ICONIC: ivermectin + immune checkpoint inhibition in solid tumors (NCT07487805) [Pending]
University of Florida Phase II randomized study comparing intermediate- vs high-dose ivermectin with standard immune checkpoint inhibitor…
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Curated updates on clinical trials, peer-reviewed papers, and reputable coverage related to compounds discussed on this site for cancer and other serious diseases. This page is informational only — not medical advice, diagnosis, or a treatment recommendation. Always consult a qualified healthcare professional. No entry implies proven efficacy.
University of Florida Phase II randomized study comparing intermediate- vs high-dose ivermectin with standard immune checkpoint inhibitor…
Systematic review synthesizing preclinical and clinical evidence for mebendazole in primary brain tumours, including CNS penetrance and m…
VA-sponsored Phase I/II randomized study adding hydroxychloroquine versus placebo around trans-arterial chemoembolization (TACE) to targe…
Published Phase I dose-escalation of hydroxychloroquine plus Suba-itraconazole in men with biochemical relapse of prostate cancer after l…
PubMed/PMC case series of three patients with advanced breast, prostate, or melanoma who self-administered fenbendazole alongside other n…
Large randomized Phase III trial (estimated n≈474) comparing standard palbociclib + endocrine therapy versus arms adding hydroxychloroq…
Phase I dose-escalation study of IV methylene blue for cytokine release syndrome (CRS) or ICANS after CAR-T or bispecific antibodies. Evi…
ASCO policy/news item summarizing clinical guidance that ivermectin and fenbendazole should not be recommended for cancer treatment outsi…
As of this curation scan, ClinicalTrials.gov queries for fenbendazole return parasitology/oxfendazole and unrelated studies — not registered human cancer treatment trials of fenbendazole. Evidence level: registry observation; human oncology evidence remains preclinical/case-level.
Indiana University Phase II pilot of hydroxychloroquine for joint pain from aromatase-inhibitor–associated musculoskeletal symptoms in breast cancer patients (n≈35). Supportive-care focus. Evidence level: planned pilot; no results yet.
Rutgers Phase I dose-finding study of hydroxychloroquine added to oral decitabine/cedazuridine and venetoclax in acute myeloid leukemia (n≈18). Evidence level: planned early Phase I; no results yet.
University of Florida Phase II randomized study comparing intermediate- vs high-dose ivermectin with standard immune checkpoint inhibitors in adults with solid tumors. Evidence level: planned interventional trial; no results yet.
Mouse study reporting that nutritional ketosis may enhance therapeutic effects of mebendazole combined with CPI-613 (devimistat) in juvenile high-grade glioma models. Evidence level: preclinical animal only.
Preclinical evaluation of mebendazole in paediatric low-grade glioma models showing antiproliferative and antimigratory effects. Evidence level: preclinical — complements existing pediatric glioma trial listings.
Systematic review synthesizing preclinical and clinical evidence for mebendazole in primary brain tumours, including CNS penetrance and multimodal anticancer mechanisms. Evidence level: systematic review — summarizes, does not replace, controlled trials.
Cell-line study reporting that ivermectin modulates ER, HER2, and TGF-β signaling in ER-positive and endocrine-resistant breast cancer models. Evidence level: preclinical in vitro only.
In vitro study reporting tumor-growth suppression by ivermectin in gastric cancer cell lines and patient-derived organoids. Evidence level: preclinical cell/organoid work — not human clinical efficacy.
Computational-experimental study reporting synergistic responses to sorafenib plus hydroxychloroquine in KRAS-mutant breast cancer models. Evidence level: preclinical — not a clinical trial result.
VA-sponsored Phase I/II randomized study adding hydroxychloroquine versus placebo around trans-arterial chemoembolization (TACE) to target ischemia-induced autophagy dependence in unresectable HCC (n≈93). Evidence level: ongoing interventional; results pending.
Recruiting Phase II combining MEK inhibitor tunlametinib, PD-1 antibody pucotenlimab, and hydroxychloroquine in metastatic NRAS-mutant melanoma. Evidence level: planned/ongoing Phase II; no results yet.
Intravesical nanoformulation combining fenbendazole with photothermal therapy reported to trigger ferroptosis and dual immunotherapy signals in bladder cancer models. Evidence level: preclinical nanomedicine — not a registered human cancer trial of fenbendazole.
Published Phase I dose-escalation of hydroxychloroquine plus Suba-itraconazole in men with biochemical relapse of prostate cancer after local therapy (HITMAN-PC), exploring lysosomal/cholesterol biology without ADT. Evidence level: Phase I published human data; efficacy not established.
Randomized double-blind placebo-controlled early Phase I proof-of-principle study of oral hydroxychloroquine before prostatectomy, measuring autophagy markers (p62, LC3-II, NBR1) in tumor tissue (n≈20). Evidence level: biomarker-focused pilot; not an efficacy trial.
Small single-arm pilot (enrolling by invitation) of topical methylene blue dressing for advanced/inoperable squamous cell carcinoma not suitable for radiotherapy. Evidence level: tiny pilot; preliminary only.
Recruiting Phase II focused on autophagy maintenance strategies including hydroxychloroquine in ovarian cancer. Evidence level: ongoing Phase II; efficacy unknown.
Laboratory investigation of ivermectin alone and with doxorubicin in OSCC models, reporting synergistic cytotoxicity signals. Evidence level: in vitro preclinical — not clinical trial data.
Pilot of topical methylene blue (0.5 mg/mL) every other day for one month in morpheaform, infiltrative, or noduloulcerative basal cell carcinoma not suitable for surgery/radiotherapy. Evidence level: small pilot; efficacy unproven.
Early Phase I pilot of local methylene blue injection as a potential radiosensitizer with concurrent radiation in locoregional recurrent head and neck squamous cell carcinoma (n≈10). Evidence level: small feasibility/safety pilot; efficacy unknown.
Mouse study combining fenbendazole with diisopropylamine dichloroacetate (DADA); authors report synergistic tumor growth inhibition and call for clinical study. Evidence level: preclinical animal only.
Preclinical study linking fenbendazole to pyroptosis via HK2/caspase-3/GSDME signaling in breast cancer cell models. Evidence level: preclinical in vitro — not human trial evidence.
Peer-reviewed review summarizing limited human clinical evidence for ivermectin as cancer therapy, noting widespread off-label interest and the need for well-designed trials. Evidence level: narrative review; not new clinical efficacy data.
PubMed/PMC case series of three patients with advanced breast, prostate, or melanoma who self-administered fenbendazole alongside other non-chemotherapy interventions; authors describe remissions but stress confounding and lack of controlled evidence. Evidence level: anecdotal case series — cannot establish causality.
Large randomized Phase III trial (estimated n≈474) comparing standard palbociclib + endocrine therapy versus arms adding hydroxychloroquine (with standard- or high-dose palbociclib). Evidence level: confirmatory-design trial planned; no results yet.
Phase I dose-escalation study of IV methylene blue for cytokine release syndrome (CRS) or ICANS after CAR-T or bispecific antibodies. Evidence level: early Phase I safety/preliminary efficacy; not an approved cancer treatment indication.
ASCO policy/news item summarizing clinical guidance that ivermectin and fenbendazole should not be recommended for cancer treatment outside well-designed clinical trials, and advising routine conversations about supplements. Evidence level: professional society guidance — not a trial result.
ASCO abstract (meeting ID 250156 / JCO e13146): combination described as safe/well tolerated in early accrual; among 8 evaluable patients, 1 PR, 1 SD, 6 PD (median PFS 2.5 months; 4-month clinical benefit rate 37.5%). Evidence level: conference abstract, small n, preliminary — not peer-reviewed full results.
Study reporting fenbendazole activity against cervical cancer cells and cancer stem cell–like populations in preclinical models. Evidence level: preclinical — not approved human cancer therapy.
Preclinical work exploring mebendazole combined with radiation therapy in an IDH-mutant glioma context. Evidence level: preclinical — not human efficacy data.
Recruiting Phase II comparing a hydroxychloroquine-containing multi-drug regimen versus regorafenib in refractory RAS-mutant metastatic colorectal cancer. Evidence level: ongoing comparative Phase II; no results yet.
Open-access Phase I report: HCQ was tolerable; RP2D 800 mg/day with continuous palbociclib 75 mg/day + letrozole; best responses included 2 partial responses and 11 stable disease among 14 evaluable patients. Evidence level: single-arm Phase I; larger randomized studies needed.
In vitro and xenograft mouse work reporting proliferation inhibition, apoptosis/mitotic catastrophe signals, and oral fenbendazole effects on tumor growth in ovarian cancer models. Evidence level: preclinical — not a human trial.
Phase Ib/II in 29 HR+/HER2− patients after first-line palbociclib failure. RP2D: HCQ 600 mg BID + palbociclib 200 mg daily. Reported ORR 41.4% in all enrolled; grade ≥3 neutropenia/leukopenia ~25%. Evidence level: single-arm Phase Ib/II; needs randomized confirmation.
Recruiting Phase I/II randomized study of hydroxychloroquine sulfate tablets versus placebo for prevention/treatment of radiation-induced oral mucositis and dysgeusia (n≈158). Supportive-care indication, not antitumor efficacy. Evidence level: ongoing supportive-care trial.
Review of preclinical anticancer activity, oral pharmacokinetics, and toxicity; emphasizes fenbendazole is not FDA/EMA-approved for human cancer and that clinical trials are needed. Evidence level: narrative review.
Recruiting study evaluating hydroxychloroquine pretreatment before radiotherapy and chemotherapy in advanced nasopharyngeal carcinoma. Evidence level: early interventional; not standard of care.
MD Anderson Phase I/II of autophagy inhibition with HCQ added to continuous low-dose palbociclib + letrozole. Completed; Phase I results published in npj Breast Cancer (see companion news entry). Evidence level: early-phase completed trial.
Completed Dana-Farber Phase II of ERK inhibitor LY3214996 alone or with hydroxychloroquine in advanced pancreatic cancer. Evidence level: completed Phase II; review registry/publications for outcomes before drawing conclusions.
Institutional patient-facing summary of the recruiting Phase I/II study pairing oral ivermectin with investigational anti–PD-1 balstilimab (NCT05318469). Notes experimental use in this setting. Evidence level: trial information page, not outcome data.
Cedars-Sinai Phase I/II study of oral ivermectin with immune checkpoint inhibitors in metastatic triple-negative breast cancer. Estimated primary completion Oct 2026. Evidence level: ongoing interventional trial; efficacy not established.
Phase II study comparing leflunomide versus MEK inhibitor plus hydroxychloroquine (± bevacizumab) in refractory RAS-mutant colorectal, pancreas, or lung cancer (n≈20). Evidence level: small ongoing Phase II; interpret cautiously.
Phase II study of hydroxychloroquine added to FOLFIRI and bevacizumab in metastatic colorectal cancer enriched for drug-tolerant persister (DTP) biology. Status: active, not recruiting. Evidence level: ongoing Phase II.
Registry record for the Gong et al. Phase Ib/II combination of hydroxychloroquine with escalated palbociclib doses in advanced HR+/HER2− disease after CDK4/6 inhibitor failure. Evidence level: see published Med paper for outcomes.
Mayo Clinic Phase III comparing methylene blue oral rinse versus standard-of-care mouthwash for oral mucositis pain in patients with solid or hematologic malignancies (n≈49). Supportive-care pain indication. Evidence level: completed Phase III supportive-care trial; not an antitumor study.
Preclinical work improving fenbendazole solubility via PLGA nanoparticles; intravenous nanoparticles reduced tumor weight in cell-line and patient-derived xenografts where oral fenbendazole did not. Evidence level: preclinical formulation study.
Northwestern Phase II of mitochondrial metabolism inhibitor CPI-613 (devimistat) plus hydroxychloroquine with 5-fluorouracil or gemcitabine in advanced chemorefractory solid tumors including biliary, colorectal, GEJ, and lung adenocarcinoma (n≈94). Evidence level: ongoing Phase II; efficacy not established.
Mouse study reporting that ivermectin reduced lung metastasis associated with neutrophil extracellular traps without reducing primary tumor growth. Evidence level: preclinical animal study — not human trial data.
Laboratory study of methylene blue photodynamic therapy reducing cell viability and MMP-9 in oral carcinoma and leukoplakia cell models. Evidence level: in vitro preclinical.
Completed Johns Hopkins Phase I safety study of mebendazole in children/young adults with recurrent medulloblastoma or high-grade glioma / DIPG. Evidence level: completed Phase I; registry results should be reviewed separately.
Randomized double-blind placebo-controlled report (n=40) claiming higher overall response (65% vs 10%) and longer PFS (9.25 vs 3 months) with mebendazole 500 mg BID added to bevacizumab/FOLFOX4. Evidence level: small single-center RCT; needs independent replication before practice change.
Medical University of South Carolina Phase I/II testing oral combinations of hydroxychloroquine with metformin, sirolimus, and optionally dasatinib/nelfinavir in advanced solid tumors and recurrent prostate cancer (n≈76). Evidence level: ongoing multi-drug early-phase study; efficacy unknown.
Phase I combination of hydroxychloroquine and chlorphenesin carbamate with mFOLFIRINOX in pancreatic cancer. Status: active, not recruiting. Evidence level: early Phase I safety study.
Phase II University of Pennsylvania trial testing abemaciclib alone vs abemaciclib + HCQ to target disseminated tumor cells / minimal residual disease after primary breast cancer therapy. Evidence level: ongoing randomized Phase II; results pending.
Fox Chase early Phase I neoadjuvant study of paricalcitol, hydroxychloroquine, and losartan (PHL) to modify stroma in resectable pancreatic ductal adenocarcinoma (n≈20). Status: active, not recruiting. Evidence level: small early-phase stroma-focused study.
In vitro study reporting dose-dependent growth inhibition, ROS-mediated mitochondrial apoptosis, and S-phase arrest in colorectal cancer cell lines. Evidence level: preclinical cell work only — not human clinical efficacy.
Phase II multi-arm study exploring avelumab or hydroxychloroquine with or without palbociclib to clear dormant breast cancer cells. Evidence level: ongoing interventional; efficacy not established.
Completed Johns Hopkins Phase I dose-finding study of mebendazole with temozolomide after chemoradiation. Protocol notes preclinical fenbendazole observations led investigators to select mebendazole for human testing. Evidence level: completed Phase I; not proof of clinical benefit.
Penn Phase II of MEK inhibitor binimetinib plus hydroxychloroquine in advanced KRAS-mutant non-small cell lung cancer (n=9 enrolled). Evidence level: completed small Phase II; review registry/publications before drawing conclusions.
Small uncontrolled case series (often cited as Stanford/Chiang) describing radiographic responses in genitourinary cancer patients who took fenbendazole with concurrent or recent standard therapies. Evidence level: anecdotal; concurrent treatments confound attribution.
HonorHealth Phase II neoadjuvant study adding hydroxychloroquine to nab-paclitaxel, gemcitabine, and cisplatin in untreated resectable, borderline, or locally advanced pancreatic adenocarcinoma (n≈19), focusing on CA19-9 normalization. Evidence level: completed small Phase II.
Phase I/II of checkpoint blockade plus HCQ in advanced melanoma. Status: active, not recruiting. Evidence level: early-phase combination; await published outcomes.
Emory Phase II of vitamin D analog paricalcitol and hydroxychloroquine combined with gemcitabine and nab-paclitaxel in advanced/metastatic pancreatic adenocarcinoma (n≈10). Evidence level: completed small Phase II.
Randomized Phase II preoperative study: HCQ arm improved pathologic and CA19-9 responses but did not improve overall or recurrence-free survival. Evidence level: randomized Phase II — mixed endpoints; no survival benefit shown.
Utah Phase I dose-escalation of ERK inhibitor ulixertinib (BVD-523) with fixed-dose hydroxychloroquine in advanced MAPK-mutated gastrointestinal adenocarcinomas (n≈16). Evidence level: completed Phase I; early safety/dose data only.
Pediatric Brain Tumor Consortium Phase I/II adding hydroxychloroquine to dabrafenib and/or trametinib in children with recurrent/progressive low- or high-grade glioma with BRAF aberration (n≈57). Status: active, not recruiting. Evidence level: ongoing early-phase pediatric combination; efficacy not established.
Norwegian Phase I of proteasome inhibitor carfilzomib plus hydroxychloroquine (with dexamethasone) in relapsed/refractory multiple myeloma (n≈19). Evidence level: completed Phase I; check publications for outcomes.
MD Anderson Phase I of binimetinib plus hydroxychloroquine in KRAS-mutant metastatic pancreatic adenocarcinoma (n≈34). Evidence level: completed Phase I dose-finding; not proof of clinical benefit.
Phase 2 randomized clinical trial in metastatic/advanced pancreatic cancer: higher response rate with HCQ but no overall survival benefit. Evidence level: randomized Phase II — response ≠ survival benefit.
Tanta University Phase III–labeled randomized study of mebendazole added to FOLFOX + bevacizumab in colorectal cancer. Evidence level: registry lists recruiting; interpret published companion results cautiously and independently.
MD Anderson Phase I/II of hydroxychloroquine with gemcitabine and docetaxel in recurrent or refractory osteosarcoma (n≈31). Status: active, not recruiting. Evidence level: early-phase sarcoma combination; await published outcomes.
Phase 2a TDM-guided study of mebendazole in advanced gastrointestinal cancer or cancer of unknown origin. Terminated for lack of effect (n=11). Evidence level: small terminated early-phase study — negative signal in this setting.
Phase II pilot of hydroxychloroquine, everolimus, or the combination for prevention of recurrent breast cancer (stage IIB+). Status: active, not recruiting. Evidence level: prevention-focused early Phase II.
Penn Phase I/II of hydroxychloroquine added to dabrafenib and trametinib in advanced BRAF-mutant melanoma (n≈50). Companion imaging study NCT05279027 exists. Evidence level: completed early-phase combination; review publications for final outcomes.
Completed early study of hydroxychloroquine alone in heavily pretreated metastatic pancreatic cancer; short median PFS/OS reported historically. Evidence level: older single-agent study — limited activity as monotherapy.
Phase I/II Northwell/Cohen Children's study of oral mebendazole plus standard regimens in pediatric low- and high-grade gliomas / DIPG. Status: active, not recruiting. Evidence level: early-phase combination safety/efficacy study.
Phase II of erlotinib with or without HCQ in chemo-naive advanced NSCLC with EGFR mutations. Status listed active, not recruiting on registry scans. Evidence level: older Phase II combination; check current publications for final outcomes.
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Reminder: Links and summaries are for exploration only. They do not imply safety, efficacy, or endorsement. Prefer peer-reviewed full papers and ClinicalTrials.gov records over headlines. Do your own due diligence and speak with a healthcare provider.