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临床试验/NCT02527200
NCT02527200已完成3 期

Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome.

Novo Nordisk A/S1 个研究点 分布在 1 个国家目标入组 56 人开始时间: 2015年11月9日最近更新:
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适应症
干预措施
相关药物

试验速览

阶段
3 期
状态
已完成
入组人数
56
试验地点
1
主要终点
Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 16 Weeks
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 trial is conducted globally. The aim of this trial is to investigate the effect of liraglutide for weight management in paediatric subjects with Prader-Willi Syndrome.

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
Double (Participant, Investigator)

入排标准

年龄范围
6 Years 至 18 Years（Child, Adult）
性别
All
接受健康志愿者
否

入选标准

  • •Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
  • •Confirmed diagnosis of PWS (Prader-Willi Syndrome) (by genetic testing)
  • •Male or female, age at the time of signing informed consent: - Part A: above or equal to 12 years and less than 18 years
  • •Tanner stage 2-5 pubertal development for part A, and Tanner stage 1 for part B
  • •BMI (body mass index) corresponding to equal or above 30 kg/m^2 for adults by international cut-off points1 and equal or above the 95th percentile for age and sex (for diagnosis of obesity)
  • •Stable body weight during the previous 90 days before screening ( below 10 kg self-reported weight change)
  • •Testing has been performed to evaluate for adrenal insufficiency and documented in medical record

排除标准

  • •Type 1 diabetes mellitus (T1DM)
  • •Type 2 diabetes mellitus (T2DM)
  • •Calcitonin equal or above 50 ng/L
  • •No change in treatment plan with growth hormone (GH) from randomisation to the end of the open-label period patients on growth hormone to stay on, patients off GH to stay off during this period. Adjustments in doses of growth hormone will be permitted)
  • •Family or personal history of Multiple Endocrine Neoplasia Type 2 (MEN2) or Medullary Thyroids Carcinoma (MTC)
  • •History of pancreatitis (acute or chronic)
  • •Treatment with any medication prescribed for weight loss within 90 days before screening (e.g. orlistat, zonisamide, topiramate/phentermine, lorcaserin, phentermine, bupropion/naltrexone,liraglutide, metformin)
  • •Untreated adrenal insufficiency
  • •Suggestive history of, or significant risk of gastroparesis (e.g. marked abdominal bloating post meal, history of vomiting, severe constipation), as judged by the Investigator

研究组 & 干预措施

Liraglutide

Experimental

干预措施: liraglutide (Drug)

Placebo

Placebo Comparator

干预措施: placebo (Drug)

结局指标

主要结局

Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 16 Weeks

时间窗: Week 0, Week 16

Change in BMI SDS from baseline to week 16 is presented. BMI SDS also called Z-scores, was calculated using the following formula: Z=\[(y / M)\^L - 1\] / S\*L; where L, M and S are median (M), Box-cox power (L) and variation coefficient (S) of children/adolescents', y= individual BMI. BMI provided for each sex and age. For each participant, a standard deviation score Z (SDS) was calculated based on age and sex referring to the values L, M and S. Possible values range from -3 to +3, a negative score being beneficial.

Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 52 Weeks

时间窗: Week 0, Week 52

Change in BMI SDS from baseline to week 52 is presented. BMI SDS also called Z-scores, was calculated using the following formula: Z=\[(y / M)\^L - 1\] / S\*L; where L, M and S are median (M), Box-cox power (L) and variation coefficient (S) of children/adolescents', y= individual BMI. BMI provided for each sex and age. For each participant, a standard deviation score Z (SDS) was calculated based on age and sex referring to the values L, M and S. Possible values range from -3 to +3, a negative score being beneficial.

