Are there ongoing or planned randomized trials testing ivermectin in cancer and what are their designs?
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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].