Pilot Study of Sonidegib and Buparlisib in Treating Patients With Advanced or Metastatic Basal Cell Carcinoma

Carcinoma, Basal Cell Clinical Trial

Official title: An Open-Label Pilot Study to Evaluate the Efficacy and Safety of a Combination Treatment of Sonidegib (LDE225) and Buparlisib (BKM120) For the Treatment of Advanced Basal Cell Carcinomas

Status Terminated

Clinical Trial Summary

This pilot trial studies how well sonidegib and buparlisib work in treating patients with basal cell carcinoma that has spread to other places in the body. Sonidegib and buparlisib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Clinical Trial Description

PRIMARY OBJECTIVES:

Estimate the overall response rate (ORR) of sonidegib (erismodegib) in combination with buparlisib (hereby referred to as "LB therapy") for patients with locally advanced or metastatic basal cell carcinoma (BCC) in Smoothened inhibitor-naive patients (Cohort 1) and those whose disease is refractory or relapsed on Smoothened inhibitor monotherapy (Cohort 2).

NOTE: This study does not compare the treatment effect between these 2 dissimilar participant groups.

SECONDARY OBJECTIVES:

• Estimate the median duration of response, on or after LB therapy.

• Assess the safety and tolerability of LB therapy.

• Assess the histopathologic effect of LB therapy in tumor biopsies obtained at baseline and following 12 weeks of treatment.

• Assess the effect of LB therapy on gene expression including Hedgehog pathway and phosphatidylinositol 3-kinase (PI3K) pathways.

• Assess correlation between gene mutations in Smoothened, suppressor of fused homolog (Sufu), patched (PTCH), glioma-associated oncogene homolog (Gli)1, 2 and gene expression profiles and response to LB therapy.

OUTLINE:

Patients receive sonidegib orally (PO) once daily (QD) and buparlisib PO QD on days 1 to 28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30 days and then every 3 months. ;

Related Conditions & MeSH terms

• Basal Cell Nevus Syndrome
• Carcinoma
• Carcinoma, Basal Cell
• Recurrent Skin Cancer
• Skin Neoplasms

• NCT number: NCT02303041
• Study type: Interventional
• Source: Stanford University
• Status: Terminated
• Phase: Phase 2
• Start date: February 2015
• Completion date: June 2017