Cyclo Therapeutics Clinical Trial Update | October 1, 2026
New Phase 3 results for Trappsol Cyclo in NPC
Cyclo Therapeutics has shared the first results from its global Phase 3 TransportNPC study. The study tested Trappsol Cyclo as a possible treatment for Niemann-Pick disease type C (NPC).
The study involved 94 people at 27 sites across 13 countries. Australians living with NPC were among those who took part, making our community directly involved in this important global research. This is one of the largest and longest placebo-controlled treatment studies conducted in NPC.
ANPDF thanks every person who took part, along with their families, researchers and clinical teams. We especially acknowledge the Australian participants and families who contributed to the trial. Taking part in a clinical trial is a major commitment, and their involvement helps build knowledge and move NPC research forward for the whole community.
What did the main study find?
The study measured changes in four important areas of NPC over 96 weeks.
When all participants were considered together, the results favoured Trappsol Cyclo over placebo. However, the difference was not statistically significant. This means the study did not meet its main goal, known as the primary endpoint.
This does not necessarily mean that the treatment had no effect. It means the study could not show, with the required level of confidence, that the difference was caused by the treatment rather than chance.
Why are rare disease trials challenging?
Statistical significance is harder to achieve when a study has a small number of participants.
Although 94 participants is a large study for NPC, it is still small compared with trials for common conditions. NPC also affects each person differently. Age, symptoms, stage of the condition and other treatments can all influence how quickly NPC changes.
These differences can make a treatment effect harder to detect. A small trial may miss a real benefit because there are not enough participants to separate the effect of the treatment from the natural differences between people.
However, a result that is not statistically significant cannot be treated as proof that a medicine works. Researchers and regulators must look closely at the full results, including the size of any benefit, safety findings, subgroup results and other supporting evidence.
What was found in participants using other treatments?
Before the study began, researchers planned to look separately at participants who were also taking miglustat and/or leucine.
This group represented about 83% of all participants. In this group, Trappsol Cyclo showed a statistically significant benefit on the same NPC severity scale.
This is an encouraging finding because the analysis was planned in advance and included most of the study population. The full data will be needed to better understand why this group responded differently and what the result may mean for people receiving other NPC treatments.
Important survival findings
The study also reported significant findings for children with infantile-onset NPC.
Researchers compared children treated with Trappsol Cyclo with similar patients from the International Niemann-Pick Disease Registry. This analysis found an 85% lower risk of death among those who received Trappsol Cyclo.
A second analysis included additional information from published studies of NPC. In that comparison, treatment was linked with a 94% lower risk of death.
These findings are especially important because infantile-onset NPC can progress quickly and has a high risk of early death. If the survival findings are confirmed, they could represent a meaningful advance for some of the youngest and most seriously affected people in our community.
However, these results need to be interpreted carefully. The Trappsol Cyclo group was compared with external and historical information, rather than with the placebo group in the trial. Even when patients are carefully matched, there may be differences between the groups that affect the result.
The size of the reported survival benefit makes these findings important and worthy of close attention. The complete data and further review will help show how strong the evidence is.
What was reported about safety?
Cyclo Therapeutics reported that Trappsol Cyclo was generally well tolerated during the 96-week study. Serious adverse events occurred more often in the Trappsol Cyclo group than in the placebo group. However, serious events considered to be related to treatment were reported at similar rates in both groups.
One severe treatment-related case of hearing loss in both ears was reported. No treatment-emergent adverse events resulted in death.
The complete safety results will be important in understanding the possible benefits and risks of treatment.
What happens next?
Cyclo Therapeutics plans to submit a New Drug Application for Trappsol Cyclo to the US Food and Drug Administration in the fourth quarter of 2026.
These are topline results only. The complete study data and survival analyses still need to be fully reviewed. The announcement does not mean the treatment has been approved or that access will change immediately.
ANPDF welcomes progress toward more treatment options for people living with NPC. We will continue to follow the results closely, seek clear answers on behalf of Australian families and share further information as it becomes available.
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References:
Rafael Holdings. (2025, June 18). 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 Analysis. https://rafaelholdings.irpass.com
MarketScreener. (2025, June 12). Rafael Holdings: Financial and Clinical Pipeline Update. https://www.marketscreener.com
GlobeNewswire. (2025, March 26). Rafael Holdings Completes Merger with Cyclo Therapeutics Following Shareholder Approvals. https://www.globenewswire.com
GlobeNewswire. (2025, June 11). Rafael Holdings Reports Third Quarter Fiscal 2025 Financial Results. https://www.globenewswire.com
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