NCT02507492Unknown2 期
RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Effect of RM-493 on Body weight
研究概览
简要总结
The purpose of this study is to evaluate the effects of a once daily subcutaneous (SC) injection of RM-493, in subjects with POMC (propiomelanocortin) or other related rare genetic mutations, on body weight, metabolic function and blood pressure. Patients who respond during the initial 84 days of treatment can enter into long-term (2-year) extensions. The study drug (RM-493) will be administered in an unblinded fashion.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent
- •Homozygous or compound heterozygous (different gene mutation on both alleles) POMC, LEPR or PCSK1 gene mutation
- •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 (Arzneimittelgesetz) (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)
- •normal values Alanine-Aminotransferase (ALT) (female): < 31 U/l
- •normal values Alanine-Aminotransferase (ALT) (male): < 41 U/l
- •normal values Aspartate-Aminotransferase (AST) (female > 17 years): < 35 U/l; (female < 17 years): 16- 46 U/l
- •normal values Aspartate-Aminotransferase (AST) (male > 17 years): < 50 U/l; (male < 17 years): 16-46 U/l
- •normal values bilirubins (male and female) up to 1,2 mg/dl
- •normal values Creatinine (female > 15 years): 0,51-0,95 mg/dl) ; (female < 15 years): 0,46-0,77 mg/dl
- •normal values Creatinine (male > 15 years): 0,67 - 1,17 mg/dl) ; (male < 15 years): 0,46-0,77 mg/dl
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 clinical 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 gene disorders
- •Renal insufficiency (Creatinine > 0,95 mg/dl (female), > 1,17 mg/dl (male))
- •Impaired liver function (Bilirubins > 1.2 mg/dl)
- •Neurological / psychiatric diseases
- •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
研究组 & 干预措施
RM-493 Once Daily
Experimental
Dose once daily in the morning
干预措施: RM-493 (Drug)
结局指标
主要结局
Effect of RM-493 on Body weight
时间窗: Day 1 through Day 84
Change of body weight after Treatment with RM-493.
次要结局
- Effect of RM-493 on metabolic serum parameters(Day 1 through Day 84)
- Effect of RM-493 on hunger(Day 1 through Day 84)
- Effect of RM-493 on body composition/Energy expenditure(Day 1 through Day 84)
- Effect of RM-493 on weight loss after treatment continuation of 2 years(Day 1 through Day 730)
- Effect of RM-493 on blood pressure(Day 1 through Day 84)
研究者
PD Dr. Susanna Wiegand
Principal Investigator
Charite University, Berlin, Germany
研究点 (1)
Loading locations...
相似试验
已完成
不适用
Efficacy of proactive therapy by topical corticosteroids for atopic dermatitisatopic dermatitisJPRN-UMIN000005059Department of dermatology, Nihon university Itabashi hospital10
已完成
不适用
Analysis of a Beneficial Effect of Prolonges Topical Steroid Treatment After Low-risk Penetrating KeratoplastyGraft RejectionNCT00450996University of Erlangen-Nürnberg Medical School400
已完成
3 期
Evaluation of AVE5026 as Compared to Placebo for the Extended Prophylaxis of Venous Thromboembolism in Patients Having Undergone Hip Fracture SurgeryVenous ThromboembolismNCT00709904Sanofi469
已完成
4 期
Efficacy of Corticosteroids to Treat Outpatients With Acute Exacerbations of COPDPulmonary DiseaseChronic Obstructive (MeSH)NCT02330952Assistance Publique - Hôpitaux de Paris189
已完成
1 期
Phase 1b/2a Study to Evaluate Safety and Efficacy of Setmelanotide in Obese PatientsOverweight and ObesityNCT02041195Rhythm Pharmaceuticals, Inc.99