| Company Name | Janssen Taiwan |
|---|---|
| Protocol Number | 66674140NPC4003 |
| Title of Study | Multicenter, Post-authorization Safety Study to Collect Disease Outcome Information from Taiwanese Research Participants with Niemann-Pick Disease Type C |
| Primary Objective | The study is designed to describe Taiwanese Niemann-Pick disease type C (NP-C) research participants with or without Zavesca treatment in terms of their clinical characteristics, disease progression, and outcomes in real-world clinical practice. Physician will be invited to include all consecutive patients with NP-C, who present for outpatient and/or inpatient visits. This study will collect real-world data to assess NP-C therapies, as well as contribute to the knowledge base of NP-C through publications. The primary objectives of this study are: 1. To collect the clinical outcomes and safety information of NP-C research participants treated with Zavesca during the observation period. 2. To observe the natural disease course of NP-C in research participants who are not treated with Zavesca. |
| Number of Sites | 2 |
| Period of Study | From：2023/10/27 to：2027/02/26 |
| Number of Patients | 6人 |
| IRB Approval Date | National Taiwan University Hospital 2023/7/13 Taipei Chang-Gung Memorial Hospital 2023/9/19 |
| Publication Plan / Date | Unkown |
