What clinical trials are currently recruiting that test ivermectin with immunotherapy in cancer patients?
Executive summary
A single, actively recruiting clinical program testing ivermectin together with immune checkpoint blockade in cancer patients has emerged in the published record: a Phase I/II trial (ClinicalTrials.gov identifier NCT05318469) combining oral ivermectin with the investigational PD‑1 antibody balstilimab (with pembrolizumab also listed in some arms) for metastatic triple‑negative breast cancer (mTNBC) [1] [2] [3]. The trial is early‑phase, driven by preclinical signals that ivermectin can “turn cold tumors hot,” and is intended to assess safety and preliminary efficacy rather than to establish definitive benefit [4] [2].
1. The recruiting trial: what it is and where it stands
The trial registered as NCT05318469 is described in multiple sources as a Phase I/II study evaluating ivermectin in combination with balstilimab (and with expansion cohorts and pembrolizumab referenced) in patients with metastatic triple‑negative breast cancer, with treatment given on three‑week cycles and follow‑up planned after treatment cessation [1] [2] [5]. Provider summaries and an ASCO abstract identify the study team and sites including City of Hope and Cedars‑Sinai and list typical Phase I/II eligibility such as adults with ECOG 0–1 and life expectancy beyond three months [3] [5] [6]. An NCI clinical trial listing also references the ivermectin/pembrolizumab combination in its database entries [7].
2. Why investigators launched this specific combination
The trial’s rationale rests on preclinical data showing that ivermectin can induce immunogenic cancer cell death, increase intratumoral CD8+ T‑cell infiltration and synergize with anti‑PD‑1 therapy in breast cancer models, prompting investigators to test whether that biology translates into safety and pharmacodynamic signals in humans when paired with immune checkpoint inhibitors [4] [2]. Multiple reviews and commentaries emphasize that these are compelling laboratory observations but underscore that clinical evidence remains scarce and that current studies are exploratory [8] [9].
3. What the trial aims to show and its limitations
As a Phase I/II program, the primary goals are safety, dosing and early evidence of tumor response or immune modulation rather than definitive survival benefit; the study protocol outlines ivermectin dosing schedules across 21‑day cycles with immune checkpoint infusions on Day 1 and follow‑up after treatment completion [6] [2]. Experts surveyed in Cancer News note that only this one active Phase I/II trial is known in this specific ivermectin + immunotherapy space and that results will be preliminary—important for hypothesis‑generation but insufficient to change standard care without later randomized, larger trials [10] [8].
4. The larger context: few human data, active preclinical interest
Despite active laboratory and animal research supporting ivermectin’s multi‑faceted anticancer effects and immune‑modulating activity, systematic reviews and recent literature stress that human clinical trials are very limited and that large‑scale randomized controlled trials confirming benefit do not yet exist [8] [11] [9]. Some reporting and trial registries even suggest just one or two registered oncology studies; however, the reliable, directly relevant recruiting study documented across clinicaltrials.gov, Gateway for Cancer Research and ASCO materials remains the NCT05318469 Phase I/II trial in mTNBC [1] [2] [3].
5. What to watch for next and competing perspectives
The field will watch early safety signals, pharmacodynamic biomarkers (evidence of tumor immune infiltration), and small‑signal efficacy readouts from NCT05318469, which some sources cited as expected to report preliminary results by 2026, but investigators caution that preclinical promise often fails to translate and that ivermectin’s prior public controversies (e.g., COVID‑19) complicate public interpretation of early oncology data [10] [6]. Independent voices—systematic reviewers and oncology experts—stress that until well‑controlled human data appear, ivermectin should be considered experimental in cancer and explored only within regulated clinical trials [8] [11].