Cyclo Therapeutics, through a collaboration with Rafael Holdings, reported preliminary findings from a Phase 3 sub-study of its investigational therapy Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin) for Niemann-Pick Disease Type C1 (NPC1).
The open-label, single-arm trial assessed patients younger than three years old over 48 weeks. According to the company, of the nine patients who reached the 48-week mark, seven demonstrated either stabilization or improvement on the Clinical Global Impression – Severity (CGI-S) scale. Three patients improved, while two showed worsening. One had not yet completed 48 weeks at the cutoff, and two withdrew earlier based on caregiver decision.
Safety outcomes were generally consistent with prior studies. By May 14, 2025, 146 adverse events had been recorded, most of which were mild (69 %) or moderate (29 %). Three were considered severe, and about 14 % were categorized as serious adverse events, though none were judged to be related to Trappsol® Cyclo™.
The broader TransportNPC study is a randomized, double-blind, placebo-controlled Phase 3 trial in patients aged three years and older, spanning more than 25 clinical sites in 13 countries. The under-3 sub-study runs outside the United States.
Cyclo Therapeutics stated that these early results support continued investigation and that further evaluation of efficacy, safety, and pharmacokinetics is planned once complete data become available.
