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 sites51 target enrollmentSeptember 1, 2015

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

September 1, 2015

End Date

TBD

Last Updated

4 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Vtesse LLC, a Mallinckrodt Pharmaceutical Company

Eligibility Criteria

Inclusion Criteria

•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.

Exclusion Criteria

•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\.