Trial of VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Subjects with Niemann-Pick Type C1 Disease

Phase 1

• Conditions: eurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2015-002548-15-DE
• Lead Sponsor: Vtesse LLC, a Mallinckrodt Pharmaceutical Company

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-09-01
• Last Update Posted: 2 August 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 51

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
OR
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.
OR
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

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified