A Phase 2b, Double-blind, Randomized, Placebo-controlled, Multi-center, 16-week Dose Finding, Safety and Efficacy Study With Open-label Extension (OLE) Period of Tesomet in Subjects With Prader-Willi Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 试验地点
- 1
- 主要终点
- Hyperphagia
研究概览
简要总结
This study will evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects with PWS.
详细描述
For the double-blind portion of the study, dosing will be initiated in a subgroup of adults who are 18-65 years of age. Following independent Data Monitoring Board review of subgroup safety data, and review and confirmation to proceed by FDA, enrollment of subjects <18 years of age will commence.
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)
入排标准
- 年龄范围
- 13 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject and their legally authorized representative must be willing to provide informed consent
- •Confirmed genetic diagnosis of PWS
- •Body mass index (BMI) within the following range at Screening:
- •Female and male subjects 18 to 65 years of age: 27 to 60 kg/m2; or
- •Female and male subjects 13 to 17 years of age with BMI that is at least 85th percentile for age and sex;
- •Female subjects must be of non-child-bearing potential
- •Documented stable body weight
- •Moderate hyperphagia at Screening and at Baseline
- •Participants must have a reliable and stable caregiver who should be able to spend an adequate amount of time with the participants to be able to address behaviors, activities and symptoms
- •Male subjects who are sexually active must be surgically sterile
排除标准
- •Females who are pregnant, breastfeeding, or actively intending to become pregnant during the study
- •Sitting BP that meets the following criteria after 5 minutes of rest at Screening:
- •Adult subjects with systolic BP >/=145 mmHg or <100 mmHg; or
- •Adult subjects with diastolic BP >/=95 mmHg or <70 mmHg; or
- •Adolescent subjects with a systolic or diastolic BP that is 95th percentile or greater for age and sex
- •Type 1 diabetes mellitus
- •History of dementia (eg, Alzheimer's disease, Parkinson's disease)
- •History of bulimia or anorexia nervosa
- •History of major depressive disorder within 2 years prior to Screening, or any history of other severe psychiatric disorder (eg, schizophrenia, bipolar disorder), or symptoms of delusions, hallucinations, or mania/hypomania within 90 days prior to Screening, as described by the Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5)
- •Uncontrolled endocrine disorders (eg, Cushing syndrome, Addison's, hypothyroidism, hyperthyroidism)
- •Medical condition or recent systemic infection that, in the opinion of the Investigator, could impact the safety of the subject
- •Use of prohibited medications, including current use of SSRIs/SNRIs
研究组 & 干预措施
Placebo
Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated Tesomet dose from the double-blind period
干预措施: Placebo (Other)
Placebo
Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated Tesomet dose from the double-blind period
干预措施: Tesomet (Drug)
Tesomet Low Dose
Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated dose from the double-blind period
干预措施: Tesomet (Drug)
Tesomet Medium Dose
Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated dose from the double-blind period
干预措施: Tesomet (Drug)
Tesomet High Dose
Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated dose from the double-blind period
干预措施: Tesomet (Drug)
结局指标
主要结局
Hyperphagia
时间窗: Baseline to Week 16
Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score
次要结局
- Overall Status Change (Clinician)(Week 16)
- Change in Body Weight(Baseline to Week 16)
- Hyperphagia Change (Caregiver)(Week 16)
- Hyperphagia Severity (Caregiver)(Baseline to Week 16)
- PWS Severity (Clinician)(Baseline to Week 16)