次要结局

  • Percentage of Participants Achieving ≥ 10% Reduction in Baseline BMI at Week 16(At week 16)
  • Percentage of Participants With no Increase in BMI SDS at Week 52(At week 52)
  • Change in BMI From Baseline at Week 16(Week 0, week 16)
  • Percentage of Participants Achieving ≥ 5% Reduction in Baseline BMI at Week 16(At week 16)
  • Percentage of Participants Achieving ≥ 5% Reduction in Baseline BMI at Week 52(At week 52)
  • Percentage of Participants Achieving ≥ 10% Reduction in Baseline BMI at Week 52(At week 52)
  • Change in BMI From Baseline at Week 52(Week 0, week 52)
  • Change in Body Weight (Kilogram (kg)) From Baseline at Week 16(Week 0, week 16)
  • Change in Body Weight (kg) From Baseline at Week 52(Week 0, week 52)
  • Change in Body Weight (Pounds (lb)) From Baseline at Week 16(Week 0, week 16)
  • Change in Body Weight (lb) From Baseline at Week 52(Week 0, week 52)
  • Change in Body Weight (Percentage [%]) From Baseline at Week 16(Week 0, week 16)
  • Change in Body Weight (%) From Baseline at Week 52(Week 0, week 52)
  • Change in Waist Circumference From Baseline at Week 52(Week 0, week 52)
  • Change in Hyperphagia Score: Total Score and Hyperphagic Behaviour, Drive and Severity Score From Baseline at Week 16(Week 0, week 16)
  • Percentage of Participants With no Increase in BMI SDS at Week 16(At week 16)
  • Change in Waist Circumference From Baseline at Week 16(Week 0, week 16)
  • Change in Total Cholesterol From Baseline at Week 52(Week 0, week 52)
  • Change in Low Density Lipoprotein (LDL)-Cholesterol From Baseline at Week 16(Week 0, week 16)
  • Change in Very Low-density Lipoprotein (VLDL) Cholesterol From Baseline at Week 52(Week 0, week 52)
  • Change in Waist-to-hip Circumference Ratio From Baseline at Week 16(Week 0, week 16)
  • Change in Waist-to-hip Circumference Ratio From Baseline at Week 52(Week 0, week 52)
  • Change in Hyperphagia Score: Total Score and Hyperphagic Behaviour, Drive and Severity Score From Baseline at Week 52(Week 0, week 52)
  • Change in High Sensitivity C Reactive Protein (hsCRP) From Baseline at Week 16(Week 0, week 16)
  • Change in High Sensitivity C Reactive Protein (hsCRP) From Baseline at Week 52(Week 0, week 52)
  • Change in Total Cholesterol From Baseline at Week 16(Week 0, week 16)
  • Change in Low Density Lipoprotein (LDL)-Cholesterol From Baseline at Week 52(Week 0, week 52)
  • Change in High Density Lipoprotein (HDL)-Cholesterol From Baseline at Week 16(Week 0, week 16)
  • Change in Non-high Density Lipoprotein (Non-HDL) Cholesterol From Baseline at Week 52(Week 0, week 52)
  • Change in High Density Lipoprotein (HDL)-Cholesterol From Baseline at Week 52(Week 0, week 52)
  • Change in Triglycerides From Baseline at Week 52(Week 0, week 52)
  • Change in Fasting Lipid: Free Fatty Acids (FFA) From Baseline at Week 52(Week 0, week 52)
  • Change in Fasting C Peptide From Baseline at Week 52(Week 0, week 52)
  • Change in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) From Baseline at Week 52(Week 0, week 52)
  • Change in Non-high Density Lipoprotein (Non-HDL) Cholesterol From Baseline at Week 16(Week 0, week 16)
  • Change in Very Low-density Lipoprotein (VLDL) Cholesterol From Baseline at Week 16(Week 0, week 16)
  • Change in Systolic and Diastolic Blood Pressure From Baseline at Week 16(Week 0, week 16)
  • Change in Systolic and Diastolic Blood Pressure From Baseline at Week 52(Week 0, week 52)
  • Change in FPG From Baseline at Week 52(Week 0, week 52)
  • Change in Fasting C Peptide From Baseline at Week 16(Week 0, week 16)
  • Change in Triglycerides From Baseline at Week 16(Week 0, week 16)
  • Change in HbA1c From Baseline at Week 52(Week 0, week 52)
  • Change in Fasting Insulin From Baseline at Week 16(Week 0, week 16)
  • Number of Participants in Glycaemic Category at Week 16(week 16)
  • Number of Participants in Glycaemic Category at Week 52(Week 52)
  • Number of Participants With Change in ECG From Baseline at Week 52(Week 0, Week 52)
  • Change in Pulse From Baseline at Week 16(Week 0, week 16)
  • Change in Pulse From Baseline at Week 52(Week 0, week 52)
  • Change in Haematology: Haemoglobin From Baseline at Week 16(Week 0, week 16)
  • Change in Haematology: Haemoglobin From Baseline at Week 52(Week 0, week 52)
  • Change in Haematology: Haematocrit From Baseline at Week 16(Week 0, week 16)
  • Change in Haematology: Haematocrit From Baseline at Week 52(Week 0, week 52)
  • Change in Fasting Lipid: Free Fatty Acids (FFA) From Baseline at Week 16(Week 0, week 16)
  • Change in Glycosylated Haemoglobin (HbA1c) From Baseline at Week 16(Week 0, week 16)
  • Change in Homeostasis Model Assessment of Beta-cell Function (HOMA-B) From Baseline at Week 52(Week 0, week 52)
  • Change in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) From Baseline at Week 16(Week 0, week 16)
  • Number of Treatment Emergent Adverse Events(From week 0 to week 54)
  • Number of Severe Treatment Emergent Episodes of Hypoglycaemia(From week 0 to week 54)
