
Trappsol Cyclo Shows Promising Results in Newborns With Niemann Pick Disease Type C1
New findings reveal early treatment with Trappsol Cyclo shows promise for young patients with Niemann-Pick disease type C1, enhancing safety and efficacy.
Newly presented data from an open-label substudy of the phase 3 TransportNPC trial (NCT04860960) highlighted the therapeutic benefits of intravenous Trappsol Cyclo (Cyclo Therapeutics) when administered early in patients aged less than 3 years old with Niemann pick disease type C1 (NPC1). Overall, early use of the investigational agent was considered safe, and led to stabilization or improvement in Clinical Global Impression-Change (CGI-C) scales in treated patients.1
Presented at the
Led by Caroline Hastings, MD, a hematologist-oncologist and neuro-oncologist at the University of California, San Francisco, the sub-study featured patients with a baseline CGI-S score ranging from very mild to severe disease. In terms of safety, the investigational drug demonstrated an acceptable safety and tolerability profile, with no related serious AEs across study participants. Overall, there were 2 patients who withdrew due to family decision, and a total of 122 treatment-emergent adverse events (TEAEs) recorded in the study. Of note, only 1 of these was assessed as possibly related to Trappsol Cyclo.
Also known as hydroxypropyl-beta-cyclodextrin, Trappsol Cyclo is an intravenously administered therapy designed to deliver first-in-class proprietary cyclodextrin intended to mobilize lysosomal cholesterol, thus targeting the root cause of NPC1. TransportNPC is an ongoing, prospective, double-blind, placebo-controlled trial that enrolled 94 patients aged 3 and older with a confirmed diagnosis of NPC1. Following the review of prespecified 48-week interim data by an independent Data Monitoring Committee, the trial is still expected to last for its original 96-week period.2
The announced continuation of the trial, which came in June of this year, also included a note that the FDA accepted the statistical analysis plan for TransportNPC. Within the ongoing trial, the primary end points are mean change in the 4-domain NPC Clinical Severity Scale (4D-NPC-CSS) score in the United States and the 5-domain NPC Clinical Severity Scale (5D-NPC-SS) score in Europe.
Cyclo Therapeutics has completed 2 studies of Trappsol prior to TransportNPC: a phase 1 study and a phase 1/2 trial (NCT02912793). In the phase 1/2 trial, 9 patients were assigned to Trappsol at doses of 1500 mg/kg (n = 2), 2000 mg/kg (n = 4), and 2500 mg/kg (n = 3) for a 48-week period. In a disease characterized by linear disease progression, treatment with Trappsol Cyclo resulted in the stabilization or improvement in 6 of 9 (67%) completer patients on the 5D-NPC-SS.3
Additional data from the phase 1/2 trial showed that 8 of 9 (89%) completer patients showed improvements over disease progression (2.15 points per annum) in the 5D-NPC-SS. Notably, all patients (9 of 9) who completed the treatment period exhibited stabilized or improved CGI-I ratings, further supporting the drug’s efficacy.
In the phase 1/2 trial, dosage data was suggestive of potential benefit on the Scale for Assessment and Rating of Ataxia (SARA), an exploratory measure. Several patients in the study exhibited stabilized or improved scores on SARA domains (gait, stance, sitting, and speech), compared with baseline, with greater benefits observed in the higher dosing cohorts. At 48 weeks, the higher 2500 mg/kg dose suggestively improved gait, stance, and speech, while the 2000 mg/kg dose improved stance and stabilized sitting and speech.
REFERENCES
1. Hastings C. TransportNPC: open-label phase 3 sub-study evaluating Trappsol Cyclo in patients under the age of 3 diagnosed with Niemann Pick Disease Type C1. Presented at: 2025 CNS Annual Meeting; October 8-11; Charlotte, NC. Abstract 362.
2. Rafael Holdings Announces Continuation of its Phase 3 Study for the Treatment of Niemann-Pick Disease Type C1 (NPC1) Following Independent Data Monitoring Committee (DMC) Review of Prespecified 48-Week Interim Data. News release. Rafael Holdings. June 18, 2025. Accessed October 21, 2025. https://firstwordpharma.com/story/5973706
3. Cyclo Therapeutics Scientific Presentation. December 2023. Accessed October 21, 2025. https://cyclotherapeutics.com/wp-content/uploads/2023/12/CYTH_Scientific-Data-Presentation_December-2023_FINAL21.pdf
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