
- •From the time of informed consent to 5 days after the last dose of study drug in this study, females of childbearing potential and fertile males must consent to use highly effective methods of contraception as described in Appendix
- •If female, agree not to become pregnant. If male, agree not to father a child or donate sperm. Inclusion Criteria for Liver Sub-study:
- •Enrollment in the Main Study of Study UX007-CL302
- •Age > 2 years
- •Liver fat content ≥ 2% and < 20% PDFF as assessed by 1H-MRS
- •Body mass index < 95th percentile
- •Able to comply with instructions (remaining still during scan) and requirements (eg, constraints on recent meals, no metallic items or implanted devices in the body, no recent contrast agents) for liver HMRS scan
排除标准
- •Enrolled in a clinical study involving concurrent use of an investigational drug product within 30 days before Screening
- •Use of a prohibited medication (eg, valproate products or pancreatic lipase inhibitors) within 30 days before Screening, or unwilling to avoid a prohibited medication or other substance that may confound study objectives
- •Treatment with triheptanoin within 60 days of Screening
- •History of known hypersensitivity to triheptanoin or MCT or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
- •Caregiver unwilling or unable to sign informed consent, or release of medical records, or follow study procedures
- •Have any comorbid conditions, including unstable major organsystem 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
- •Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study. Exclusion Criteria for Liver Sub-study
- •Acute or chronic liver disease other than LC-FAOD that presents with increased risk of liver fat (eg, hepatic cirrhosis, viral toxic or drug hepatitis, diabetes mellitus) and/or metabolic syndrome
- •Need for anesthesia/sedation to perform liver H-MRS
结局指标
主要结局
Annualized event rate of MCEs
Annualized event rate of MCEs
次要结局
- Annualized frequency and duration of cardiomyopathy-MCEs
- Annualized duration of hypoglycemic-MCEs
- Left ventricular ejection fraction, left ventricular systolic volume, and left ventricular wall mass
- Annualized frequency and duration of rhabdomyolysis-MCEs
- Annualized duration of MCEs
- Annualized hypoglycemic event-rate captured as MCEs and HCEs
- CGI-C scale score
- Change from baseline in scores for: Caregiver-reported PedsQL 4.0 Generic Core Scale (physical health summary, psychosocial health summary, and total scores) (2 years of age and older) OR PedsQL Infant Scale (physical health summary, psychosocial health summary, and total scores) (ages 1 to < 24 months)
- Survival time
- Annualized hospitalization days
- Number of missed school or learning opportunity days
- Frequency, severity, and relationship to study drug of TEAEs, serious TEAEs, and AESIs
- Incidence of TEAEs and serious TEAEs leading to dose modifications, dose reductions, treatment interruptions, discontinuations from study drug, and discontinuations from the study
- Plasma concentration levels of heptanoate and betahydroxypentanoate (BHP)
- Acceptability and Palatability Survey scores of triheptanoin mixed with oral liquids
- Change from baseline to 6 months in hepatic PDFF%, assessed by 1HMRS in subjects enrolled in the Liver Substudy
研究者
Medical Information
Scientific
Ultragenyx Pharmaceutical Inc.
研究点 (7)
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