NCT04725240已完成2 期
A Phase 2, Open-Label 20-Week Study to Evaluate the Safety and Efficacy of Setmelanotide in Subjects With Hypothalamic Obesity
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 5
- 主要终点
- Percentage of Participants With ≥ 5% Reduction in BMI From Baseline After 16 Weeks of Setmelanotide Treatment
研究概览
简要总结
Open-label, single-arm study designed to evaluate the body weight response to setmelanotide administered subcutaneously (SC) daily in participants with hypothalamic obesity (HO).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 40 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must meet all of the following criteria to be eligible for study participation:
- •Participant has documented evidence of HO, including:
- •Recent evidence of hypothalamic injury on magnetic resonance imaging (MRI); AND
- •Diagnosis of craniopharyngioma or other non-malignant brain tumor affecting the hypothalamic region; AND
- •Has undergone surgery, or chemotherapy, or radiation ≥6 months and ≤15 years before Screening.
- •Participant has either unilateral hypothalamic lesions or bilateral hypothalamic lesions, as assessed by MRI.
- •Obesity, documented by a body mass index (BMI) ≥35 kilograms/square meter (kg/m^2) for participants ≥18 years of age or BMI ≥95th percentile for age and gender for participants 6 to <18 years of age.
- •Documented increase in BMI (change from pre-surgery baseline in BMI z-score ≥0.2 for participants <18 years of age or BMI >5% for participants >18 years of age) either during the first 6 months following surgery or within 1 year before surgery AND still present at Screening.
- •More than 6 months after the end of post-tumor treatment, including chemotherapy, surgery, or radiation.
- •Highly effective contraception throughout the study and for 90 days following the study.
- •Ability to communicate well with the Investigator, understand and comply with the requirements of the study, and understand and sign the written informed consent, or, for participants aged <18 years, a parent/legal guardian that can sign.
- •If receiving hormone replacement therapy, the dose of such therapy has remained stable for at least 2 months prior to Screening.
排除标准
- •Participants meeting any of the following criteria are not eligible for study participation:
- •Weight gain >5% in the previous 3 months.
- •Weight loss ≥2% in the previous 3 months.
- •Bariatric surgery or procedure within the last 6 months.
- •Diagnosis of severe psychiatric disorders
- •Glycated hemoglobin (HbA1c) >10.0% at Screening.
- •Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results.
- •Glomerular filtration rate (GFR) <30mL/min/1.73m^2 during Screening.
- •Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.
- •History or close family history (parents or siblings) of skin cancer or melanoma
- •Participation in any clinical study with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first setmelanotide dose.
- •Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
- •Inability to comply with QD injection regimen.
- •Pregnant and/or breastfeeding, or desiring to become pregnant during this trial.
- •Cognitive impairment that, in the Investigator's opinion, precludes participation to the study and completions of study procedures or questionnaires.
- •Participant is, in Investigator's opinion, otherwise not suitable to participate in the study.
研究组 & 干预措施
Setmelanotide
Experimental
Participants received setmelanotide once daily (QD) via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 milligrams (mg) QD.
干预措施: Setmelanotide (Drug)
结局指标
主要结局
Percentage of Participants With ≥ 5% Reduction in BMI From Baseline After 16 Weeks of Setmelanotide Treatment
时间窗: Baseline to 16 weeks
BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m\^2. Baseline was defined as the most recent measurement prior to the first administration of study drug.
次要结局
- Percentage of Participants Aged ≥18 Years With ≥5% Reduction of Body Weight From Baseline After 16 Weeks of Setmelanotide Treatment(Baseline to Week 16)
- Change From Baseline in Daily Hunger Questionnaire Score in Participants Aged <12 Years After 16 Weeks of Setmelanotide Treatment(Baseline, Week 16)
- Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years(Baseline, Week 16)
- Change From Baseline in Waist Circumference in Participants Aged ≥18 Years After 16 Weeks of Setmelanotide Treatment(Baseline, Week 16)
- Composite Percentage of Participants Aged ≥6 to <18 Years With ≥0.2 Reduction of BMI Z-Score or Participants Aged ≥18 Years With 5% Reduction of Body Weight From Baseline After 16 Weeks of Setmelanotide Treatment(Baseline to Week 16)
- Percentage of Participants Aged ≥6 to <18 Years With ≥0.2 Reduction of BMI Z-Score From Baseline After 16 Weeks of Setmelanotide Treatment(Baseline to 16 weeks)
- Change From Baseline in Daily Hunger Questionnaire Scores in Participants Aged ≥12 Years After 16 Weeks of Setmelanotide Treatment(Baseline, Week 16)
- Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years(Baseline, Week 16)
- Number of Participants With Adverse Events (AEs)(From first dose of study drug up to Day 141)
研究者
研究点 (5)
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