Clinical Research Details Clinical Research Phase 3, Double blind, Randomized, Placebo controlled, Multicenter Study for the Safety, Tolerability, Efficacy of Trappsol® Cyclo™ vs. Placebo in Niemann Pick Disease Study Description The main purpose of this study is to investigate the effect of the study drug Trappsol Cyclo and the current standard-of-care, compared to placebo and the current standard of care, on the disease progression and symptoms of a rare genetic disorder called Niemann Pick Disease Type C1 (NPC). Those with this condition can experience many symptoms related to a decline in cognitive (thinking) and motor (movement) function, as well as difficulty swallowing, sleep disturbances, and pneumonia. The study drug is provided by the Sponsor. Total participation in this study will be up to 100 weeks (23 months). The patient will need to visit the clinic at least 52 times and will receive treatment every 2 weeks. Patients will have blood work, exams, EKGs, lung function tests, hearing tests, questionnaires, swallowing tests, and urine tests. It is open for patients over 3 years of age. Inclusion/Exclusion Criteria Patients who have NPC with at least one neurological symptom. Open Enrollment Contact Name: Amanda PrinceContact Phone: (904) 427-7079Contact Email: amanda.prince@jax.ufl.edu Investigators Estrella Lizbeth Mellin Sanchez, M.D. Pediatrics Pamela L. Trapane, M.D. Pediatrics