An Early Look at Promising Results for Trappsol® Cyclo™
Cyclo Therapeutics has released preliminary findings from a Phase 3 sub-study of its investigational drug Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin), aimed at treating a rare genetic disorder known as Niemann-Pick Disease Type C1 (NPC1). The study, conducted in collaboration with Rafael Holdings, focused on very young patients, specifically those under three years old.
Study Design and Findings
This open-label, single-arm trial followed nine patients over a period of 48 weeks. Of these, seven showed either stabilization or improvement on the Clinical Global Impression – Severity (CGI-S) scale, a tool used by clinicians to rate a patient’s overall illness severity. Three patients showed a clear improvement, while two showed a worsening of their condition. Two patients withdrew from the study earlier due to caregiver decisions, and one had not yet reached the 48-week mark at the time of the report.
Niemann-Pick disease type C1 is a rare, progressive, and fatal genetic disorder. It’s characterized by the body’s inability to properly transport cholesterol and other fats within cells, leading to their abnormal accumulation in various tissues, including the brain. This can cause severe neurological problems and organ damage. The fact that the drug led to stabilization or improvement in a majority of patients in this progressive disease is seen as a positive sign.
Safety and Broader Context
The safety profile of Trappsol® Cyclo™ in this sub-study was generally consistent with prior research. Out of 146 adverse events recorded, the majority were classified as mild (69%) or moderate (29%). Three events were severe, and while 14% were considered serious, none were deemed to be related to the study drug itself.
These preliminary results come from a sub-study of the larger TransportNPC trial, which is a randomized, double-blind, placebo-controlled Phase 3 study for patients aged three and older. The sub-study, conducted outside the United States, focuses on a younger, particularly vulnerable patient population where early intervention is crucial for potential long-term benefits. The company plans to conduct a more complete evaluation of efficacy, safety, and pharmacokinetics as more data become available from all participants.