International Niemann–Pick Disease Alliance

Active studies that are enrolling patients

Disease Study Drug Route Sponsor Trial Name Sites Enrolling Patients Ref. No. Contact for Enrollment Questions
Niemann Pick Type C adrabetadex (2-Hydroxypropyl-Beta-Cyclodextrin) IV Washington University Study of IV VTS-270 for Infantile Liver Disease associated with NPC US NCT03471143 Name: Patricia I Dickson, MD
Phone: 314-273-2943
Niemann Pick Type C Hydroxypropyl-beta-cyclodextrin/Trappsol® cyclodextrin IV Cyclo Therapeutics Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 (TransportNPC) US, Australia, Brazil, Germany, Isreal, Italy, Poland, Spain, Taiwan, Turkey, United Kingdom NCT04860960 Name: Lori M Gorski
Phone: 1-508-410-0104
ASMD Olipudase alfa IV Sanofi A prospective observational study to assess the long term safety and immunogenicity of Olipudase Alfa therapy during routine clinical care in pediatric patients less than 2 years of age with acid sphingomyelinase deficiency London, Ontario, Canada NCT06192576
ASMD Olipudase alfa IV Sanofi Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France (OPERA) France NCT05359276

Further resources to understand clinical trials and drug development:

What patients need to know about clinical trials – resource from the FDA

The drug development process – resource from the FDA

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