EUCTR2015-000660-33-BE进行中（未招募）1 期
Randomized, Double-Blind, Placebo Controlled, Phase 3 Trial of Beloranib in Obese Subjects with Prader-Willi Syndrome to Evaluate Food-related Behavior, Total Body Weight, and Safety Over 52 Weeks - bestPWS|E
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Zafgen Inc.
- 入组人数
- 150
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) Age 12-50 years, inclusive.
- •2) Confirmed diagnosis of PWS due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect. Documentation of PWS, including variant of genetic defect in the subject’s medical record is sufficient.
- •3) HQ-CT score =11 (scale of 0-36).
- •a. Age 12-17 years: Body mass index (BMI) =90th percentile for age and gender (per United Kingdom [UK] Royal College of Pædiatrics and Child Health BMI charts)
- •b. Age 18-50 years: BMI =27 to =70 kg/m^2
- •5) Stable body weight for 3 months (self or guardian-reported loss/gain <10%).
- •6) If a subject has current diagnosis of T2DM, the following criteria must be met:
- •a. HbA1c <10.0%
- •b. Fasting glucose <240 mg/dL (<13.3 mmol/L)
- •c. No history of ketoacidosis or hyperosmolar coma
- •7) All study participants must agree to use an approved method of contraception throughout the study (unless subject is confirmed to be infertile or has a history of and/or commitment of long-term abstinence).
- •a. Intra-uterine device (IUD) in place from Screening through study completion as deemed appropriate for patient use by the treating physician's judgement.
- •b. Stable hormonal contraception (with approved oral, transdermal, or depot regimen) for at least 3 months prior to Screening through study completion
- •Male partners of female subjects must use a barrier method (e.g. condom) in addition to the female subject having an IUD in place or using stable hormonal contraception.
- •Males that are sexually active
- •c. Barrier method (e.g., condom) from Screening through study completion
- •AND one of the following:
- •d. Female partner has IUD in place from Screening or stable hormonal contraception for at least 3 months prior to Screening through study completion
- •e. Female partner is infertile
- •(Infertility for females is defined as surgically sterile or those who have had 12 months of amenorrhea and negative urine pregnancy test at Screening.)
- •8) Subjects must be able to provide or have a guardian who is able to provide written informed consent.
- •9) Subject must be current on immunizations prior to enrollment, as prescribed by their health care professional according to the investigative site’s standard of care.
- •10) Subjects must have at least one consistent and reliable primary caregiver who is able to accurately evaluate changes in the subject’s mood, AEs, behavior, and document MRU (e.g., planned and unplanned visits to healthcare providers, emergency room visits, hospitalization, etc.) throughout the study. The caregiver must have been caring for the subject for at least 6 months prior to study entry, is anticipated to be the subject’s primary caregiver for the duration of the trial, and spends at least 4 waking hours per day on average with the subject. The caregiver must be able to read and understand the local language or be able to communicate with Investigator/site staff.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 60
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 90
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1) Poorly controlled severe psychiatric disorders (e.g., schizophrenia, bipolar disorder, or major depressive order), recent (within 6 months) psychotic episodes, history of suicide attempts or suicidal ideation, or any other psychiatric disorders that the Investigator believes will interfere significantly with study compliance.
- •2) History of any bleeding disorders, deep vein thrombosis (DVT), or thromboembolic disease.
- •3) Current liver, renal, pulmonary, cardiac, oncologic, or GI disease which the Investigator believes is clinically significant, including:
- •a. Significant cardiovascular disease including history of congestive heart failure (CHF), coronary artery disease, myocardial infarction (MI), second degree or greater heart block , prolonged time from electrocardiogram Q wave to the end of the T wave corresponding to electrical systole (QT) syndrome, or clinically significant arrhythmias.
- •b. Fridericia-corrected QT interval (QTcF) >460 msec for males and QTcF >480 msec for females pre-dose on Day 1.
- •c. Liver disease or liver function tests, aspartate aminotransferase (AST) and alanine aminotransferase (ALT) >3x upper limit of normal (ULN), alkaline phosphatase (ALP) or serum bilirubin >3x ULN, or history of hepatic cirrhosis.
- •d. History or presence of impaired renal function as indicated by clinically significant abnormal creatinine, blood urea nitrogen (BUN), or urinary constituents or moderate to severe renal dysfunction as defined by the Cockcroft-Gault equation (<60 mL/min).
- •4) Type 1 diabetes mellitus or current or recent use of insulin (more than 1 week within 3 months prior to Screening).
- •5) Uncontrolled endocrine disorders (e.g., Cushing syndrome, Addison’s, hypothyroidism, hypogonadism), except T2DM with HbA1c<10%.
- •6) Clinically significant and abnormal Screening hematology lab results or recurring infections, or if any of the following are observed (regardless of the Investigator’s assessment of clinical significance):
- •a. Hemoglobin <10 g/dL (<100 g/L)
- •b. Absolute neutrophil count (ANC) <2000/mm^3
- •c. Platelets <135 x 10^9/L
- •7) Results of Screening clinical laboratory tests (complete blood count [CBC] with differential and platelets, chemistry, and urinalysis profile) and ECG outside normal range and considered to be clinically significant by the Investigator.
- •8) Subjects on the following systemic concomitant medications who have not been on stable dose (or stable weight based dose), defined as no more than ±25% variation in dose, for at least 3 months prior to study entry:
- •a. Growth hormone
- •b. Glucocorticoids
- •c. Vasopressin
- •d. Thyroid hormone
- •e. Testosterone
- •f. Other hormone or hormone replacement therapies
- •g. Anti-obesity agents (must have been stable for =6 months)
- •h. Anti-diabetes medications (except for glucagon-like peptide-1 [GLP-1] analogues or sodium-glucose linked transporter 2 (SGLT2) inhibitors must have been stable for =6 months)
- •i. Modafinil
- •j. Atypical anti-psychotics
- •k. Anti-depressive
- •l. Attention deficit hyperactivity disorder (ADHD) medications
- •9) Vital signs unstable, or with the following values:
- •a. Systolic blood pressure >160 mm Hg
- •b. Diastolic blood pressure >100 mm Hg
- •c. Pulse rate >100 beats per minute (bpm)
- •10) Recent (within the last year) and/or recurrent history of autonomic dysfunction (e.g., unexplained syncope or palpitations).
- •11) Current or anticipated chronic use (more than 2 days) of narcotics or opiates.
- •12) Significant history of abuse of drugs or sol
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