International Niemann–Pick Disease Alliance
The International Niemann-Pick Disease Alliance (INPDA) is pleased to share an important update from Cyclo Therapeutics, marking a significant milestone in global research efforts for Niemann-Pick disease type C (NPC).
Cyclo Therapeutics has announced that the final patient has completed their last visit in the Phase 3 TransportNPC™ clinical study, which is evaluating the investigational therapy Trappsol® Cyclo™. This milestone marks the completion of the active phase of one of the most significant clinical studies undertaken in NPC and represents an important step forward in the ongoing effort to identify effective treatment options. For the international NPC community, this progress reflects years of shared commitment and collaboration across borders.
The TransportNPC™ study stands as the most comprehensive clinical trial conducted in NPC to date, involving 94 patients across 27 sites in 13 countries. This global scale highlights the importance of international cooperation in advancing rare disease research, bringing together clinicians, researchers, and patient communities worldwide.
In addition to the main study, a sub-study included ten patients from newborn to 3 years of age, helping to broaden understanding of NPC across different stages of life and further strengthening the knowledge base for future research and care approaches.
Cyclo Therapeutics has indicated that top-line results from the study are expected later this year, alongside plans to submit a New Drug Application (NDA) in the United States in the second half of the year. While the results are still to come, reaching this milestone marks a critical point in a development programme that began with early compassionate use efforts in 2009 and has since evolved into a large-scale, global clinical trial.
This achievement reflects the dedication and resilience of the entire NPC community, including patients, families, healthcare professionals, researchers, and advocacy organisations around the world. Progress in rare disease research depends on strong international partnerships, and this milestone demonstrates what can be achieved when the global community works together towards a shared goal.
As the community looks ahead to the release of study results, there is a collective sense of hope that this research will help shape future treatment pathways and improve outcomes for people living with NPC worldwide.
For more detailed information, you can read more on the Cyclo Therapeutics website.