NCT03262610已完成2 期
Expanded-access for the Use of Setmelanotide in a Single Patient With Partial Lipodystrophy (LD) Associated With Leptin Deficiency and Multiple Autoimmune Diseases
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
- 1
- 试验地点
- 1
- 主要终点
- Fasting Triglycerides (TG) levels
研究概览
简要总结
The objective of this single patient study is to assess the safety and efficacy of setmelanotide as a treatment of severe metabolic abnormalities resulting from LD, especially refractory hypertriglyceridemia leading to recurrent bouts of pancreatitis.
详细描述
This is a single-patient study to assess the safety and efficacy of setmelanotide in a unique patient with partial LD and severe metabolic abnormalities, most notably refractory hypertriglyceridemia leading to recurrent pancreatitis despite ongoing plasmapheresis treatment.
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
- 15 Years 至 15 Years（Child）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Signed written informed consent
- •Ability to comply with visits and procedures required by program.
- •Has physician-confirmed partial lipodystrophy and the following characteristics:
- •Has atypical lipodystrophy.
- •Has presence of neutralizing antibody to metreleptin
- •Patient has life threatening hypertriglyceridemia and has had >8 episodes of pancreatitis, requiring weekly plasmapheresis
- •Has Type 1 diabetes mellitus with HbA1c > 10%.
- •Female, under the age of 18 years
排除标准
- •Diagnosis of schizophrenia, bipolar disorder, personality disorder or other Diagnostic and Statistical Manual of Mental Disorders (DSM-III) disorders that the investigator believes will interfere significantly with study compliance. Neurocognitive disorders affecting ability to consent will not be disqualifying as long as an appropriate guardian able to give consent has been appointed.
- •History or close family history (parents or siblings) of skin cancer or melanoma, or patient history of ocular-cutaneous albinism.
- •Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions, determined as part of a screening comprehensive skin evaluation performed by a qualified dermatologist.
- •Significant hypersensitivity to study drug.
- •Inability to comply with QD injection regimen.
研究组 & 干预措施
Setmelanotide daily subcutaneous injection
Experimental
Up to 18 weeks setmelanotide treatment.
干预措施: Setmelanotide (Drug)
结局指标
主要结局
Fasting Triglycerides (TG) levels
时间窗: 12 to 20 weeks
The mean change from baseline in fasting triglycerides after setmelanotide treatment.
次要结局
未报告次要终点
研究者
研究点 (1)
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