International Niemann–Pick Disease Alliance
| Disease | Study Drug | Route | Sponsor | Trial Name | Trial sites with enrolled patients | Clinicaltrails.gov reference # | Current status |
|---|---|---|---|---|---|---|---|
| NPC | Adrabetadex 2-hydroxyproply-beta cyclodextrin | Intrathecal | Mandos Health | Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease | Australia, France, Germany, New Zealand, Singapore, Switzerland, Turkiye United Kingdom, USA |
NCT04958642 | NDA submitted to FDA |
| NPC | Adrabetadex 2-hydroxyproply-beta cyclodextrin |
IV | Washington University | Study of IV VTS-270 for infantile liver disease associated with NPC | St Louis, Missouri, USA |
NCT03471143 | Study completed |
| NPC | Arimoclomol/Miplyffatm | oral | Zevra Therapeutics | Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C | Denmark, France, Italy, Poland, Germany, Spain, Switzerland, United Kingdom USA |
NCT02612129 | Approved by FDA September 2024. Further regulatory submissions including EMA pendin |
| NPC | N-acetyl-L-leucine/Aqneursatm | oral | IntraBio | A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C | Denmark, France, Italy, Poland, Germany, Spain, Switzerland,, United Kingdom, USA |
NCT05163288 | Approved by FDA September 2024. NDA submitted to EMA. Further regulatory submissions pending |
| ASMD | Olipudase Alfa/Xenpozymetm | IV | Sanofi | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (ASCEND) | Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, UK, USA | NCT02004691 | Regulatory approval received from FDA, EMA and others, global submissions ongoing, Not all regulatory approvals are accompanied by HTA approvals for funding |
| ASMD | Olipudase Alfa/Xenpozymetm | IV | Sanofi | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency (ASCEND-Peds) | Brazil, France, Germany, Italy, United Kingdom USA |
NCT02292654 | As above |
| ASMD | Olipudase Alfa/Xenpzymetm | 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 | Study complete |
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