CTIS2022-502950-14-00招募中1 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 - APL-22
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 14
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 年龄范围
- 0 至 65+（—）
- 性别
- All
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
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.
研究者
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