Are there ongoing or planned randomized trials testing ivermectin in cancer and what are their designs?
Executive summary
Two registered clinical studies testing ivermectin in cancer exist and are at early phase stages; most detailed public reporting centers on a Phase I/II trial (NCT05318469) combining ivermectin with the anti–PD‑1 antibody balstilimab (also reported with pembrolizumab) in metastatic triple‑negative breast cancer (mTNBC) [1] [2] [3]. The literature remains dominated by preclinical evidence (cell lines, animal work) and reviews that call clinical data “scarce,” with no large randomized Phase III trials shown in the available reporting [4] [5] [6].
1. Early human testing: one clear Phase I/II immunotherapy combination
The clearest, repeatedly cited clinical study is a Phase I/II trial evaluating ivermectin plus balstilimab in metastatic triple‑negative breast cancer (ClinicalTrials.gov identifier NCT05318469) that is described in a Gateway for Cancer Research synopsis and an ASCO abstract; the trial tests safety and preliminary efficacy and includes an expansion cohort for PD‑L1–negative TNBC [1] [2] [3]. Trial descriptions note the concept that ivermectin may convert “cold” tumors to “hot” by increasing T‑cell infiltration, which is the biological rationale for combining it with checkpoint blockade [1].
2. Trial design details reported in abstracts and registries
Public summaries and meeting abstracts indicate this is a Phase I dose‑finding/escalation followed by a Phase II expansion to assess activity of the combination; some press/summary reporting lists dosing schedules used in the study (e.g., ivermectin given orally on intermittent days of a 21‑day cycle with balstilimab or pembrolizumab given intravenously on Day 1) and a planned treatment duration of up to ~2 years or until progression/toxicity [7] [1]. ClinicalTrials.gov holds a record for NCT05318469 but the public registry entry is the authoritative source for precise eligibility, endpoints and status [3].
3. Scope: mostly early‑phase, small, non‑randomized or randomized?
Available reporting frames the trial as Phase I/II safety and preliminary efficacy testing; the sources do not describe a completed randomized, large‑scale RCT showing benefit. Reviews and commentaries emphasize that clinical trial literature is scarce and that large randomized trials confirming benefit in humans are lacking [4] [5]. Patient‑facing summaries from 2025 note that trial abstracts reported “no real effect” in at least one early report, underscoring that efficacy is unproven in humans so far [8] [2].
4. Preclinical rationale driving the trials
Preclinical work—cell culture and animal models—drives the clinical interest: ivermectin shows antiproliferative, pro‑apoptotic and immune‑modulating effects in multiple cancer models and can regulate pathways such as Wnt/β‑catenin, PAK1/Akt and others; synergy with other agents (e.g., recombinant methioninase in pancreatic cell lines) is reported in lab studies [9] [10] [6]. Reviews caution that these effects have largely been shown in vitro or in animals and do not yet translate into definitive clinical benefit [4] [5].
5. Conflicting signals and caution from clinicians and fact‑checks
Fact‑checking outlets and oncologists stress that preclinical promise is not proof of human benefit: Associated Press reporting cites investigators preparing trials but notes “no reports of clinical trials that yielded successful results in humans” and warns against claims that ivermectin is a withheld cancer cure [11]. Oncology commentaries and specialty journals urge caution and note very limited human data and the risk of misinformation steering patients from proven therapies [4] [12].
6. What is not shown in the sources
Available sources do not describe completed randomized Phase III trials demonstrating clinical benefit, nor do they provide full peer‑reviewed phase II/III results showing survival advantage; they do not list multiple randomized studies across tumor types—reporting is concentrated on one registered Phase I/II immunotherapy combination and a handful of preclinical/early reports [4] [1] [2].
7. What to watch next and implications for patients
Watch for full results from NCT05318469 in peer‑reviewed form and for any registration of randomized confirmatory trials; current guidance in oncology reviews urges that ivermectin remains investigational for cancer and should not replace standard care [1] [4] [12]. Because the strongest human data are early‑phase and mixed, clinicians and patients should treat reports of ivermectin as promising preclinical science that requires rigorous randomized testing before clinical adoption [4] [11].
Limitations: this analysis relies only on the supplied sources; if you want up‑to‑date registry data, the ClinicalTrials.gov record NCT05318469 is the primary registry to consult for trial status and protocol details [3].