NCT02303041

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

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
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Anne Chang · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

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

Interventions

Type	Name	Description
DRUG	Buparlisib	Administered orally at starting dose of 80 mg/day
DRUG	Sonidegib	Administered orally at starting dose of 200 mg/day

Timeline

• Start date: 2015-02-01
• Primary completion: 2017-04-01
• Completion: 2017-06-01
• First posted: 2014-11-27
• Last updated: 2019-01-29
• Results posted: 2019-01-29

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study