EUCTR2021-000127-12-SE进行中（未招募）1 期
A Phase 2b, Double-blind, Randomized, Placebo-controlled, Multi center, 16-week Dose finding, Safety and Efficacy Study with Open-label Extension Period of Tesomet in Adult and Adolescent Subjects with Prader-Willi Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Saniona A/S
- 入组人数
- 120
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. The subject and their legally authorized representative (ie, parent, legal guardian) and the caregiver must be willing to sign and receive a copy of their respective informed consent form(s) (ICF) after the nature and risk of study participation have been fully explained;
- •a. Informed consent(s) [and assent(s) for subjects below the legal age of consent when applicable] signed by the subject, parent, or legal guardian, as appropriate, prior to the initiation of any study procedures; and
- •b. informed consent(s) from the caregiver, signed prior to the initiation of any study procedures;
- •2. Confirmed genetic diagnosis of PWS via the central laboratory (Note: past genetic documentation is acceptable for enrollment with confirmatory methylation test done in parallel);
- •3. Female and male subjects aged 13 to 65 years at the time of informed consent;
- •4. Female subjects of non-childbearing potential (WONCBP); defined as follows: surgically sterile (ie, had a hysterectomy, or bilateral oophorectomy, or bilateral salpingectomy =6 months prior to the first dose of study drug); or Postmenopausal (no menses) for at least 1 year prior to the first dose of study drug. Postmenopausal status must be confirmed by follicle-stimulating hormone (FSH) testing at screening;
- •5. Body mass index (BMI) within the following range at Screening:
- •a. Female and male subjects 18 to 65 years of age: 27 to 60 kg/m2; or
- •b. Female and male subjects 13 to 17 years of age with a BMI ?85th percentile for age and sex;
- •6. Documented stable body weight (gain/loss <10%) for at least 90 days prior to Screening;
- •7. Total Hyperphagia Questionnaire for Clinical Trials (HQ-CT, Appendix A) score of >=13 at both Screening and at Baseline (prior to randomization) with no more than ± 3 points difference between scores;
- •8. A score of 4 (moderately ill) or higher on the Clinical Global Impression of PWS Severity scale (CGIS, Appendix A) as assessed by the Clinician at Screening and at Baseline;
- •9. Subjects must have a consistent and reliable caregiver, able and willing to be trained and comply with study requirements, and should spend at least 4 waking hours/day for a minimum of 5 days a week with this caregiver for at least 6 months prior to Screening with no planned changes in caregiver and/or duration of time spent with the caregiver throughout study participation;
- •10. Normal lipid profile as per central laboratory normal ranges at Screening or, if elevated, (ie, outside normal ranges, per the central laboratory), the subject is well managed on a stable dose of lipid-lowering medication(s) for >=90 days prior to Screening and during the study;
- •11. Growth hormone and sex hormones are allowed, but the subject must be on a stable dose of these medications ?90 days prior to Screening with no plans for dose modification during the course of the study;
- •12. Type 2 diabetes mellitus (T2DM) is allowed, provided that all of the following criteria are met:
- •a. Hemoglobin A1c (HbA1c) <8.5% at Screening;
- •b. Subjects being treated for T2DM must have been on a stable dose of anti-diabetic medication for >= 90 days prior to Screening;
- •13. Females of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline;
- •14. Females of childbearing potential and males must be willing to use a highly effective form of birth control from Screening until 90 days after their last dose of study drug;
- •15. Male subjects must agree not to donate sperm fro
排除标准
- •1. Use of any prohibited medication
- •2. Planned change of residency during the course of the study
- •3. Genotypic CYP2D6-poor metabolizers and CYP2D6 ultra-rapid metabolizers as confirmed by the central laboratory at Screening
- •4. Sitting BP that meets the following criteria after 5 minutes of rest at Screening:
- •a. Adult subjects with systolic BP >= 145 mmHg or <100 mmHg; or
- •b. Adult subjects with diastolic BP >= 95 mmHg or <70 mmHg; or
- •c. Adolescent subjects with a systolic or diastolic BP >=95th percentile for age and sex;
- •5. HR >= 95 bpm or <50 bpm at Screening and at Baseline
- •6. Corrected QT interval using Fridericia's formula >450 msec for males and >470 msec for females at Screening (by central read)
- •7. Hypersensitivity or contraindication to tesofensine or metoprolol
- •8. Type 1 diabetes mellitus
- •9. History of dementia
- •10. History of bulimia or anorexia nervosa
- •11. Subject has a rare hereditary problem of fructose intolerance, glucose galactose malabsorption, or sucrase-isomaltose insufficiency
- •12. History of clinically significant cardiac disorders as determined by the Investigator including, but not limited to, arrhythmia, myocardial infarction, prolonged QT syndrome, cardiomyopathy, New York Heart Association classification level II or greater heart failure, or decompensated heart failure as determined by the Investigator
- •13. Medical history of stroke or transient ischemic attack
- •14. Subjects who, in the Investigator’s judgment, are actively suicidal and therefore deemed to be at significant risk for suicide, or:
- •a. Subjects who answer yes” to Question 4 of the Columbia-Suicide Severity Rating Scale (C-SSRS); or
- •b. Subjects who answer yes to Question 5 of the C-SSRS (Active Suicidal Ideation with Specific Plan and Intent) on the Suicidal Ideation portion of the C-SSRS or
- •c. Subjects who answer yes to any of the suicide-related behaviors (actual attempt, interrupted attempt, aborted attempt, preparatory act or behavior) on the Suicidal Behavior portion of the C-SSRS; and the ideation or behavior occurred within 2 years prior to Screening;
- •15. Subjects who experience any of the following active clinical symptoms within 90 days prior to Screening, as defined by the Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5) and clinical judgment supported by the Brief Psychiatric Rating Scale (BPRS) administered centrally:
- •a. Delusions; b. Hallucinations; c. Major depressive disorder; d. Hypomania; or e. Suicidality;
- •16. Subjects who answer yes” to the self-mutilation/self-injury question in the C-SSRS at Screening and/or Baseline
- •17. Participation in another interventional study within 90 days prior to Screening or any prior participation in a tesofensine or Tesomet clinical study
- •18. Uncontrolled endocrine disorders
- •19. Underlying liver impairment as evidenced by abnormal liver function test results at Screening, including alanine aminotransferase or aspartate aminotransferase abnormalities >3 x the upper limit of normal, or total bilirubin >1.5 x the upper limit of normal (except in cases of diagnosed Gilbert’s Syndrome)
- •20. Underlying kidney impairment, defined as estimated glomerular filtration rate <= 60 mL/min/1.73 m2 (using the Schwartz equation), as determined by Screening laboratory assessments performed by the central laboratory
- •21. Planned major surgery which, in the Investigator’s opinion, will interfere significantly with subject safety or compliance
- •22. Subjects with a history of substance abu
研究者
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