Clinical trials are research studies that test how well new medical treatments or interventions work in people. These trials are essential for advancing medical knowledge and improving patient care. Visit the NPC Corner to learn more about impacts and benefits for someone living with Niemann-Pick disease, NP-C Corner: Will I be a lab rat? (npcd.org.au)

Overall, clinical trials play a crucial role in shaping the future of medicine by ensuring that new treatments are safe, effective, and beneficial for patients.

For an overview of the different phases of clinical trials, see our Therapy Pipeline Summary, Therapy Summary (npcd.org.au)

NPD Clinical Trials in Australia

If you are considering involvement in a clinical trial, you will first need to speak with your neurologist or leading clinician to assess your eligibility.

International Niemann-Pick Disease Registry (INPDR)

The INPDR is a disease-specific registry created by our community for our community – benefiting patients and families but also clinicians, researchers and other key stakeholders.

Having an international registry for Niemann-Pick diseases is particularly important given their ultra-rare status. There’s a real need for disease-specific information to support diagnosis, improve clinical care and advance research.

The INPDR encompasses Acid Sphingomyelinase Deficiency (ASMD, types A & B) and Niemann-Pick disease type C (NPC). We record information that accurately reflects the patient experience – in order to improve that experience

The ANPDF's Mandy Whitechurch is proud to be Australia's ambassador for the INPDR.

To find out more, or to register today, visit INPDR International Niemann-Pick Disease Registry