TransportNPC
phase 3research program · high · Tue Feb 04 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate intravenous Trappsol Cyclo in pediatric and adult patients with confirmed NPC1, including patients age 3 and older and an open-label sub-study for patients below age 3.
Prospective, randomized, double-blind, placebo-controlled, multi-center Phase 3 study of 2000 mg/kg IV Trappsol Cyclo compared with standard of care, plus an open-label sub-study in infants and children under 3 years of age.
2025 WORLDS poster described open-label treatment in the TransportNPC sub-study in patients under age 3 diagnosed with NPC1.
Trappsol Cyclo for Niemann-Pick Disease Type C1
phase 3drug program · high · Tue Feb 04 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Treat Niemann-Pick Disease Type C1 by facilitating transport of accumulated cholesterol out of cellular lysosomes and assessing safety, tolerability, efficacy, disease severity, neurologic symptoms, and cholesterol-related biomarkers.
Intravenous proprietary hydroxypropyl beta cyclodextrin formulation administered in clinical studies, including dose-ranging Phase 1/2 studies, long-term treatment, expanded access, and the Phase 3 TransportNPC program.
2025 WORLDS posters reported long-term efficacy and safety data from 4 clinical studies and an ongoing Expanded Access Program.
A 48-week Phase I/II study reported that IV Trappsol Cyclo was generally well tolerated; among 9 completers, 8 improved in at least two 17D-NPC-CSS domains and 7 were judged improved by treating physicians while 2 remained stable.
Trappsol Cyclo for Early Alzheimer's Disease
undiscloseddrug program · high · Fri Sep 23 2022 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate safety and potential efficacy of Trappsol Cyclo in patients aged 50 to 80 with early Alzheimer's disease.
Six-month U.S. clinical study enrolling approximately 90 patients into three blinded treatment groups: two active-dose groups and one placebo group, with screening, treatment, and follow-up periods.
ClinicalTrials.gov record for a 6-month early Alzheimer's disease study was published on 2022-09-23.
Trappsol Cyclo NPC1 Pharmacokinetics and Biomarker Study
phase 1biomarker · medium · Wed Oct 11 2017 00:00:00 GMT+0000 (Coordinated Universal Time)
Assess Trappsol Cyclo safety, pharmacokinetics, and effects on potential biomarkers of NPC1, including cholesterol metabolism and tissue effects.
Intravenous Trappsol Cyclo at two dose levels every two weeks with blood, urine, cerebrospinal fluid sampling, liver and skin biopsy specimens, filipin staining, cholesterol metabolism analysis, splenic and hepatic elasticity ultrasound, hearing tests, questionnaires, and clinical assessments.
The related ClinicalTrials.gov record describes the Phase I trial that later supported continued-access treatment under the long-term safety and efficacy study.
