Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of Niemann-Pick Type C1
Read the full statement from Cyclo Therapeutics here:
Here is a summary of the article:
Landmark Milestone: Cyclo Therapeutics has completed enrollment for their Phase 3 TransportNPC™ trial, which is a pivotal study for treating Niemann-Pick Disease Type C1 (NPC1).
Study Details: The study enrolled 104 patients globally and includes a substudy for children aged 0-3 years. It aims to evaluate the efficacy of Trappsol® Cyclo™ for systemic and neurological symptoms of NPC1.
Interim Analysis: Topline data from a 48-week interim analysis is expected in the first half of 2025. If the data shows significance, the company plans to submit a New Drug Application to the FDA and a Marketing Authorization Application to the EMA.
Disease Impact: NPC1 is a rare genetic disease that affects 1 in 100,000 live births, leading to cholesterol accumulation in cells and causing dysfunction in various organs.
For more information about the Company’s TransportNPC™ pivotal Phase 3 study, visit www.ClinicalTrials.gov and reference identifier NCT04860960.
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