International Niemann–Pick Disease Alliance
| Disease | Study Drug | Route | Sponsor | Trial Name | Trial sites with enrolled patients | Clinicaltrails.gov reference # | Contact for enrollment questions | |
|---|---|---|---|---|---|---|---|---|
| NPC | Hydroxypropyl-beta-cyclodextrin/Trappsol® cyclodextrin | IV | CycloTherapeutics | Transport NPC Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 |
Austalia, Brazil, Germany, Israel, Italy, Poland, Spain, Taiwan, Turkiye, United Kingdom, USA | NCT04860960 | Name: Lori M Gorski Email: Lori.Gorski@cyclodex.com Phone: 1-508-410-0104 |
|
| NPC | Efavirenz | oral | No pharma sponsor, small clinical trial supported by Fundación Niemann-Pick de España |
Efavirenz clinical trial in aducts with Niemann-Pick Type C | 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.