EUCTR2021-004597-65-DE进行中（未招募）1 期
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) with a Crossover to Open-Label Once Weekly Setmelanotide in Patients with Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation - Setmelanotide - Daily vs weekly with crossover to open label
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
- 30
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
- 性别
- All
入选标准
- •1. All patients must have met the criteria for diagnosis of a gene defect in the MC4R pathway (BBS, biallelic PPL, heterozygous PPL), for which they are being treated with QD setmelanotide.
- •2. Patients must be =6 years old at screening.
- •3. Patients must have been taking the setmelanotide QD formulation for at least 6 months in the LTE trial with acceptable safety and tolerability, and the dose level must have been stable at 2, 2.5 or 3 mg of setmelanotide for at least the last 3 months prior to starting the Run-in Period.
- •4. Patient and/or parent or guardian is able to communicate well with the Investigator, to understand and comply with the requirements of the trial and is able to understand and sign the written informed consent/assent.
- •5. Patient must meet one of the following requirements:
- •Female participants of childbearing potential, defined as fertile, following menarche and until becoming post-menopausal unless permanently sterile (hysterectomy, bilateral salpingectomy, or bilateral oophorectomy), must be confirmed non-pregnant and agree to use a highly effective form of contraception throughout the trial and for 90 days following the trial. Highly effective forms of contraception are detailed below and in Section 8.8.7:
- •- Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal)
- •- Progestin-only hormonal contraception associated with inhibition of ovulation (oral, implantable, or injectable)
- •- Intrauterine device (IUD)
- •- Intrauterine hormone-releasing system
- •- Bilateral tubal occlusion
- •- Vasectomy/vasectomized partner (provided that the vasectomized partner is the sole sexual partner of the female participant, and the vasectomized partner has received medical assessment of surgical success)
- •- Sexual abstinence, only if it is the preferred and usual lifestyle of the patient
- •Female participants of non-childbearing potential, defined as: permanently sterile (status post hysterectomy, bilateral oophorectomy, or bilateral salpingectomy), or post-menopausal for at least 12 months (and confirmed with a screening follicle-stimulating hormone (FSH) level in the post-menopausal lab range) do not require contraception during the trial .
- •Younger female patients who have not achieved sexual maturity at trial entry will be assessed for Tanner staging and required to comply with contraception requirements at first menarche.
- •Male participants with female partners of childbearing potential must agree to use a highly effective method of contraception if they become sexually active during the trial or within 90 days following their participation in the trial. Male patients must also not donate sperm during and for 90 days following their participation in the trial.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 20
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.HbA1C >9.0% at screening.
- •2.Has taken a medication that is approved to treat obesity (e.g., orlistat, lorcaserin, phentermine-topiramate, naltrexone-bupropion) within 3 months prior to starting the Run-in Period. Glucagon-like peptide-1 (GLP) -1) receptor agonists being prescribed for the treatment of obesity are not allowed.
- •3.History of significant liver disease or liver injury, or a current liver assessment due to abnormal liver tests for an etiology other than nonalcoholic fatty liver disease (NAFLD). Thus, any underlying etiology besides NAFLD, including diagnosed nonalcoholic steatohepatitis (NASH), other causes of hepatitis, or history of hepatic cirrhosis is exclusionary, but the presence of NAFLD is not exclusionary.
- •4.Moderate to severe renal dysfunction as defined by a glomerular filtration rate <30 mL/min. (based upon the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] creatinine equation 2021 from the National Kidney Foundation). In patients <18 years of age the Bedside Schwartz Equation should be used to calculate estimated glomerular filtration rate (eGFR).
- •5.Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion), determined as part of comprehensive skin evaluation performed by the Investigator during screening. Any concerning lesions identified during the Screening Period will be biopsied and results must be known to be benign prior to enrollment. If the pretreatment biopsy results are of concern, the patient should be excluded from the trial.
- •6.Diagnosis of schizophrenia, bipolar disorder, personality disorder, or other psychiatric disorders that the Investigator believes will interfere significantly with trial compliance. Neurocognitive disorders affecting ability to consent will not be disqualifying as long as an appropriate guardian able to give consent has been appointed.
- •7.Clinically significant depression or suicidality as defined by: any suicidal ideation of type 4 or 5 on the C SSRS, any lifetime history of a suicide attempt, or any suicidal behavior in the last month or a Patient Health Questionnaire-9 (PHQ-9) score of =15 during Screening in patients with no significant neurocognitive deficits.
- •8.Patient is not suitable, in the opinion of the Investigator, to participate in the trial.
- •9.Hypersensitivity to the active substance or to any of the excipients of the investigational products (active or placebo).
- •10.Inability to comply with the QW and QD injection regimens.
- •11.Participation in any clinical trial with an investigational drug/device within 3 months prior to the first day of dosing, with the exception of a setmelanotide clinical trial.
- •12. Legally protected persons per local regulations (e.g., those that fall under the L1121-6 article of the Public Health code in France) or other applicable local laws.
- •13. The patient or a relative of the patient is the investigator or a sub-investigator, research assistant, pharmacist, trial coordinator, or other staff directly involved with the conduct of the trial.
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