A Phase 2/3 Study of Indoximod or Placebo Plus Pembrolizumab or Nivolumab in Subjects With Unresectable or Metastatic Melanoma


About this study

This is an adaptive clinical trial that includes a dose escalation phase followed by a randomized controlled Phase 3 trial. The purpose of the dose escalation phase is to establish the Phase 3 dose of indoximod in combination with pembrolizumab or nivolumab in subjects with unresectable or metastatic melanoma and evaluate PK. Subsequently the efficacy, safety and tolerability of indoximod plus pembrolizumab or nivolumab versus placebo plus pembrolizumab or nivolumab will be studied in subjects with unresectable or metastatic melanoma in the Phase 3 portion of the trial.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Have histologically- or cytologically-confirmed unresectable stage III or stage IV melanoma not amenable to local therapy
  • At least one radiologically measurable lesion as per RECIST 1.1
  • Have documentation of V600-activating BRAF mutation status or consent to BRAF V600 mutation testing during the screening period.
  • ECOG performance status 0 or 1
  • Ability to ingest oral medications

Exclusion Criteria:

  • Has Ocular Melanoma
  • Has received prior systemic treatment for unresectable or metastatic melanoma (except BRAF directed therapy).
  • Has received prior therapy with an anti-PD-1, anti-PD-L1, anti-PD-L2, anti-CD137, or IDO1 inhibitor or any other antibody or drug specifically targeting checkpoint pathways other than anti-CTLA-4 which is permitted in the adjuvant setting.
  • Has received prior adjuvant therapy, monoclonal antibody or an investigational agent or device within 4 weeks or 5 half-lives (whichever is longer)
  • Has received prior radiotherapy within 2 weeks of therapy.
  • Is pregnant or breast-feeding or expecting to conceive or father children within the projected duration of the study, starting with the screening visit through 120 days after the last dose of study treatment.
  • Patients who have active, chronic, or on active treatment for Hep B or Hep C are excluded.
  • Patients who have received a live attenuated vaccine with in 30 days of randomization
  • Patients who take a MAOI or drug with significant MAOI activity
  • Patients who have had Serotonin Syndrome after receiving a serotonergic drug

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Robert McWilliams, M.D.

Closed for enrollment

Contact information:

Cancer Center Clinical Trials Referral Office

(855) 776-0015

More information


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