Cyclo Therapeutics' Phase 3 NPC1 Study Advances with Positive Interim Data Review
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Cyclo Therapeutics, a subsidiary of Rafael Holdings, is progressing with its Phase 3 TransportNPC study for the treatment of Niemann-Pick Disease Type C1 (NPC1) using Trappsol® Cyclo™. The study continues after a positive interim review by an independent Data Monitoring Committee, confirming safety and efficacy at the 48-week mark. The FDA has approved the statistical analysis plan, and the investigational candidate shows a safety profile consistent with earlier studies. This study is noted for its comprehensive scale and clinical outcomes assessment.
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