EUCTR2021-002855-12-ES进行中（未招募）1 期
A 2-Stage (Open-Label Run-in followed by Randomized Withdrawal), Double-Blind, Placebo-Controlled, Phase 2 study of Setmelanotide in Patients with Specific Gene Defects in the Melanocortin-4 Receptor Pathway - Setmelanotide in patients with specific gene defects
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 500
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
- 性别
- All
入选标准
- •1. Patients must have a pre-identified genetic variant in an established MC4R pathway gene that contributes to obesity. For a gene variant to be eligible for inclusion in the study, the variant must be categorized by a CLIA/CAP/ISO15189 certified laboratory using American College of Medical Genetics (ACMG) criteria as (1) Pathogenic, (2) Likely Pathogenic, or (3) a Variant of Uncertain Significance (VUS). In the case where an investigator has genetic results on a patient who may be eligible for the study, but the genetics have not yet been categorized by a CLIA/CAP/ISO15189 certified laboratory, then the Sponsor may provide testing and/or categorization through a third-party laboratory.
- •2. Patients between the ages of 6 and 65, inclusive, at the time of signing Informed Consent or Assent are eligible for the study.
- •3. Obese, defined as BMI> or = 40 kg/m2 for patients > or =18 years of age or BMI > or =97th percentile for age and gender for patients 6 up to <18 years of age based on the United States (US) Centers for Disease Control and Prevention criteria.
- •4. Study participant and/or parent or guardian is able to communicate well with the Investigator, to understand and comply with the requirements of the study (including QD injection regimen and all other study procedures) and is able to understand and sign the written informed consent/assent. Patients who are unable to comply with all study procedures due to cognitive limitations or any other reason should not be enrolled into the study.
- •5. If male or a childbearing female, including pre-pubertal females if relevant, agrees to use a highly reliable form of contraception throughout the study and for 90 days following the study. Highly reliable acceptable forms of contraception include hormonal (i.e., oral, implantable, or injectable) AND singlebarrier method (i.e., condom), or an intrauterine device (IUD) AND single-barrier method (i.e., condom) or vasectomy/vasectomized partner. True abstinence is acceptable only if it is the preferred and usual lifestyle of the patient. Female participants of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation), post-menopausal for at least 12 months (and confirmed with a screening follicle-stimulating hormone [FSH] level in the post-menopausal lab range), and failure to have achieved menarche, do not require contraception during the study. Female patients must not become pregnant and male patients must not donate sperm during and for 90 days following their participation in the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 175
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 325
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Recent intensive (within 2 months) diet and/or exercise regimen with or without the use of weight loss agents including herbal medications that has resulted in >3% weight loss.
- •2. Use of any medication that is approved to treat obesity within 3 months of first dose of study drug (e.g., orlistat, lorcaserin, phentermine-topiramate, naltrexone-bupropion).
- •3. Bariatric surgery within the previous 6 months.
- •4. Diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive disorder, or other psychiatric disorder(s) that the Investigator believes will interfere significantly with study compliance.
- •5. Any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) during Screening, any suicide attempt in the past 20 years or any suicidal behavior in the last month.
- •6. Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results. Any patient with a potentially clinically significant disease should be reviewed
- •with the Sponsor to determine eligibility.
- •7. Has significant features of (or meets the diagnostic criteria for) a genetic syndrome that is associated with obesity, such as Tatton-Brown-Rahman syndrome (DNMT3A), Rett Syndrome (MECP2), Chung-Jansen syndrome (PHIP), Schaaf-Yang syndrome (MAGEL2), ulnar mammary syndrome (TBX3), or Rubinstein-Taybi syndrome (CREBBP). Note: Although some of the genetic variants that are eligible to be enrolled into this study are associated with specific syndromes, the intent of this study is not to enroll children with significant cognitive impairment or other significant comorbidities. Patients with the correct genetic variants, but who otherwise do not exhibit the syndrome, are eligible for enrollment.
- •8. Glycated hemoglobin (HbA1C) >10.0% at Screening.
- •9. History of significant liver disease other than non-alcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH).
- •10. Glomerular filtration rate (GFR) <60 mL/min at Screening.
- •11. History or close family history (parents or siblings) of melanoma, or patient history of oculocutaneous albinism. Note: If the type of skin cancer is not known, then the patient should not be enrolled into the study.
- •12. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion), determined as part of a comprehensive skin evaluation performed by the Investigator during Screening. Any concerning lesions identified during Screening will be biopsied and results known to be benign prior to enrollment. If the pretreatment biopsy results are of concern, the patient may need to be excluded from the study.
- •13. Patient is, in the opinion of the Investigator, not suitable to participate in the study.
- •14. Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.
- •15. Patients previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
- •16. Significant hypersensitivity to any excipient in the study drug.
- •17. Females who are breastfeeding or nursing.
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