Ignite Creation Date:
2024-05-06 @ 4:05 PM
Last Modification Date:
2024-10-26 @ 2:03 PM
Study NCT ID:
NCT04860960
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-06-04
First Post:
2021-04-15
Brief Title:
Phase 3 Study to Evaluate Intravenous TrappsolR CycloTM in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
Sponsor:
Cyclo Therapeutics Inc
Organization:
Cyclo Therapeutics Inc
Study Overview
Official Title:
Phase 3 Double-blind Randomized Placebo-controlled Parallel-group Multicenter Study to Evaluate the Safety Tolerability and Efficacy of 2000mgkg of TrappsolCyclo Hydroxypropyl-B-cyclodextrin and Standard of Care Compared to Placebo and Standard of Care in Patients With Niemann-Pick Disease Type C1 TransportNPC
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-09
Last Known Status:
None
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:
TransportNPC
Brief Summary:
A prospective randomized double-blind placebo controlled multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 NPC1 The objective of this study is to evaluate the safety tolerability and efficacy of 2000 mgkg dose of Trappsol Cyclo hydroxypropyl betacyclodextrin administered intravenously compared to standard of care An open-label sub-study in countries following European Medicines Agency EMA guidance will enroll asymptomatic or symptomatic patients from infancy up to age 3 to evaluate safety in that population
Detailed Description:
The TransportNPC study is a prospective randomized double-blind placebo controlled therapeutic study for 93 patients age 3 and older with confirmed diagnosis of NPC1 The objective of this study is to evaluate the safety tolerability and efficacy of 2000 mgkg dose of Trappsol Cyclo hydroxypropyl betacyclodextrin administered intravenously by slow infusion every two weeks in addition to standard of care as compared to placebo and standard of care Standard of care may include Miglustat or leucine products that are not currently under investigation as a therapeutic Patients will be randomized to receive Trappsol Cyclo or placebo at a 21 ratio The study duration is 96 weeks with an unblinded interim analysis at 48 weeks An open-label extension of up to 96 weeks follows the interventional study Patients whose disease progression worsens by two levels in the Clinical Global Impression of Severity scale over 12 weeks starting at week 36 may be moved to open label treatment Efficacy will be measured at week 48 and week 96 by a composite score of major disease features A sub-study will be conducted in countries following EMA guidance for up to 12 patients age 0 - 3 years who may be asymptomatic Outcomes for the sub-study are safety clinical and caregiver impression of disease
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