  • Number of Blood Glucose Confirmed Symptomatic Episodes of Hypoglycaemia(From week 0 to week 54)
  • Number of Participants With Occurrence of Anti-liraglutide Antibodies(From week 0 to week 54)
  • Number of Participants With Change in Electrocardiogram (ECG) From Baseline at Week 16(Week 0, Week 16)
  • Change in Fasting Plasma Glucose (FPG) From Baseline at Week 16(Week 0, week 16)
  • Change in Fasting Insulin From Baseline at Week 52(Week 0, week 52)
  • Change in Homeostasis Model Assessment of Beta-cell Function (HOMA-B) From Baseline at Week 16(Week 0, week 16)
  • Change in Haematology: Thrombocytes, Leukocytes, Eosinophils, Neutrophils, Basophils, Lymphocytes, Monocytes From Baseline at Week 16(Week 0, week 16)
  • Change in Haematology: Thrombocytes, Leukocytes, Eosinophils, Neutrophils, Basophils, Lymphocytes, Monocytes From Baseline at Week 52(Week 0, week 52)
  • Change in Haematology: Erythrocytes From Baseline at Week 16(Week 0, week 16)
  • Change in Haematology: Erythrocytes From Baseline at Week 52(Week 0, week 52)
  • Change in Biochemistry: Creatinine and Bilirubin (Total) From Baseline at Week 16(Week 0, week 16)
  • Change in Biochemistry: Creatinine and Bilirubin (Total) From Baseline at Week 52(Week 0, week 52)
  • Change in Biochemistry: Creatine Kinase, Amylase, Lipase, Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (ALP) From Baseline at Week 16(Week 0, week 16)
  • Change in Biochemistry: Creatine Kinase, Amylase, Lipase, Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (ALP) From Baseline at Week 52(Week 0, week 52)
  • Change in Biochemistry: Urea, Sodium, Potassium, Calcium Total and Calcium Albumin-corrected From Baseline at Week 16(Week 0, week 16)
  • Change in Biochemistry: Urea, Sodium, Potassium, Calcium Total and Calcium Albumin-corrected From Baseline at Week 52(Week 0, week 52)
  • Change in Biochemistry: Albumin From Baseline at Week 16(Week 0, week 16)
  • Change in Biochemistry: Albumin From Baseline at Week 52(Week 0, week 52)
  • Change in Hormone Level: Carcinoembryonic Antigen (CEA) From Baseline at Week 16(Week 0, week 16)
  • Change in Hormone Level: Carcinoembryonic Antigen (CEA) From Baseline at Week 52(Week 0, week 52)
  • Change in Hormone Level: Calcitonin From Baseline at Week 16(Week 0, week 16)
  • Change in Hormone Level: Calcitonin From Baseline at Week 52(Week 0, week 52)
  • Change in Hormone Level: Thyroid Stimulating Hormone (TSH) and Prolactin From Baseline at Week 16(Week 0, week 16)
  • Change in Hormone Level: Thyroid Stimulating Hormone (TSH) and Prolactin From Baseline at Week 52(Week 0, week 52)
  • Change in Hormone Level: Free Thyroxine (Free T4) and Adrenocorticotropic Hormone (ACTH) From Baseline at Week 16(Week 0, week 16)
  • Change in Hormone Level: Free Thyroxine (Free T4) and Adrenocorticotropic Hormone (ACTH) From Baseline at Week 52(Week 0, week 52)
  • Change in Hormone Level: Insulin-like Growth Factor-1 (IGF-1) and Cortisol From Baseline at Week 16(Week 0, week 16)
  • Change in Hormone Level: Insulin-like Growth Factor-1 (IGF-1) and Cortisol From Baseline at Week 52(Week 0, week 52)
  • Change in Hormone Level: Dehydroepiandrosterone Sulfate (DHEAS) From Baseline at Week 16(Week 0, week 16)
  • Change in Hormone Level: Dehydroepiandrosterone Sulfate (DHEAS) From Baseline at Week 52(Week 0, week 52)
  • Change in Hormone Level: Luteinising Hormone (LH), Follicle Stimulating Hormone (FSH) From Baseline at Week 16(Week 0, week 16)
  • Change in Hormone Level: Luteinising Hormone (LH), Follicle Stimulating Hormone (FSH) From Baseline at Week 52(Week 0, week 52)
  • Change in Hormone Level: Estradiol (Females) From Baseline at Week 16(Week 0, week 16)
  • Change in Hormone Level: Estradiol (Females) From Baseline at Week 52(Week 0, week 52)
  • Change in Hormone Level: Testosterone (Males) From Baseline at Week 16(Week 0, week 16)
  • Change in Hormone Level: Testosterone (Males) From Baseline at Week 52(Week 0, week 52)
  • Number of Participants With Change in Pubertal Status From Baseline at Week 16(Week 0, week 16)
  • Number of Participants With Change in Pubertal Status From Baseline at Week 52(Week 0, week 52)
  • Number of Participants With Change in Physical Examination From Baseline at Week 16(Week 0, week 16)
  • Number of Participants With Change in Physical Examination From Baseline at Week 52(Week 0, week 52)
  • Height Velocity at Week 16(Week 16)
  • Height Velocity at Week 52(week 52)
  • Part A: Number of Participants With Change in Columbia Suicidality Severity Rating Scale (C-SSRS) at Week 16(Week 0, week 16)
  • Part A: Number of Participants With Change in Columbia Suicidality Severity Rating Scale (C-SSRS) at Week 52(Week 0, week 52)
  • Part A: Change in Patient Reported Health Questionnaire-9 (PHQ-9) From Baseline at Week 16(Week 0, week 16)
  • Part A: Change in Patient Reported Health Questionnaire-9 (PHQ-9) From Baseline at Week 52(Week 0, week 52)

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (1)

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