EUCTR2021-002855-12-FR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 inPatients with Specific Gene Defects in the Melanocortin-4 ReceptorPathway - Setmelanotide in patients with specific gene defects
适应症
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
- •3. Obese, defined as BMI =40 kg/m2 for patients =18 years of age or BMI =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 0
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
- •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.
研究者
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