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 - A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Nieman

CTD HOLDINGS, INC.0 个研究点目标入组 12 人开始时间: 2021年9月7日最近更新: 2021年10月8日

适应症

试验速览

暂无简介。

•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 six patients will be restricted to individuals aged = 5 years. In the event that three patients aged >18 (i.e. adults) are recruited before the total recruitment has reached n=6, no more adults will be randomised to treatment until the first cohort of 6 patients is complete. Once the first six are recruited and randomised, 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:
•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

•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

发起方

CTD HOLDINGS, INC.