Niemann-Pick Disease, Type C clinical trials
6 active trials name this condition. Describe your situation to see which ones look like a fit.
By location
- 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)
- A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
- 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, GM1 Gangliosidosis or GM2 Gangliosidosis
- Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
- Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Guides for people searching for a trial
- Why people get turned down — and what to do nextWhat eligibility criteria are for, what keeps people out, and where to go when the first door closes.
- How your biomarkers decide which trials you qualify forEGFR, HER2, BRAF, PD-L1 and the rest — what they are and why they increasingly gate access.
- How to search for a trial without losing your mindWhere to look, what to gather first, and how to read what you find.
- What Phase 1, 2 and 3 actually mean for youWhat each phase is testing, and how to weigh an early-phase trial against a later one.
- 10 questions to ask before you say yesTreatment, time, cost, safety and your rights. Worth bringing to a screening visit.
- You matched with a trial. What happens now?Phone screens, the screening visit, informed consent, and day one.
Showing active trials that list “Niemann-Pick Disease, Type C” as a condition. Eligibility shown on each trial is an estimate — the trial's study team makes the final decision.