LDE225 in Patients With Advanced or Metastatic Hepatocellular Carcinoma and Child-Pugh A/B7 Cirrhosis

Completed · Phase 1

Conditions studied: Hepatocellular Carcinoma, Cirrhosis

In brief

The purpose of this study is to test the safety and determine the maximum safe dose of an experimental drug called LDE225 (hedgehog inhibitor) in people with liver cancer. We have identified hedgehog dysregulation as a novel mechanism for hepatocarcinogenesis and hepatic fibrosis/cirrhosis. Therefore, we hypothesize that the hedgehog inhibitor may be an ideal drug target for treating both hepatocellular carcinoma (HCC) and Child-Pugh A cirrhosis (CPA).

Key facts

Study ID

NCT02151864

Run by

Jason K. Sicklick, M.D.

People needed

9

Starts

2014-07-01

Expected to finish

2017-09-01

Last updated by the study team

2019-03-05

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age 18 years or older
• Pathological confirmation of advanced or metastatic HCC
• HCC not amenable to surgical resection, liver transplantation, chemoembolization, or ablation therapy
• Patients with Child-Pugh A and Child-Pugh B7 (if due to low albumin but not elevated INR or bilirubin) cirrhosis are allowed.
• Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
• At least one measurable site of disease (as defined by Response Evaluation Criteria in Solid Tumors)
• Patients with adequate bone marrow, liver and renal function
• Inability to tolerate first-line treatment with sorafenib
• Patient amenable to liver tumor biopsy

You may not qualify if…

• Child-Pugh B or Child-Pugh C cirrhosis
• Patients with known Gilbert's Syndrome
• Ongoing alcohol use or abuse defined as > an average of 2 alcoholic beverages daily
• Patients who have had major surgery within 4 weeks of initiation of study medication
• Patients with known brain metastases
• Patients with concurrent uncontrolled medical conditions that may interfere with their participation in the study
• Patients with known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)-related illness
• Patients with Hepatitis B and/or Hepatitis C infection are excluded if they are on any of the following viral suppressive agents: boceprevir (Victrelis), ribavirin (Rebetol, Ribatab, Ribasphere), telaprevir (Incivek)
• Patients unable to take oral drugs or with lack of physical integrity of the upper gastrointestinal tract or known malabsorption syndromes
• Patients who have previously been treated with systemic LDE225 or with other Hh pathway inhibitors
• Patients who have neuromuscular disorders or are on concomitant treatment with drugs that are recognized to cause rhabdomyolysis
• Patients who are planning on embarking on a new strenuous exercise regimen after initiation of study treatment
• Patients who have taken part in an experimental drug study within 4 weeks of initiating study treatment with LDE225
• Patients who are receiving other anti-neoplastic therapy concurrently or within 2 weeks of starting study treatment with LDE225
• Patients who are receiving any anti-coagulation or anti-platelet therapy
• Patients who are receiving treatment with medications known to be strong inhibitors or inducers of cytochrome P450 3A4 (CYP3A4/5) or drugs metabolized by cytochrome P450 2B6 (CYP2B6) or cytochrome P450 2C9 (CYP2C9) that have narrow therapeutic index, and that cannot be discontinued before starting study treatment with LDE225
• Impaired cardiac function or clinically significant heart disease
• Patients with contraindications for MRI and/or MRI contrast agents
• Pregnant or nursing (lactating) women
• Women of child-bearing potential must use highly effective contraception during the study and through 6 months after the final dose of study treatment
• Sexually active males who are unwilling to use a condom during intercourse while taking drug and for 6 months after stopping investigational medications and agree not to father a child in this period

Where it is running

• UCSD Moores Cancer Center — La Jolla, California, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.