International Niemann–Pick Disease Alliance
| Disease | Study Drug | Route | Sponsor | Trial Name | Sites Enrolled Patients | ClinicalTrials.gov Ref. No. |
|---|---|---|---|---|---|---|
| ASMD | olipudase alfa | IV | Sanofi | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (ASCEND) | US, Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, UK | NCT02004691 |
| Niemann-Pick Type C | N-acetyl-L-leucine | oral | IntraBio | A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C | US, Australia, Czechia, Germany, Netherlands, Slovakia, UK, Switzerland | NCT05163288 |
| Niemann-Pick Type C | N-acetyl-L-leucine | oral | IntraBio | N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) | US, German, Slovakia, Spain, UK | NCT03759639 |
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