International Niemann–Pick Disease Alliance
Disease | Study Drug | Route | Sponsor | Trial Name | Sites Enrolling Patients | ClinicalTrials.gov 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 Email: pdickson@wustl.edu 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 Email: Lori.Gorski@cyclodex.com 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 https://www.fda.gov/patients/clinical-trials-what-patients-need-know/basics-about-clinical-trials
The drug development process – resource from the FDA https://www.fda.gov/patients/learn-about-drug-and-device-approvals/drug-development-process