EUCTR2014-002392-28-DE进行中（未招募）1 期
Setmelanotide (RM-493) Treatment Trial in Patients with rare genetic disorders of obesity - RM-493-Supplementation-Therapy
Charité Univeritaetsmedizin Berlin0 个研究点目标入组 30 人开始时间: 2014年8月4日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 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 clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Written informed consent by the patients or the responsible relatives
- •Rare genetic disease populations in adults (=18 years):
- •o Homozygous or compound heterozygous (different gene mutation on both alleles) POMC, LEPR, MC4R or PCSK1 gene mutation
- •o Heterozygous POMC, MC4R mutations
- •o POMC hypermethylation (epigenetic) variant (>51.92 % POMC methylation intensity at the specific analysed POMC region)
- •o Bardet-Biedl Syndrome
- •o Alstrom’s Syndrome
- •Non-adult adolescent patients (= 12 years of age)
- •o Homozygous or compound heterozygous POMC, LEPR or MC4R gene mutations
- •o POMC hypermethylation (epigenetic) variant (> 35.79% POMC methylation intensity for individuals younger than 30 years at the specific analysed POMC region)
- •o As substantial efficacy is shown in adult patients within each rare genetic disorder, then adolescent patients (greater than or equal to 12 years of age) can enter the study.
- •Obesity (BMI > 30 kg/m2; + 2 BMI SDS)
- •No other therapeutic option, which might cure the patient (e.g. bariatric surgery (see chapter 8))
- •Negative Pregnancy test
- •Highly effective contraception in women (defined as pearl index < 1), if necessary also for partners of test persons)
- •No participation in other clinical trials according to AMG (2 months before and after) at the time of this trial
- •Normal or minimally elevated blood pressure (measured in 24RR monitoring or similar methods) according the guidelines of the ESH (European Society of Hypertension) and Deutsche Hochdruckliga: systolic > 159 mmHg/diastolic 99 mmHg
- •sufficient kidney and liver function (Creatinine, ALT, AST)
- •o normal values Alanin-Aminotransferase (ALT) (female): < 31 U/l
- •o normal values Alanin-Aminotransferase (ALT) (male): < 41 U/l
- •o normal values Aspartat-Aminotransferase (AST) (female > 17 years): < 35 U/l; (female < 17 years): 16-46 U/l
- •o normal values Aspartat-Aminotransferase (AST) (male > 17 years): < 50 U/l; (male < 17 years): 16-46 U/l
- •o normal values Bilirubine (male and female) up to 1,2 mg/dl
- •o normal values Creatinine (female > 15 years): 0,51-0,95 mg/dl) ; (female < 15 years): 0,46-0,77 mg/dl
- •o normal values Creatinine (male > 15 years): 0,67 – 1,17 mg/dl) ; (male < 15 years): 0,46-0,77 mg/dl
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 2
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Pregnancy or Breastfeeding
- •All contraindications against study medication (including auxiliary substances)
- •Interactions with study medication
- •Participation of the patient in a clincial study within the last 2 months
- •Intolerance against albumin
- •Concomitant diseases, impaired organ functions, except for known, concurrent GI disorders or other clinical findings expected in PCSK1 or LEPR or MC4R gene disorders
- •Renal insufficiency (Creatinine > 0.95 mg/dl (female), > 1.17 mg/dl (male))
- •Impaired liver function (Bilirubine > 1.2 mg/dl)
- •history of Neurological / psychiatric diseases
- •history of HIV Infection
- •history of Active Hepatitis B or C
- •Melanoma or Melanoma occurrence in the family history
- •Non-compliance
- •Subjects who are legally detained in an official institution
- •HIV Infection
- •Active Hepatitis B or C
- •Melanoma or Melanoma occurrence in the family history
- •Non-compliance
- •Subjects who are legally detained in an official institution
研究者
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