Nivolumab With Vismodegib in Patients With Basal Cell Nevus Syndrome

Conditions

• Basal Cell Nevus Syndrome

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Vismodegib — DRUG
  150 mg PO daily
• Nivolumab — DRUG
  480 mg IV every 4 weeks
• Ipilimumab — DRUG
  1 mg/kg IV every 6 weeks

Study Details

This is a single-arm, phase II study to assess the efficacy of combined SMO and PD-1 inhibition with Vismodegib (SMO inhibitor) and Nivolumab (anti-PD-1 antibody) in BCNS patients (target enrollment of 22 patients), with a primary endpoint of 18-month disease control rate. The purpose of this study is to test the hypothesis that Nivolumab and Vismodegib will improve the percentage of BCNS patients who achieve disease control (defined as total tumor burden \<50% of baseline) at 18 months from 50% to 80%. Baseline and on-treatment biopsies will be obtained to characterize the immune effects of combined SMO and PD-1 inhibition.

Key Dates

Start date

Jul 31, 2019

Status verified

Dec 2019

Primary completion

Dec 31, 2019

Completion

Feb 29, 2020

Study Design

Enrollment

0 participants (actual)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Nivolumab, Vismodegib, Ipilimumab
  Patients will receive a two week run-in of Vismodegib 150 mg PO daily followed by concurrent Nivolumab 480 mg IV every 4 weeks and Vismodegib 150 mg PO daily. In an exploratory fashion, patients will have the option to receive combination Ipilimumab 1 mg/kg IV every 6 weeks and Nivolumab 360 mg IV every 3 weeks at the time of disease progression.

Primary Outcome Measure

Disease control rate [ Time Frame: 18 months ]