Trailblazing WIND-PSC Study is Enrolling Patients and Adding Study Sites
- PSC Partners Patient Registry
- Apr 8
- 3 min read
Updated: Apr 9
WIND-PSC is a PSC Partners-led and -sponsored initiative that was inspired by the call of the PSC community to identify new treatment options for PSC and address unmet medical needs of PSC patients. It is the inaugural project of the PSC Partners International Collaborative Research Network (ICRN), a research community that brings representatives from the patient community together with those from the scientific, medical, and clinical research realms to the same table for collaborations.
The primary goal of the WIND-PSC study is to generate key data designed specifically to remove barriers for drug development. To achieve this, clinical data, biosamples, and patient-reported experiences will be collected in a central database and biorepository for research, drug development, and regulatory needs. Patient recruitment and enrollment is underway and study sites are coming on board!
The multi-year study has been under development since 2022 and enrolled its first patient in 2024. Study participants will be followed prospectively for five years. Medical centers from around the world have been invited to contribute clinician-reported data, and study participants will contribute annual blood samples and patient-reported data on symptom and quality-of-life experiences.
Currently, eight medical centers from three countries (United States, Canada, and Germany) are enrolling patients in WIND-PSC, and more centers will be joining the initiative in the coming months. The goal is to enroll 2,000 patients across up to 20 sites.
The primary aim of WIND-PSC is to serve as a source of real-world data to construct an appropriate “external control group” for certain confirmatory clinical drug trials for PSC. In drug development, data typically is collected from an enrolled, blinded control group receiving a placebo along the same timeline as a group receiving an experimental drug. However, regulatory agencies have affirmed that, for rare diseases and under certain conditions, it is allowable to submit data from an external control group in lieu of incorporating a placebo group as part of the drug trial. The requirements for this use have been built into the design of WIND-PSC.
By leveraging WIND-PSC instead of a traditional placebo group, clinical trials can enroll fewer patients, thus speeding up and reducing the enormous costs associated with running clinical trials, both of which pose significant barriers in developing treatments for rare diseases such as PSC.
A second aim of the study is to contribute to the development of biomarkers that could be used to demonstrate effectiveness of investigational drugs for PSC. One reason clinical trials for PSC are challenging is because there is a lack of noninvasive biomarkers proven to correlate with PSC disease progression.
The WIND-PSC initiative launched at an exciting time. A number of therapeutics are in clinical trials for PSC, making now the best time to begin collecting data to serve as an external control arm. As these drugs advance through clinical trial phases and – hopefully – come to market, WIND-PSC data will be ready to support and accelerate drug development efforts.
To read more details about the WIND-PSC initiative and the ICRN, please follow these links;
History and summary of WIND-PSC
June 2024 news release - first WIND-PSC patient enrolled in study
ClinicalTrials.gov listing - WIND-PSC study details
The International Collaborative Research Network (ICRN)