Trappsol Cyclo for Niemann-Pick Disease
(TransportNPC Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing a medicine called Trappsol Cyclo, given through an IV, for patients with Niemann Pick disease type C1. The medicine helps remove harmful fat buildup in cells. The study will also look at safety in younger children. Trappsol Cyclo has shown promising results by mobilizing cholesterol, extending life, and reducing neurological damage in Niemann-Pick disease type C1.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop all current medications, but you must not use curcumin or fish oil within 12 weeks before enrolling. If you are taking an investigational drug, you need to stop it at least 3 months before the study, unless it has a short half-life, in which case the washout period is 1 month.
How is the drug Trappsol Cyclo different from other treatments for Niemann-Pick Disease?
Trappsol Cyclo is unique because it uses 2-hydroxypropyl-β-cyclodextrin to help restore cholesterol balance in the body and brain, which is crucial for treating Niemann-Pick Disease Type C1. Unlike other treatments, it is administered intravenously and has shown potential in improving liver function and reducing neurodegeneration.12345
What data supports the effectiveness of the drug Trappsol Cyclo for Niemann-Pick Disease?
Who Is on the Research Team?
Karen Mullen, MD
Principal Investigator
Cyclo Therapeutics, Inc.
Are You a Good Fit for This Trial?
This trial is for pediatric and adult patients with Niemann-Pick Disease Type C1. Participants must have a confirmed diagnosis, be able to follow the study's procedures, and weigh between 4.5 kg and 125 kg. They should not have severe kidney disease, recent liver transplant or active liver disease unrelated to NPC1, nor be pregnant or breastfeeding.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive 2000 mg/kg of Trappsol Cyclo or placebo intravenously every two weeks
Interim Analysis
Unblinded interim analysis to evaluate efficacy and safety
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants may opt into continuation of treatment long-term
What Are the Treatments Tested in This Trial?
Interventions
- Trappsol(R) Cyclo(TM)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Cyclo Therapeutics, Inc.
Lead Sponsor