跳至主要内容
MedPath Trial logoMedPath TrialMedPath Trial Home
临床试验/NCT03514420
NCT03514420已完成2 期

An Open-label Phase 2 Study of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Subjects With Familial Partial Lipodystrophy

Akcea Therapeutics1 个研究点 分布在 1 个国家目标入组 4 人开始时间: 2018年6月15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
2 期
状态
已完成
入组人数
4
试验地点
1
主要终点
Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27)
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

This is a single-center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of fasting triglycerides in participants with familial partial lipodystrophy.

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
18 Years 至 —（Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •Must give written informed consent to participate in the study.
  • •Clinical diagnosis of familial partial lipodystrophy plus diagnosis of type 2 diabetes mellitus and hypertriglyceridemia.
  • •Diagnosis of diabetes mellitus, made at least 6 months prior to the Screening with hemoglobin A1c (HbA1c) ≥ 7% to ≤ 12% at Screening and on anti-diabetic therapy as defined in study protocol.
  • •Hypertriglyceridemia as defined by fasting triglycerides (TG) levels ≥ 500 milligrams per deciliter (mg/dL) at both Screening and Qualification visits. Participants with the clinical diagnosis of FPL and with fasting TG levels ≥ 200 (≥ 2.26 millimoles per liter [mmol/L]) to < 500 mg/dL (≥ 5.7 mmol/L) who meet the genetic or family history criteria for study inclusion may be further screened and enrolled in the study.
  • •Presence of hepatosteatosis (fatty liver), as evidenced by a Screening magnetic resonance imaging (MRI) indicating a hepatic fat fraction (HFF) ≥ 6.4%.

排除标准

  • •Diagnosis of generalized lipodystrophy.
  • •Diagnosis of acquired partial lipodystrophy (APL).
  • •Acute pancreatitis within 4 weeks of Screening.
  • •Acute coronary syndrome within 6 months of Screening.
  • •Major surgery within 3 months of Screening.
  • •Have any other conditions in the opinion of the investigator which could interfere with the participant participating in or completing the study.

研究组 & 干预措施

AKCEA-ANGPTL3-LRx 20 mg

Experimental

Participants received AKCEA-ANGPTL3-LRx 20 milligrams (mg) administered every week for 26 weeks by subcutaneous (SC) injection.

干预措施: AKCEA-ANGPTL3-LRx (Drug)

结局指标

主要结局

Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27)

时间窗: Baseline and End of the Treatment (Week 27)

The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.

次要结局

  • Change From Baseline in AUC of Serum Ghrelin as Assessed by MMT at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in ApoC-III: LDL at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in ApoC-III: HDL at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Lipoprotein a (Lp[a]) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Free Fatty Acid (FFA) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in AUC of Serum Insulin as Assessed by MMT at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in ApoB at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Area Under the Curve (AUC) of Plasma Glucose as Assessed by Mixed Meal Test (MMT) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in AUC of Serum C-peptide as Assessed by MMT at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in ApoB-48 at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Apolipoprotein B 100 (ApoB-100) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Non-HDL-C at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in ApoA-1 at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in ApoC-III at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in ApoC-III: Chylomicron at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in ApoC-III: VLDL at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in AUC of Free Fatty Acid (FFA) as Assessed by MMT at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in AUC of Incretin Hormone (Gastric Inhibitory Polypeptide [GIP]) as Assessed by MMT at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in AUC of Incretin Hormone (Glucagon-like Peptide -1 [GLP-1]) as Assessed by MMT at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in AUC of Peptide Tyrosine Tyrosine (PYY) as Assessed by MMT at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in HDL-C at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in LDL-C at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Total Cholesterol (TC) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in VLDL-C at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Glycerol Levels at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Lipoprotein Particle Size at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Hemoglobin A1c (HbA1c) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Homeostasis Model Assessment-Estimated Insulin Resistance (HOMA-IR)(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Adiponectin at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in and Leptin at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Hepatic Fat Fraction (HFF) as Assessed by Magnetic Resonance Imaging (MRI) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Skinfold Thickness at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Changes From Baseline in Body Fat Distribution for Total Bone Mineral Density in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Visceral Adipose Tissue (VAT) as Measured by Magnetic Resonance Imaging (MRI) at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Subcutaneous Adipose Tissue (SAT) as MRI at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Body Weight at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Waist Circumference at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Waist/Hip Ratio at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Quality of Life (QoL)(Baseline and End of the Treatment (Week 27))
  • Change From Baseline in Pain Score at End of the Treatment(Baseline and End of the Treatment (Week 27))
  • Number of Participants With Treatment-emergent Adverse Events (TEAEs)(From signing of informed consent to end of follow up period (Up to week 40))

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (1)

Loading locations...

相似试验

已完成
2 期
Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)Familial Chylomicronemia SyndromeLipoprotein Lipase DeficiencyHyperlipoproteinemia Type 1
NCT03360747Akcea Therapeutics3
撤回
2 期
Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)Homozygous Familial Hypercholesterolemia
NCT03455777Akcea Therapeutics
已完成
2 期
3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)Amyotrophic Lateral Sclerosis
NCT05039268Macquarie University, Australia16
Unknown
3 期
TONKA Versus Legalon for Lowering Hepatic Enzymes in Liver Function Disorder Patients.Liver Function Failure
NCT03216668Nhat Nhat Pharmaceutical Company140
已完成
3 期
A Study of Atezolizumab (Tecentriq) in Combination With Bevacizumab to Investigate Safety and Efficacy in Patients With Unresectable Hepatocellular Carcinoma Not Previously Treated With Systemic Therapy-AmethistaCarcinoma, Hepatocellular
NCT04487067Hoffmann-La Roche152