International Niemann–Pick Disease Alliance
| Disease | Study Drug | Route | Sponsor | Trial Name | Sites Enrolled Patients | ClinicalTrials.gov Ref. No. | Contact for Enrollment Questions |
|---|---|---|---|---|---|---|---|
| Niemann Pick Type C | arimoclomol | oral | Zevra | Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C | US, Denmark, France, Italy, Poland, Germany, Spain, UK, Switzerland | NCT02612129 | |
| 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 | |
| Niemann Pick Type C | AZ-3102 | oral | Azafaros | Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease (RAINBOW) | US, Brazil | NCT05758922 | Name: Christian Freitag, MD Email: chris.Freitag@azafaros.com Phone: +41 78 259 80 80 |
| Niemann Pick Type C | Efavirenz | Oral | No pharma sponsor, small trial supported by FNP | Efavirenz clinical trial in Niemann-Pick Type C (NPC) | Spain | no reference number |
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
This lecture will present the Australian collaboration and latest research on NP-C, a rare genetic disorder that leads to childhood dementia.
The importance of the patient, clinician and scientist working together, especially for rare diseases, is increasingly recognised as key to achieving better health outcomes for the patient. This lecture will feature presentations from the Australian NPC disease Foundation (Niemann-Pick Disease Type C patient advocacy and support group), Professor Mark Walterfang (neuropsychiatrist) and Dr. Ya Hui Hung (scientist), who are working together with a common goal to improve the health of Niemann-Pick Disease Type C (NP-C) patients. Watch below, and read more here.