Personalized Clinical Core
Dr. Clancy, who will direct this Core, has been in the forefront of drug development and clinical trials of VX-770, VX-809, VX-661, ataularen, Nivalis, QR-010, and combination therapies. The Core activities include clinical trial design and conduct in vivo assays of CFTR (sweat Cl, NPD, ICM) and use of established and novel tools to monitor interventions and bioactivity (spirometry, lung clearance index, FeNO, CT, UTE-MRI and HP129Xe imaging), biomarker identification and standardization, and manuscript preparation.
Dr. Clancy was one of the original members to study the safety and adverse profile of VX-770 (Kalydeco), which has since been approved by the FDA to treat CF caused by G551D mutation. Under his leadership and assisted by Drs. McPhail and Woods, this Core will offer a wide range of services, including:
- Nasal potential difference measurements
- Intestinal current measurements
- spirometry
- FeNO measurements
- MBW
- Infant pulmonary function test
- Sweat testing.
The Core will also be responsible for coordinating the nasal and tracheal brushings and biopsies, bronchial biopsies, BALF and serum procurement, processing, storage and distribution, and tissue procurement from lung transplants. The Core will also perform transformative lung imaging of CF patients, controls, and other relevant populations (e.g., transplant patients) by using novel and emerging MRI methods (such as UTE and hyperpolarized Xe and He gases, with Dr. Jason Woods). In addition, the Core will also coordinate the procurement of intestinal/colonic/rectal tissues. Biostatistics support will be provided by Rhonda Szczesniak. This Core will serve not only the research interests of the CF-Center at Cincinnati Children's (and other interested investigators) but also external investigators.
