Are there any registered randomized controlled trials planned to test ivermectin in oncology beyond phase I/II combination studies?
Executive summary
There are no registered large-scale randomized controlled trials (phase III or higher) planned or listed in the provided reporting that test ivermectin as a standalone anticancer agent; the only clearly documented human studies are early-phase combination trials (phase I/II) pairing ivermectin with immune checkpoint inhibitors for metastatic triple-negative breast cancer (TNBC) [1] [2] [3]. Preclinical data motivate further testing, but the literature and trial registries in these sources show a gap between promising laboratory findings and any planned randomized phase III oncology trials [4] [5] [6].
1. What is actually being tested now: early‑phase combination trials, not randomized phase III studies
The dominant, documented clinical activity is a phase I/II trial (ClinicalTrials.gov NCT05318469) studying ivermectin given with PD‑1–directed immunotherapy (balstilimab/pembrolizumab variants) in metastatic TNBC; this is explicitly listed as phase I/II and designed to assess safety, dose and early signals of efficacy rather than provide definitive, randomized evidence [1] [2] [7] [3].
2. Why investigators went to the clinic: laboratory rationale but not proof of clinical benefit
Preclinical studies show ivermectin can increase tumor T‑cell infiltration and synergize with anti‑PD‑1 therapy in mouse models, providing a biologic rationale for combination trials [4]. Reviews and mechanistic papers call for rigorous clinical testing to establish dosing, formulation and therapeutic window before any definitive claims are possible [5] [8]. Those same reviews note the absence of large-scale randomized trials confirming benefit in humans [6].
3. What the reporting says about plans beyond phase I/II: none documented in the supplied sources
Multiple oncology news summaries and expert commentaries referenced in the dataset emphasize that even if phase I/II results look promising, a phase III randomized controlled trial would still be required and that no such large RCTs are documented in these sources; several articles explicitly state the need for phase III follow‑up and indicate no current phase III RCTs are registered in the material provided [9] [10] [11].
4. The misinformation and off‑label use backdrop that clouds interpretation
Coverage also highlights how public enthusiasm and social‑media narratives around ivermectin (and other antiparasitics) have outpaced clinical evidence, prompting oncologists to counsel caution; reviews warn about self‑medication risks and stress that human clinical evidence remains limited [6] [11] [12]. Some non‑peer outlets and practitioner blogs promote protocols and anecdotal outcomes that are not supported by registered randomized trials in the materials supplied [13].
5. Limits of the available reporting and what remains unknown
The assembled sources document phase I/II combination work and preclinical promise but do not list any registered randomized phase III trials planned or underway; however, absence from these sources is not definitive proof no RCTs exist anywhere—this analysis is restricted to the provided reporting and registries cited [1] [2] [3] [6].
6. Bottom line for the research landscape
Based on the trial registrations, conference abstracts and review articles provided, ivermectin in oncology has reached first‑in‑human combination testing (phase I/II) for TNBC but has not progressed to planned randomized controlled phase III testing in the documented materials; investigators and commentators in these sources uniformly call for larger, randomized trials if early signals justify them [1] [2] [9] [10] [3].