EUCTR2015-002548-15-DE进行中（未招募）1 期
A Phase 2b/3 Prospective, Randomized, Double-blind, Sham-controlled Trial of VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
Vtesse LLC, a Mallinckrodt Pharmaceutical Company0 个研究点目标入组 51 人开始时间: 2015年9月1日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 51
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
- •Study Parts A and B – not applicable as Part A and B are completed.
- •Study Part C – NO NEW SUBJECTS ARE ALLOWED TO ENTER THE STUDY AS OF 20 JANUARY 2021
- •Inclusion Criteria:
- •1. Subject has completed Study Part B, meets the criteria for dose reduction for a second time or meets the criteria for the rescue option
- •2. Subject is a current participant in the NIH phase 1/2a open-label study and:
- •a. Subject agrees to convert from the dose of VTS-270 currently receiving as a subject in the NIH phase 1/2a protocol to the dose chosen for Parts B and C of this study, 900 mg.
- •b. Subject agrees to convert from the monthly dosing regimen used in the NIH phase 1/2a protocol to an every 2-week dosing regimen.
- •c. In instances where NIH phase 1/2a subjects eligible to enroll into Study Part C are unable to convert from their current NIH phase 1/2a dose or monthly regimen, the investigator must receive prior written authorization from the sponsor for the subject to enter Part C of the study on an amended dose and/or regimen.
- •3. Subject has received prior written authorization from Vtesse to enroll directly into Study Part C.
- •4. Females of childbearing potential (not surgically sterile) must use a medically acceptable method of contraception and must agree to continue use of this method for the duration of the study and for 30 days after participation in the study. Acceptable methods of contraception include barrier method with spermicide, intrauterine device, steroidal contraceptive in conjunction with a barrier method, abstinence, or same-sex partner.
- •5. Subject or parent/guardian must provide written informed consent to participate in the study. In addition to parental consent, assent to participate must also be sought from minor children.
- •Subject Inclusion Criteria for European Site-specific Device Safety and Tolerability Substudy – Not applicable – enrollment in the Device Substudy concluded Aug 2018.
- •Subject Inclusion Criteria for the European Site-specific Device Pharmacokinetic (PK) Substudy – Not applicable – no subject consented to participate in the PK Substudy.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 35
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 16
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Study Parts A and B – not applicable as Part A and B are completed.
- •Study Part C – NO NEW SUBJECTS ARE ALLOWED TO ENTER THE STUDY AS OF 20 JANUARY 2021
- •Subject Exclusion Criteria for the European Site-specific Device Safety and Tolerability Substudy – Not applicable - enrollment in the Device Substudy concluded Aug 2018.
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