EUCTR2015-005761-23-GB进行中（未招募）1 期
A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes
CTD Holdings, Inc.0 个研究点目标入组 12 人开始时间: 2016年8月3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
- 12
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
入选标准
- •1. Confirmed diagnosis of NPC-1 defined as one of the following:
- •a) Two NPC-1 mutations on genotyping
- •b) One NPC-1 mutation and positive filipin staining (current or prior)
- •c) Vertical supranuclear gaze palsy [VSNGP] plus either = one NPC-1 mutation or positive filipin staining and no NPC-2 mutations
- •2. NIH NPC Severity Score <30 and with no more than 4 individual domains with a score = 3.
- •3. Age range: 2 years upwards
- •a) Inclusion of the first three paediatric patients will be restricted to individuals aged = 5 years. Once the first three paediatric patients have safely completed stage 1, study entry will be open to all ages =2 years as per the protocol.
- •4. Negative pregnancy test for females of child bearing potential
- •5. Written, informed consent
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 6
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 6
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. The presence of NPC-2 mutations on genotyping
- •2. Previous receipt of cyclodextrin therapy
- •3. Lanksy score < 50 if aged =16 or Karnofsky score < 40 if aged > 16.
- •4. Inability to comply with the proposed protocol assessments
- •5. Concurrent treatment with any type of cholesterol lowering agents such as statins, fibrates, ezetimibe
- •6. Concurrent medical conditions representing a contraindication to any of the study medications
- •7. Stage 3 chronic kidney disease (CKD) or worse as indicated by an eGFR < 60 mL/min/1.73 m2. In patients aged = 18 years, eGFR is calculated according to the Schwartz equation (ref: Schwartz GJ & Work DF) and in patients aged > 18 years eGFR is calculated using the MDRD equation
- •8. Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or international normalised ratio (INR) >1. 8
- •9. Involvement in another interventional clinical trial within the previous 6 months from screening
- •10. Weight >100 kg
- •11. Females of childbearing potential who are not willing to use a method of highly effective contraception (hormonal contraception, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner, or sexual abstinence) during the study
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