Study of oral FGF401 in adult patients with liver cancer or other types of cancer

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 240

Inclusion Criteria

1. Patients (male or female) = 18 years of age
2. ECOG Performance Status = 1
3. Presence of at least one measurable lesion according to RECIST v1.1.
4. FGF401 single agent-Phase II, Group 3: Patients with confirmed positive expression of FGFR4 and KLB at pre-screening.
5. For HCC patients: the diagnosis must be made based on AASLD Guidelines with confirmed stage C advanced HCC (BCLC staging classification). Current cirrhotic status of Child-Pugh class A (5-6 points), with no encephalopathy and/or ascites.
6. FGF401 single agent Phase I: Patients with HCC or advanced solid tumors, who have progressed despite standard therapy or are intolerant of standard therapy, or for whom no standard therapy exists.
7. FGF401 single agent Phase 2: HCC patients previously treated with sorafenib for advanced HCC with documented disease progression during or after discontinuation of sorafenib treatment, or intolerance to sorafenib treatment.
8. FGF401 in combination with PDR001-PhaseI and Phase II: Advanced HCC patients who have received up to 2 previous lines of systemic
treatment and one treatment must have included sorafenib with documented disease progression during or after discontinuation of sorafenib treatment, or intolerance to sorafenib treatment.
9. FGF401 in combination with PDR001-Phase II-Stage 2: Documented FGF19 expression level and/or FGF19 pathway activation

Other protocol-defined inclusion criteria may apply.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 150
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 90

Exclusion Criteria

1. Previous treatment with a FGF19-FGFR4 targeting therapy.
2. For HCC patients in FGF401 single agent Phase II part: any previous systemic anti-cancer therapies other than sorafenib or any anti-cancer
therapy (including locoregional therapy) after disease progression during or after sorafenib treatment.
3. Ongoing active diarrhea requiring medications (e.g. BAS, loperamide)
4. Irritable bowel syndrome with signs/symptoms and requires medications
5. Symptomatic CNS metastases which are neurologically unstable or requiring increasing doses of steroids to control their CNS disease.
6. Patient having out of range laboratory values defined as:
• Hematology
Hemoglobin = 9 g/dL (SI Units: 90 g/L)
Platelet count < 75000/mm3
Absolute neutrophil count (ANC) < 1500/mm3
• Chemistry
Total bilirubin > 1.5 x ULN
AST and/or ALT > 3 x ULN
Serum creatinine > 1.5 x ULN and/or creatinine clearance < 40 mL/min
• Coagulation: PT > 4 seconds more than the ULN or INR > 1.7
7. Unable to stop any prohibited medications, including CYP1A2, CYP2C9 and CYP3A4/5 substrates with a narrow therapeutic index and known BSEP efflux transporter inhibitors.
8. Additional exclusion criteria for FGF401 and PDR001 combination:
- Impaired cardiac function
- History of liver or other organ transplantation
- Patients who discontinued prior anti-PD-1/PD-L1 therapy due to an anti PD-1/PD-L1-related toxicity

Other protocol-defined exclusion criteria may apply.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Related Trials