BTC Master Protocol, an umbrella trial for patients with biliary tract cancer (BTC) harboring rare aberrations, will serve as an efficient and patient-centric approach for research and drug development in BTC. This Master Protocol will allow for the concurrent study of multiple experimental treatment options for patients affected by this deadly disease. The main purpose of the trial is to evaluate experimental therapies and support their validation and approval in BTC. The validation/approval stage will consist of efficacy and safety assessments of identified agents followed by a confirmatory study if a signal is found. Future plans may include an exploratory stage for the discovery and study of new target aberrations in BTC.
The BTC Master Protocol is currently under development. Please follow our social media for trial updates and progress.
BTC MASTER PROTOCOL TRIAL LEADERSHIP
Tonios Bekaii-Saab, MD (Mayo Clinic)
Mitesh Borad, MD (Mayo Clinic)
Milind Javle, MD (MD Anderson)
Katie Kelley, MD (UCSF)
Stacie Lindsey (CCF)
Funda Meric-Bernstam, MD (MD Anderson)
Juan Valle, MB ChB, MSc, FRCP (CCF)
Meredith Buxton, PhD, MPH (GCAR)
BTC MASTER PROTOCOL KEY DESIGN ASPECTS
- Eligible patients to include newly diagnosed and progressive/recurrent unresectable locally advanced and/or metastatic BTC, including intrahepatic or extrahepatic CCA, GBC, and carcinoma of Ampulla of Vater
- All patients will undergo NGS testing to determine trial eligibility, providing a targeted therapeutic approach to treatment
- Primary endpoint: Overall Response Rate (ORR)
BTC MASTER PROTOCOL SCHEMA
- Master Protocol will provide more opportunities for patients to be assigned to a targeted therapy.
- Design will allow for multiple rare aberrations in BTC to be studied simultaneously with new experimental arms for individual target aberrations being added at any time.
- Lead by KOLs in the field.
- In collaboration with the Cholangiocarcinoma Foundation and GCAR.
BTC Master Protocol is sponsored by Global Coalition for Adaptive Research (GCAR), a non-profit, research delivery organization, in partnership with the Cholangiocarcinoma Foundation, and in collaboration with leading physicians in medical oncology and biopharmaceutical companies. GCAR’s core competencies include expertise in Master Protocols, and efficiencies in recruitment, cost, and trial infrastructure development.
