A 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily SC Metreleptin Treatment in Patients With Generalized Lipodystrophy
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 11
- 试验地点
- 8
- 主要终点
- Evaluate the Immunogenicity Associated With Daily Subcutaneous (SC) Metreleptin Treatment in Patients With Congenital Generalized Lipodystrophy (CGL) or Acquired Generalized Lipodystrophy (AGL).
研究概览
简要总结
MYALEPT™ (metreleptin) has been approved as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (MYALEPT Prescribing Information). This study is a multicenter, open-label, Phase 4 trial to provide an assessment of the immunogenicity associated with metreleptin and of any major potential risks due to development of antibodies to metreleptin. The study is being conducted to comply with a postmarketing requirement.
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
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provision of informed consent prior to any study specific procedures. If <18 years of age, has a parent or guardian able to read, understand, and sign the Informed Consent Form (ICF) and a Child Assent form, communicate with the Investigator, and understand and comply with protocol requirements. Adolescent patients must also read and understand the Child Assent Form. If the child is too young or unable to read, then the Child Assent form must be explained to the child.
- •Female and/or male patients ≥1 years of age.
- •Physician-confirmed diagnosis of congenital or acquired generalized lipodystrophy and will begin treatment with MYALEPT for the first time.
- •Negative pregnancy test (urine or serum) for female patients of childbearing potential.
- •Female patients of childbearing potential must be 1 year postmenopausal, surgically sterile, or be willing to use an acceptable method of contraception (an acceptable method of contraception is defined as a barrier method in conjunction with a spermicide) for the duration of the study (from the time they sign consent). In addition, oral contraceptives, approved contraceptive implant, long-term injectable contraception, intrauterine device, or tubal ligation are allowed. Oral contraception alone is not acceptable; additional barrier methods in conjunction with spermicide must be used.
- •Male patients must be surgically sterile or be willing to use an acceptable method of contraception (defined as barrier methods in conjunction with spermicides) for the duration of the study (from the time they sign consent).
- •Patients who are blood donors should not donate blood during the study and for 3 months following their last dose of metreleptin.
排除标准
- •Involvement in the planning and/or conduct of the study (applies to both Aegerion staff and/or staff at the study site.)
- •Previous treatment with metreleptin.
- •Participation in another clinical study with an investigational product during the last 6 months.
- •Patients with prior severe hypersensitivity reactions to metreleptin or to any of the product components.
- •Known to have tested positive for human immunodeficiency virus, are immunocompromised, or are receiving immunomodulatory drugs.
- •Known history of drug or alcohol abuse within 1 year of screening.
- •Creatinine clearance <30 mL/min using institutional standards:
- •e.g., calculated using Cockcroft-Gault formula for patients ≥18 years of age; calculated using Schwartz equation for patients <18 years of age.
- •For women only - currently pregnant (confirmed with positive pregnancy test) or breast-feeding.
- •Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the patient or could render the patient unable to successfully complete the study.
研究组 & 干预措施
Metreleptin
Subjects will receive prescribed dosage of metreleptin as indicated in the USPI Patients (males and females) ≤ 40 kg: 0.06mg/kg Male patients > 40 kg: 2.5mg Female patients > 40 kg: 5mg
干预措施: Metreleptin (Drug)
结局指标
主要结局
Evaluate the Immunogenicity Associated With Daily Subcutaneous (SC) Metreleptin Treatment in Patients With Congenital Generalized Lipodystrophy (CGL) or Acquired Generalized Lipodystrophy (AGL).
时间窗: 36 months
Anti-metreleptin, anti-human leptin (HuL) binding antidrug antibody (ADA) titers over time. Category of in vitro cell-based neutralizing antibody (NAb) activity to metreleptin, and titer in the receptor blocking (RB) NAb assay in ADA positive samples over time.
次要结局
- Assess 2 Methods of Measuring in Vitro NAb Activity to Metreleptin.(36 months)
- Evaluate the Safety and Tolerability in Relation to the Development of or Absence of Anti-metreleptin and Anti-HuL Binding ADAs, and/or in Vitro NAb Activity to Metreleptin in Patients With CGL or AGL(36 months)