NCT03149445已完成2 期
A Double-Blind, Randomized, Placebo-Controlled, Multiple-Dose, Multi-Center Safety and Efficacy Study of Co-Administration of Tesofensine/Metoprolol for 12 Weeks in Adult and Adolescent Patients With Prader-Willi Syndrome (PWS), Followed by Two Open Label 12 Weeks Extension Periods for Adolescent Patients
Saniona2 个研究点 分布在 2 个国家目标入组 18 人开始时间: 2017年3月30日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 18
- 试验地点
- 2
- 主要终点
- Percent Change From Baseline to End of Treatment in Mean Body Weight
研究概览
简要总结
Two-centre, double-blind, placebo-controlled, randomized, and multiple-dose clinical study followed by two open label extension periods.
详细描述
Two-centre, double-blind, placebo-controlled, randomized, and multiple-dose clinical study. Study medication will be administered for 91 days. The study will be conducted in two steps:
- Step 1 - 9 adult subjects with PWS was treated.
- Sponsor review - following the completion of the treatment of the adult subjects, unblinded efficacy, safety, Pharmacokinetic (PK) data as well as all data from the study in subjects with type 2 diabetes (TM001) will be reviewed by sponsor and an interim analysis will be done. Following competent authority positive opinion regarding the interim analysis and unblinded data the study will proceed to:
- Step 2 - 9 adolescent subjects with PWS was treated.
- OLE (Open Label Extension) I - Participation in a 12-week OLE I was offered to subjects who completed Step 2. 8 subjects entered OLE I.
- OLE (Open Label Extension) II - Participation in a 12-week OLE II was offered to subjects who completed OLE I. 6 subjects continued to OLE II.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
double-blind
入排标准
- 年龄范围
- 12 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females with a confirmed genetic diagnosis of Prader-Willi syndrome
- •Step 1: Adults aged 18-30
- •Step 2: Adolescents aged 12-17
- •Body Mass Index (BMI):
- •Step 1: Adults with ≥25 kg/m2
- •Step 2: Children with a BMI >85th percentile for the same age and sex
- •Normal Blood Pressure (BP) or well managed hypertension (only if dose of BP medication(s) has been stable for >2 months)
- •Normal lipid profile or well managed dyslipidemia (only if dose of lipid-lowering medication(s) has been stable for >2 months)
- •Growth hormone is allowed; but patient must be on stable dose of growth hormone >2 months
- •Type 2 diabetes is allowed, but the following criteria must be met:
- •HbA1c <10.0 % not being managed with insulin within the past 3 months
- •Patients taking GLP-1 analogues (e.g. exenatide, liraglutide) must have been on stable dose for >3 months
- •Fasting plasma glucose <11.0 mmol/l
排除标准
- •Step 1: Adults with >140/90
- •Step 2: Adolescents with ≥95th percentile for gender, age, and height
- •Heart Rate (HR) ≥ 90, <50 bpm
- •Hypersensitivity to tesofensine/metoprolol
- •Type 1 diabetes
- •Heart failure New York Heart Association (NYHA) level II or greater, decompensated heart failure
- •Previous myocardial infarction or stroke
- •Diagnosis of schizophrenia, bipolar disorder, personality disorder or other DSM-III disorders, or any other psychiatric condition, which in the investigator's opinion will interfere significantly with study compliance
- •History of major depressive disorder or suicidality
- •Any clinically significant cardiac arrhythmia
- •Treatment with calcium channel blockers and beta blockers
- •Concomitant use of monoaminooxidase inhibitors
- •Bulimia or anorexia nervosa
- •Any agent used for weight loss in the past 3 months
- •Untreated hypo- or hyperthyroidism
- •Clinically significant liver (>3x ULN (Upper Limit of Normal range)) and/or kidney impairment
- •More than 5% weight loss within the last 3 months
- •Any other clinically meaningful condition, in the opinion of the investigator, which would make participation potentially unsafe
- •Contraindications to administration of metoprolol per current Summary of Product Characteristics
研究组 & 干预措施
Tesofensine/Metoprolol
Experimental
Tesofensine + metoprolol administered once a day, in the morning with a meal
干预措施: Tesofensine/Metoprolol (Drug)
Tesofensine/Metoprolol placebo
Placebo Comparator
Placebo tablets matching tesofensine + metoprolol administered once a day, in the morning with meal
干预措施: Placebos (Drug)
结局指标
主要结局
Percent Change From Baseline to End of Treatment in Mean Body Weight
时间窗: DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271
Percent change from baseline to end of treatment in mean body weight. LOCF.
次要结局
- Change From Baseline to End of Treatment in Mean Body Weight(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Steady State Concentrations of Tesofensine and Metoprolol as Measured by Trough Values(DB Step 1: Day 29; DB Step 2: Day 29; OLE I: Day 120; OLE II: Day 210)
- Change From Baseline to End of Treatment in Fat- and Fat Free Mass (%) by Dual X-ray Absorptiometry (DEXA)(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in Bone Mineral Density (BMD) by Dual X-ray Absorptiometry (DEXA)(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in Bone Mineral Content (BMC) by Dual X-ray Absorptiometry (DEXA)(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in Heart Rate (HR)(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Total Number of Adverse Events(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in PR Interval(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in Electrocardiogram (ECG) Parameters(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in HbA1c(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in Insulin(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Change From Baseline to End of Treatment in Fasting pl. Glucose, Triglycerides, Low-density Lipoprotein (LDL) and High-density Lipoprotein (HDL) Cholesterol(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
- Number of Subjects With Adverse Events (AE) and Serious Adverse Events (SAE)(DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271)
研究者
研究点 (2)
Loading locations...
相似试验
招募中
2 期
Study of Eblasakimab in Male or Female Moderate-to-Severe Atopic Dermatitis Patients Previously Treated With DupilumabAtopic DermatitisNCT05694884ASLAN Pharmaceuticals75
Unknown
2 期
Phase 2a Randomized, Double-Blind Study of Oleoyl-Estrone in Male Morbidly Obese AdultsObesityNCT00449254Manhattan Pharmaceuticals24
已完成
1 期
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial MyopathyMitochondrial MyopathyNCT02367014Stealth BioTherapeutics Inc.36
已完成
2 期
Mepolizumab in Nasal PolyposisNasal PolypsNCT01362244GlaxoSmithKline109
已完成
不适用
Study of Flavocoxid (Limbrel) vs Naproxen in Subjects With Mod-Severe Osteoarthritis of the KneeOsteoarthritisNCT00435292Primus Pharmaceuticals350