Nieman-Pick Disease Type C1 Treatment with Trappsol® Cyclo™ – Phase III

Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg Trappsol® Cyclo™ (Hydroxypropyl-β-cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann-Pick Disease Type C1

Protocol Description

This multi-center international study tests the effects of Trappsol Cyclo combined with standard-of-care medication for people with Nieman-Pick disease, type C1 (NPC1). Standard of care may include other approved therapeutic drugs. NPC1 is a rare genetic disorder causing a buildup of lipids (fats) in the liver, brain, lungs, and spleen, which can lead to a decline in cognitive and motor function (movement), as well as a difficulty swallowing, sleep disturbances, and pneumonia.

Study Level: Phase III

Eligibility Criteria

Subject to certain exclusion criteria, this study is accepting people with NPC1, ages 3 and older.
Males and Females: Ages 3 years and older


Based on random selection, study participants will be divided into groups that will receive either the study drug or a placebo administered by intravenous infusion every 2 weeks at the study clinic while still receiving standard of care. During visits, participants will receive physical examinations and other tests that may include blood and urine sampling, electrocardiogram, and respiratory function tests among others.
Visits: At least 52
Duration: 23 months

Status: Open to Enrollment

Source(s) of Support

Cyclo Therapeutics, Inc.

Primary Investigator

Damara Ortiz, MD

Contact Information

For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC