Which registered clinical trials are testing ivermectin in combination with immune checkpoint inhibitors for cancer and what are their inclusion criteria?

1. The registered trials and their design

The clearest, repeatedly cited trial is NCT05318469, described as a phase I/II study at Cedars‑Sinai (investigator‑initiated IIT2022‑07) evaluating safety, tolerability and preliminary efficacy of oral ivermectin combined with an anti‑PD‑1 agent (balstilimab) in metastatic triple‑negative breast cancer (mTNBC), with mention of pembrolizumab in some site summaries and an expansion cohort for PD‑L1 negative tumors; the trial is listed on ClinicalTrials.gov and on Cedars‑Sinai’s trial portal and Gateway for Cancer Research materials ^{[1] [2] [4]}. The NCI database also lists a trial pairing ivermectin with pembrolizumab (NCI‑2022‑02421), but the source searched does not provide full eligibility or design text to confirm phase or specifics ^[3].

2. Who the trials are recruiting — core inclusion criteria

Across the Cedars‑Sinai / Gateway descriptions, eligibility centers on histologically confirmed metastatic triple‑negative breast cancer defined as ER and PR ≤10% with HER2 negative status by ASCO/CAP criteria, prior progression after one or two systemic therapies in the metastatic setting, adequate organ and cardiac function, and ECOG performance status typically 0–1; prior PD‑1/PD‑L1 therapy in the metastatic setting is generally an exclusion (neoadjuvant/adjuvant ICI allowed if last dose was ≥1 year before study start), and patients with previously treated, radiologically stable brain metastases without recent steroids may be eligible ^{[5] [6] [4] [2]}. Specific dose‑cohorts, cycle schedules, and precise lab thresholds are listed in the trial protocol record on ClinicalTrials.gov and institutional pages but are not fully reproduced in the reviewed reporting ^{[1] [2]}.

3. Why investigators are testing this combination — the scientific rationale

Preclinical publications and institutional press describe consistent mechanistic rationale: ivermectin induces immunogenic cancer cell death and increases intratumoral T‑cell infiltration, effectively converting “cold” tumors into “hot” ones and synergizing with anti‑PD‑1 therapy to produce tumor regressions in mouse models; these mouse studies reported statistically significant synergy and durable responses that justified human testing (Draganov et al. and related preclinical work) ^{[7] [8] [9]}. Trial descriptions explicitly cite those laboratory data as the impetus for the phase I/II effort at Cedars‑Sinai supported in part by Gateway for Cancer Research ^{[4] [10]}.

4. What early human data and safety signals exist so far

An ASCO abstract and interim summaries from the phase I/II program report that the ivermectin + balstilimab combination was generally tolerated with low‑grade rash, isolated grade‑3 anemia, and other manageable events, and reported a 4‑month clinical benefit rate of 37.5% in a small, heavily pretreated cohort—results characterized as encouraging but preliminary and warranting continued study (ASCO abstract citing NCT05318469; p1_s2). The broader clinical literature cautions that human evidence is still limited and no large randomized controlled trials have confirmed anticancer benefit of ivermectin in patients ^[11].

5. Limits, conflicts and alternative viewpoints

Reporting is consistent that the human evidence base remains early‑phase and small; systematic reviews warn against overinterpreting preclinical success because dosing, pharmacology and safety in cancer patients can differ from animal models ^{[11] [7]}. Some dissemination outlets emphasize affordability and promise ^{[10] [9]}, while funders and investigator‑initiated trial sponsors such as Gateway and Cedars‑Sinai have explicit interests in advancing the program—important context when appraising press accounts ^{[4] [2]}. The NCI listing ^[3] suggests additional activity but the sources reviewed do not supply full eligibility or design text for that entry.

6. Bottom line

At present the only clearly documented registered trial pairing ivermectin with an immune checkpoint inhibitor for cancer in the reviewed sources is the Cedars‑Sinai phase I/II study NCT05318469 (ivermectin + balstilimab with pembrolizumab mentions and PD‑L1 expansion), with defined inclusion criteria focused on metastatic triple‑negative breast cancer (ER/PR ≤10%, HER2 negative), prior metastatic therapy, adequate organ function, ECOG 0–1, and exclusion of recent metastatic PD‑1/PD‑L1 therapy; early safety signals are tolerable but efficacy data remain preliminary and no large randomized evidence yet supports routine clinical use ^{[1] [2] [6] [5] [11]}. Sources reviewed could not confirm additional registered randomized trials or full protocol details for the NCI entry, so further verification on ClinicalTrials.gov and institutional registries is recommended for investigators and patients seeking enrollment ^{[1] [3]}.