跳至主要内容
MedPath Trial logoMedPath TrialMedPath Trial Home
临床试验/NCT06049680
NCT06049680招募中4 期

A Single-arm Open-label Safety Study of SMOFlipid to Evaluate the Risk of Developing Essential Fatty Acid Deficiency (EFAD) and/or Parenteral Nutrition-associated Cholestasis (PNAC) in Pediatric Patients 1 Month to 17 Years of Age and in Adult Patients, Who Are Anticipated to Need 8 Weeks or Longer of Parenteral Nutrition Treatment

Fresenius Kabi9 个研究点 分布在 1 个国家目标入组 100 人开始时间: 2024年10月28日最近更新:
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit
适应症
干预措施
相关药物

试验速览

阶段
4 期
状态
招募中
入组人数
100
试验地点
9
主要终点
Incidence of PNAC
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

Evaluate the risk of developing EFAD and/or PNAC in adult and pediatric patients 1 month of age and older, who are anticipated to need 8 weeks or longer of parenteral nutrition treatment with SMOFlipid.

研究设计

研究类型
Interventional
分配方式
Na
干预模型
Single Group
主要目的
Other
盲法
None

入排标准

年龄范围
1 Month 至 17 Years（Child）
性别
All
接受健康志愿者
否

入选标准

  • •Age Limits: and Adults
  • •Inclusion Criteria:
  • •Male or female patients, at least 1 month of age.
  • •Patients who require PN for at least 5 days/week.
  • •Patients who receive 80% or more of their total energy requirements as PN at enrollment and who are expected to receive 80% or more of their total energy requirements as PN for at least 56 days.
  • •Written informed consent. In case of pediatric patients, informed consent must be obtained from parent(s) or legal representatives. If possible, the assent of the pediatric patient must also be obtained (according to local law).

排除标准

  • •Use of any other lipid injectable emulsion than SMOFlipid within 6 months prior to study participation
  • •Known hypersensitivity to fish, egg, soybean, or peanut proteins, or to any of the active ingredients or excipients of SMOFlipid.
  • •Hyperlipidemia or disorders of lipid metabolism characterized by hypertriglyceridemia (serum triglyceride concentration >250 mg/dL in infants or >400 mg/dL in older pediatric and adult patients).
  • •Inborn errors of amino acid metabolism.
  • •Cardiopulmonary instability (including pulmonary edema, cardiac insufficiency, myocardial infarction, acidosis and hemodynamic instability requiring significant vasopressor support).
  • •Hemophagocytic syndrome.
  • •Liver enzymes (either AST, or ALT, or GGT) exceeding 2 x upper limit of normal range
  • •Direct bilirubin exceeding 2 x upper limit of normal range
  • •INR exceeding 2 x upper limit of normal range and patient not receiving oral anticoagulants.
  • •Any known hepatic condition outside of IFALD that will increase direct bilirubin ≥2.0 mg/dL.
  • •Clinically significant abnormal levels of any serum electrolyte (sodium, potassium, magnesium, calcium, chloride, phosphate).
  • •Active bloodstream infection demonstrated by positive blood culture at screening.
  • •Severe renal failure (eGFR <15 ml/min per 1.73 m2) including patients on renal replacement therapy.
  • •Abnormal blood pH, oxygen saturation, or carbon dioxide.
  • •Pregnancy or lactation.
  • •Participation in another interventional clinical study.
  • •Unlikely to survive longer than 56 days.

研究组 & 干预措施

Single arm SMOFlipid® (lipid injectable emulsion)

Other

Investigational drug: SMOFlipid® (lipid injectable emulsion).

干预措施: SMOFlipid® (lipid injectable emulsion) (Drug)

结局指标

主要结局

Incidence of PNAC

时间窗: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).

defined as direct bilirubin level \> 2 mg/dL, confirmed by a second sample collected 7 days after the first sample

Incidence of EFAD

时间窗: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).

Triene/tetraene ratio in total plasma to assess EFAD (Holman 1960), Severity of EFAD will be graded based on triene/tetraene ratio, as suspected ≥ 0.05, moderate ≥ 0.20 and severe ≥ 0.40 (Cober et al 2012)

Time to direct bilirubin > 2mg/dL

时间窗: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).

(confirmed by a second sample collected 7 days after the first)

Incidence of clinical EFAD

时间窗: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).

Fatty acids

时间窗: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).

analyzed in total plasma, including linoleic acid, α-linolenic acid, arachidonic acid, docosahexaenoic acid, eicosapentaenoic acid and Mead acid

次要结局

  • Laboratory values: alkaline phosphatase (ALP)(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: creatinine(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values hematology: International normalized ratio(At baseline before start of treatment to assess eligibility)
  • Vital signs: Blood pressure(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: direct bilirubin(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: urea nitrogen(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values hematology Hematocrit(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Incidence of adverse events(Start of Treatment until After End of Last Study PN (+6 months for pediatric patients). Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: total bilirubin(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: gamma-glutamyl transferase (GGT)(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: electrolytes(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: glucose(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: total protein(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: triglycerides(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: aspartate aminotransferase (AST)(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values hematology: white blood cell count (WBC), red blood cell count (RBC), and platelet count(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Vital signs: Body temperature(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • BMI(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values: alanine aminotransferase (ALT)(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Laboratory values hematology: Hemoglobin(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)
  • Vital signs: Heart rate(Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).)

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (9)

Loading locations...

相似试验

已完成
1 期
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)Hypophosphatasia (HPP)
NCT00744042Alexion Pharmaceuticals, Inc.11
已完成
1 期
A Dose Escalation Study of SF1126, a PI3 Kinase (PI3K) Inhibitor, Given By Intravenous (IV) Infusion in Patients With Solid TumorsAdvanced or Metastatic Solid TumorsSolid CancersCancer
NCT00907205Semafore Pharmaceuticals44
终止
不适用
Amlodipine And Olmesartan Medoxomil In Hypertensive Filipino PatientsHypertension
NCT01200407Pfizer615
终止
1 期
Safety and Efficacy Study of GCS-100LE in the Treatment of Multiple MyelomaMultiple Myeloma
NCT00609817La Jolla Pharmaceutical Company9
终止
2 期
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase DeficiencyLysosomal Acid Lipase Deficiency
NCT02193867Alexion Pharmaceuticals, Inc.10