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 | Nizubaglustat / AZ-3102 | Oral | Azafaros | A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) | 30 clinical trial sites including: Argentina, Australia, Brazil, Canada, Germany, India, Italy, Portugal, Spain, Switzerland, Türkiye, United Kingdom, USA (see ClinicalTrials.gov for details) | NCT07082725 | info@azafaros.com www.azafaros.com |
| ASMD | Olipudase alfa | IV | Sanofi | A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD) | The 5 U.S. sites are located in:
|
NCT06192576 | contact-us@sanofi.com |
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