An Open Label, Phase 2a Clinical Trial for the Evaluation of Safety and Efficacy of RZL-012 for the Treatment of Women With Lipedema Involving Substantial Fat Above the Knee or of Women and Men With Nodular Dercum's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- The Incidence of Any Grade 3 or Greater Event Intolerable Side Effect Experienced in a Cohort
研究概览
简要总结
This is an open label, 2 cohort, clinical trial in women with lipedema with substantial fat above the knee and men with Dercum's disease. Each cohort will have 6 subjects who will receive RZL-012.
详细描述
This is an open label, 2 cohort, clinical trial in women with lipedema with substantial fat above the knee and men with Dercum's disease.
- The 1st cohort will be comprised of subjects with Dercum's disease.
- The 2nd cohort will be comprised of subjects with lipedema with substantial fat above the knee
Within each cohort dosing of the subjects will progress consecutively from one individual to the other with a minimum 7 days between subjects to asses safety. The study design will allow the physicians to monitor safety for at least 7 days prior to dosing the next subject. Cohort 2 will be conducted in a dose escalation manner and the decision to proceed to the next dose level will be made after reviewing all safety data collected by Day 14 within 2+/-1 day of the last dosed subject. The trial will proceed within a cohort provided that no more than one subject experience intolerable side effects in a cohort, and based on the decision made by the Principal Investigator (PI) and the medical monitor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 20 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Post-menopausal (at least 2 years) women no more than 65 years old, with lipedema involving substantial fat above the knee or nodular Dercum's disease in such women and in men 20 - 65 years with nodular Dercum's disease.
- •For Dercum's disease subjects - at least 2 nodules to be injected of at least 2cm diameter each, as determined by ultrasound
- •For lipedema subjects - Significant subcutaneous fat above the knee as determined by circumference of 50cm
- •Generally considered healthy according to medical history, physical examination, ECG and laboratory evaluation with a special emphasis on metabolic parameters (fasting glucose concentration < 100 mg, normal blood pressure).
- •Subjects must be able to adhere to the visit schedule and protocol requirements and be available to complete the study.
- •Subjects must sign an informed consent indicating that they are aware of the investigational nature of the study.
排除标准
- •Unable to tolerate subcutaneous injection.
- •Subjects with uncontrolled cardiac, hepatic, renal or neurologic/psychiatric disorders, that in the opinion of the investigator, put the subject at significant risk, are not eligible.
- •Positive blood screen for Hepatitis B surface antigen (HbSAg), Hepatitis C virus (HCV), or Human immunodeficiency virus (HIV), or positive urine screen for alcohol or drugs of abuse (unless prescribed by a physician).
- •Subjects with a clinical history of primary or secondary immunodeficiency, autoimmune disease or subjects taking immunosuppressive drugs such as corticosteroids are ineligible.
- •As a result of medical review, physical examination, the PI (or medically qualified nominee) considers the subject unfit for the study.
- •Known sensitivity to components of the injection formulation.
- •Prior wound, tattoo or infection in the treated area.
- •Prior invasive treatment such as surgery or injectable drug at the RZL-012 injected area.
研究组 & 干预措施
Cohort 1 - Dercum's Disease
Nodular size - diameter (cm) 2-2.9, 3-3.9, 4-8 Total dose of RZL-012(mg) 10 15 20 Dose per NOAEL 1/25th , 1/18.75th, 1/12.5th Number of injections: 2,3,4
干预措施: RZL-012 (Drug)
Cohort 2 - Lipedema
Total dose of RZL-012 (mg) 60 , 80 Dose per NOAEL: 1/4.6888, 1/3.125 Number of injections: 12, 16
干预措施: RZL-012 (Drug)
结局指标
主要结局
The Incidence of Any Grade 3 or Greater Event Intolerable Side Effect Experienced in a Cohort
时间窗: 0-14 days
Trial will proceed as long as no more than one subject experiences an intolerable side effect in a cohort
次要结局
未报告次要终点