2022-502950-14-00招募中4 期
A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients with Partial Lipodystrophy
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 11
- 试验地点
- 13
- 主要终点
- Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%.
研究概览
简要总结
To evaluate the efficacy of metreleptin treatment in patients with PL.
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Follow-up period
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 65+ years（18-64 Years, 65+ Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Male and female patients aged ≥12 years of age at the time of signing the ICF and Child Assent Form (if applicable) prior to initiation of any study specific activities/procedures.
- •Patient and/or his/her legal representative has/have been informed, has/have read and understood the patient ICF and Child Assent Form (if applicable), has/have given written informed consent and is/are willing to comply with the protocol requirements. If the child is too young or unable to read, then the Child Assent Form must be explained to the child.
- •Confirmed diagnosis of familial or acquired PL.
- •Confirmation by the Investigator that the potential differential diagnosis of LD has been excluded (e.g., Cushing’s syndrome, anorexia nervosa, cachexia, diencephalic syndrome, Rabson-Mendenhall syndrome, leprechaunism, SHORT syndrome, mandibuloacral dysplasia, progeroid syndromes, localised scleroderma, lichen sclerosus et atrophicus, annular lipodystrophy, semi-circular lipoatrophy, local panniculitis due to connective tissue diseases and autoimmune disorders, intradermal or subcutaneous related lipoatrophy [e.g., insulin, acupuncture, recombinant growth hormone], progressive hemifacial atrophy [Parry- Romberg syndrome])
- •Patients must have: a) HbA1c level ≥6.5% and/or b) Fasting serum TG levels ≥500 mg/dL (5.65 mmol/L)
- •Standard treatments have failed to achieve adequate metabolic control: a) Patients with HbA1c level ≥6.5% must be on stable dose of anti-diabetic therapy for at least 90 days prior to screening (diet and/or antidiabetic medications) and their diet (as reported by the patients) should be stable and in line with medical recommendations. b) Patients with fasting serum TG levels ≥500 mg/dL (5.65 mmol/L) must be on stable dose of lipid-lowering agents for at least 6 weeks prior to screening (unless these medications were not tolerated or are contra-indicated) and their diet (as reported by the patients) should be stable and in line with medical recommendations.
- •Patients receiving antidiabetic and/or lipid-lowering therapy prior to the beginning of the study must be kept stable on optimised treatment based on the Investigator’s judgement and stable during the screening period. For patients on insulin, a stable dose is defined as no more than 20% change in total daily insulin dose. For all other therapies, a stable dose is defined as no dose change.
- •Patients are willing to follow the dietary restrictions recommended by the Investigator.
- •Metreleptin naïve and planned to receive commercial supply of metreleptin or currently treated with commercially supplied metreleptin. Patients currently treated with metreleptin can only be enrolled subject to medical monitor approval and should meet all the following criteria: a) Initiated treatment within 6 months prior to Screening b) Have retained serum samples taken prior to initiation of metreleptin, that can be used to evaluate leptin levels and immunogenicity c) Have documented HbA1c and TG levels prior to initiation of metreleptin (unless these can be assessed based on the retained serum samples).
- •Females of childbearing potential must be postmenopausal (defined as cessation of menses for at least 1 year), surgically sterile (hysterectomy, bilateral oophorectomy, or tubal ligation), or willing to use an effective method of contraception (such methods include combined [estrogen and progestogen containing] hormonal contraception: oral / intravaginal; transdermal / progestogen-only hormonal contraception: oral / injectable; implantable / intrauterine device [IUD] / intrauterine hormone-releasing system [IUS] / bilateral tubal occlusion / vasectomised partner/ sexual abstinence / condoms) for the duration of the study (from the time they sign an ICF and Child Assent Form (if applicable), until 4 weeks after the last dose of study drug). Hormonal contraception alone, including oral, injectable, transdermal, and implantable forms, is not acceptable; an additional barrier method must be used. Intravaginal hormonal contraception or IUS alone are allowed per the Investigator’s discretion. Patients on oral contraceptives will not be required to discontinue medication. Female patients will not be permitted to commence oral contraceptives while taking study treatment during the study.
排除标准
- •Known to have tested positive for human immunodeficiency virus (HIV) or known to be diagnosed with HIV-related LD
- •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 (e.g., life expectancy <12 months).
- •Known history of severe hypersensitivity reactions to any of the metreleptin product components.
- •Known history of drug or alcohol abuse within 1 year prior to Screening as assessed according to the Investigator’s judgment.
- •Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m2 calculated by Bedside Schwartz for patients <18 years and CKD-EPI for patients ≥18 years.
- •Patients whose anti-diabetic/lipid-lowering therapies and/or other concomitant medications that may affect the primary endpoint results (such as appetite suppressing medications) are not stable at Screening.
- •Treatment with any Investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half-life of the corresponding IMP, whichever is longer, before the screening visit.
- •For females only - currently pregnant (confirmed with positive pregnancy test) or breastfeeding.
- •Positive pregnancy test (urine or serum) for females of childbearing potential.
结局指标
主要结局
Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%.
Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%.
Number of patients with decrease of at least 30% in triglycerides (TG) at Month 12 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L).
Number of patients with decrease of at least 30% in triglycerides (TG) at Month 12 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L).
次要结局
- Number of patients with decrease of at least 0.5% in HbA1c at Month 24 compared to Baseline or HbA1c <6.5 % at Month 24, in patients with baseline HbA1c ≥6.5%.
- Number of patients with decrease of at least 30% in TG levels at Month 24 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L).
- Change from baseline in HbA1c at Month 12 and Month 24 in patients with baseline HbA1c ≥6.5% (Subgroup analysis for patients with HbA1c ≥8% will be conducted)
- Percent change from baseline in TG levels at Month 12 and Month 24 in patients with baseline TG ≥500 mg/dL (5.65 mmol/L).
- Number of patients achieving at least 1 of the below criteria: o 0.5% decrease in HbA1c at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12 o 30% decrease in fasting serum TG at Month 12
- Change from baseline in liver volume at Month 12 and Month 24.
- Change from baseline in Homeostatic Model Assessment of Insulin Resistance (HOMAIR) (fasting glucose and insulin) in noninsulin treated patients at Month 12 and Month 24.
- Number of patients who maintained, decreased, or increased dose and/or dosing frequency of anti-diabetic medications.
- Number of patients who maintained, decreased, or increased dose and/or dosing frequency of lipid-lowering medications.
- Number of patients with ≥25% reduction in insulin requirements at Month 12 and Month 24.
研究者
Shir Fuchs Orenbach
Scientific
Amryt Pharmaceuticals Designated Activity Company
研究点 (13)
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