Ignite Creation Date:
2024-05-06 @ 12:58 PM
Last Modification Date:
2024-10-26 @ 1:06 PM
Study NCT ID:
NCT03893071
Status:
UNKNOWN
Last Update Posted:
2021-06-18
First Post:
2019-02-20
Brief Title:
Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo HPβCD in Niemann-Pick Disease Type C
Sponsor:
Cyclo Therapeutics Inc
Organization:
Cyclo Therapeutics Inc
Study Overview
Official Title:
An Open-Label Extension Study of the Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo HP-β-CD in Patients With Niemann-Pick Disease Type C NPC-1
Status:
UNKNOWN
Status Verified Date:
2021-02
Last Known Status:
ACTIVE_NOT_RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101 when administered at doses of 1500 mgkg and 2500 mgkg by slow IV infusion over a period of 8 to 9 hours every two weeks
Detailed Description:
This is an open-label extension study of intravenous HP-β-CD administered as Trappsol CycloTM in patients with NPC-1 to evaluate long-term safety and efficacy by providing continued access to treatment following completion of study CTD-TCNPC-101 Patients will receive medication at their home or local site under the supervision of a home nurse professional or at the parent site under the supervision of the site principal investigator Patients will visit the parent site for safety and efficacy evaluations after receiving treatment for 3 months in the extension protocol
Thereafter visits to the parent site will be every 6 months until cessation The study will continue until Trappsol Cyclo TM becomes commercially available or studysitepatient discontinuation All patients who complete study CTD-TCNCP-101 and pass the screening criteria will be eligible for this study The study will be directed from parent site in the US Vital signs AEs Adverse Events AEs and concomitant meds will be recorded by the home nurse professional or deputy at the local site and reported immediately to the parent site for entry into the Electronic Data Capture EDC and reporting to the sponsor
For logistical reasons home infusions will be permitted as long as AEs infusion details and concomitants medications are reported directly to the parent site by an authorised deputy according to local guidelines A Safety Review Committee SRC consisting of an independent advisor the medical monitor and the principal investigator will be established to review AEs and laboratory data throughout the study
Thereafter visits to the parent site will be every 6 months until cessation The study will continue until Trappsol Cyclo TM becomes commercially available or studysitepatient discontinuation All patients who complete study CTD-TCNCP-101 and pass the screening criteria will be eligible for this study The study will be directed from parent site in the US Vital signs AEs Adverse Events AEs and concomitant meds will be recorded by the home nurse professional or deputy at the local site and reported immediately to the parent site for entry into the Electronic Data Capture EDC and reporting to the sponsor
For logistical reasons home infusions will be permitted as long as AEs infusion details and concomitants medications are reported directly to the parent site by an authorised deputy according to local guidelines A Safety Review Committee SRC consisting of an independent advisor the medical monitor and the principal investigator will be established to review AEs and laboratory data throughout the study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None