CTIS2022-501781-22-00进行中（未招募）1 期
Open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia - APL-20
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 25
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 17（—）
- 性别
- All
入选标准
- •Male and female subjects aged < 6 years of age at the time of LAR signing the informed consent form (ICF) prior to initiation of any study specific activities/procedures and < 6 years of age at Enrolment/ Baseline (Visit 2)., Metreleptin treatment naive, Confirmed diagnosis of generalised LD as demonstrated by one of the following criteria (a or b or c): a. Documented genetic diagnosis of generalised LD (mutations in genes known to be associated with congenital generalised LD) OR b. Imaging (e.g., dual-energy x-ray absorptiometry, whole body magnetic resonance imaging) documenting near total fat loss OR c. Clinical diagnosis of generalised LD supported by low leptin levels for age/gender and evidence of insulin resistance (e.g., fasting insulin > 30 mcU/ml, acanthosis nigricans, metabolic abnormalities due to insulin resistance). Clinical diagnoses need to be reviewed by the medical monitor., Confirmation by the Investigator that the potential differential diagnosis of generalised LD has been excluded (e.g., auto-inflammatory syndromes, progeroid syndromes, SHORT syndrome, malnutrition/starvation, anorexia nervosa, cachexia, HIV-associated wasting, diencephalic syndrome, thyrotoxicosis, adrenocortical insufficiency, severe chronic inflammation, poorly controlled type 1 diabetes mellitus)., Elevated HbA1c =6.5% and/or fasting triglycerides =2.3 mmol/L (200 mg/dL)., Subjects with diabetes should be receiving stable treatment regimen (diet and/or antidiabetic treatment) for at least 90 days prior to Screening (Visit 1) and during the screening period. If a subject is on insulin, a stable dose is defined as no more than a 20% fluctuation in insulin dose. Diet (as reported by the subject/LAR/caregiver) should be stable and in line with medical recommendations., Subjects with elevated fasting TG levels should be receiving stable treatment regimen (diet and/or lipid lowering therapy) for at least 6 weeks prior to Screening (Visit 1) and during the screening period. Diet (as reported by the subject/LAR/caregiver) should be stable and in line with medical recommendations., LAR capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. Subject assent to be obtained per local requirements., Subject with stable mental and physical health per Investigator’s opinion.
排除标准
- •Weight <9 kg at Screening (Visit 1), Any clinically significant uncontrolled medical condition, that in the Investigator’s opinion would jeopardise subject participation or the interpretation of study results., Undergone major surgery/surgical therapy for any cause within 1 month prior to Screening (Visit 1) or planned procedure during the study., Treatment with any investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half-life of the corresponding IMP, whichever is longer, prior to Screening (Visit 1)., Known allergy or hypersensitivity to any of the investigational product or materials., ALT or AST >8 x ULN. If a subject has AST or ALT between 3-8 x ULN, the Investigator should discuss the case with the Sponsor to determine whether it is in the interest of the subject to be enrolled., Symptomatic biliary obstruction or hyperbilirubinemia (i.e., total bilirubin >2x ULN, except with a documented diagnosis of Gilbert’s disease)., Chronic renal insufficiency with glomerular filtration rate (GFR) <60 mL/min calculated using the Schwartz Formula, Known active Hepatitis B, Hepatitis C or autoimmune hepatitis. Note: No testing for Hepatitis B and Hepatitis C is required unless mandated by local health authority., In the judgement of the Investigator, precocious puberty or endocrine disorder that would affect growth (e.g., uncontrolled hypothyroidism, premature adrenarche)., History of malignancy (except for treated basal cell or squamous cell skin cancer) occurring within the past 3 years., Subjects with ongoing or recent (within last 3 months) episode of acute pancreatitis., Diagnosis of clinically significant hematological abnormalities (including but not limited to clinically significant leukopenia, neutropenia, bone marrow abnormalities, leukemia or lymphoma, or clinically significant pathological lymphadenopathy).
研究者
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