A Phase II, Single Arm Study of BGJ398 in Patients With Advanced Cholangiocarcinoma

Adult patients with histologically or cytologically confirmed advanced or metastatic
cholangiocarcinoma with FGFR genetic alterations who have evidence of radiologic progression
following a cisplatin-and gemcitabine-containing regimen for advanced disease or a
gemcitabine-containing regimen for those who are considered intolerant to cisplatin will be
enrolled. Approximately 55 adult patients over age 18, both male and female will be enrolled.
Of these 55 patients, 40 patients will have cholangiocarcinoma with identified FGFR2 gene
fusions and up to 15 patients may have other FGFR genetic alterations. All patients will
receive oral BGJ398, once daily, on a three weeks on (21 days), one week off (7 days)
schedule. One treatment cycle will consists of 28 days.

Inclusion criteria:

- Adult patients with histologically or cytologically confirmed cholangiocarcinoma at the
time of diagnosis.

Patients with cancers of the gallbladder or ampulla of Vater are not eligible.

- Patients must have received at least one prior regimen containing gemcitabine with or
without cisplatin for advanced/ metastatic disease. Patient should have evidence of
progressive disease following prior regimen, or if prior treatment discontinued due to
toxicity must have continued evidence of measurable or evaluable disease.

Exclusion criteria:

- Prior or current treatment with a MEK or selective FGFR inhibitor

- insufficient organ function

- Absolutely Neutrophil Count (ANC) < 1,000/mm3 [1.0 x 109/L]

- Platelets < 75,000/mm3 [75 x 109/L]

- Hemoglobin < 109.0 g/dL

- Total bilirubin > 1.5x ULN

- Aspartate aminotransferase/glutamic oxaloacetic transaminase/GOT (AST/SGOT) and
Alanine aminotransferase/glutamic pyruvic transaminase/GPT (ALT/SGPT) > 2.5x ULN
(AST and ALT) > 5x upper limit of normal (ULN) in the presence of liver
metastases)

- Serum creatinine > 1.5x ULN and a calculated or measured creatinine clearance <
45 mL/min

- Inorganic phosphorus outside of normal limits

- Total and ionized serum calcium outside of normal limits

Other protocol-defined inclusion/exclusion criteria may apply.