Clinical Trial: Secured Access to Nivolumab for Adult Patients With Selected Rare Cancer Types

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Secured Access to Nivolumab for Adult Patients With Selected Rare Cancer Types

Brief Summary: This is a Phase 2, non-randomised, open-label, multicentric study to investigate the efficacy and safety of nivolumab monotherapy in 5 cohorts of patients with specific rare cancers who have unresectable locally advanced or metastatic disease, which is resistant or refractory to standard therapy, or for which standard therapy does not exist, or is not considered appropriate, and for which no other experimental treatment options are available.

Detailed Summary:

The study plans to enrol up to 250 patients in total with between 20 and 50 patients assigned to each cohort according to indication, as follows:

  • Cohort 1: Non-clear cell RCC
  • Cohort 2: Rare head and neck cancer
  • Cohort 3: Rare skin cancer
  • Cohort 4: MSI-nonCRC
  • Cohort 5: Penile cancer

The study will use a two-stage Bayesian enrichment design. The first stage treats all patients from the different cohorts with the investigational product and identifies possibly sensitive indications. The second stage will compare outcomes among subsets of patients in the identified cohorts to distinguish between subpopulations of patients who may benefit from the treatment and patients for whom there is no evidence of efficacy.


Sponsor: UNICANCER

Current Primary Outcome: Objective response rate [ Time Frame: measured at the first scheduled disease assessment following study treatment initiation (Day 84, ± 7 days) ]

ORR will be assessed per cohort by an IRC according to RECIST v1.1.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Progression-free survival [ Time Frame: From date of inclusion until the date of first documented progression or date of death from any cause, whichever comes first, assessed up to 36 months ]
    Assessed according to RECIST v1.1
  • Overall survival [ Time Frame: From date of inclusion until the date of death from any cause, assessed up to 36 months ]
  • Best response [ Time Frame: From inclusion up to 36 months ]
    Assessed according to RECIST v1.1
  • Response duration [ Time Frame: from first observation of objective response until date of first documented progression or date of death from any cause, whichever comes first, assessed up to 36 months ]
    Assessed according to RECIST v1.1
  • Time to response [ Time Frame: from inclusion first observation of objective response, assessed up to 36 months ]
    Assessed according to RECIST v1.1
  • Frequency and severity of adverse events [ Time Frame: from inclusion until 100 days after last dose of investigational product ]
    assessed according to the NCI-CTCAE v4
  • Objective response rate in subgroups of subjects with high versus low expression (cutoff set at the median for the population measured) of different immune markers (PD-L1, CD4+, FOXP3+, Fas-L, OX40, VEGF, CD31; CD34) [ Time Frame: measured at the first scheduled disease assessment following study treatment initiation (Day 84, ± 7 days) ]
    ORR will be assessed per cohort by an IRC according to RECIST v1.1.
  • Progression-free survival in subgroups of subjects with high versus low expression (cutoff set at the median for the population measured) of different immune markers (PD-L1, CD4+, FOXP3+, Fas-L, OX40, VEGF, CD31; CD34) [ Time Frame: From date of inclusion until the date of first documented progression or date of death from any cause, whichever comes first, assessed up to 36 months ]
    Assessed according to RECIST v1.1.
  • Overall survival in subgroups of subjects with high versus low expression (cutoff set at the median for the population measured) of different immune markers (PD-L1, CD4+, FOXP3+, Fas-L, OX40, VEGF, CD31; CD34) [ Time Frame: From date of inclusion until the date of death from any cause, assessed up to 36 months ]


Original Secondary Outcome: Same as current

Information By: UNICANCER

Dates:
Date Received: January 2, 2017
Date Started: June 2017
Date Completion: December 2023
Last Updated: May 3, 2017
Last Verified: May 2017