Comparing key 2020–2026 clinical trials and high-value clinical datasets.
| Authors and Year | Indication/pathogen focus | Design & size | Route/dosing (high-level) | Key results (selected) | Critical notes | Evidence level (CEBM) | Strength of evidence |
|---|---|---|---|---|---|---|---|
| Petrovic Fabijan et al. (2020)[56] | Severe S. aureus infection incl. endocarditis/septic shock | Single-arm, non-comparative under expanded access; n = 13 | IV phage cocktail AB-SA01 adjunct to antibiotics | No adverse reactions reported; dosing rationale suggested for further trials | GMP-quality preparation and pre-specified safety primary outcome strengthen translational credibility within early-phase evidence; however, single-arm design and small n (13) preclude efficacy inference and limit PK/PD generalization; immune neutralization not assessed, leaving a relevant safety dimension uncharacterized; salvage population further restricts external validity. | Level 4—Single-arm non-comparative; safety/PK primary | Uncontrolled—structured |
| Pirnay et al. (2024)[10] | Difficult-to-treat infections, multiple pathogens | Retrospective observational; n = 100 BT cases (subset from > 1,000 requests) | Personalized products; various routes; often + antibiotics | Clinical improvement 77.2%; eradication 61.3%; eradication ~70% less likely without antibiotics; 15 adverse events (7 suspected ADR) resolved | Consecutive case ascertainment substantially mitigates the success bias typical of retrospective phage cohorts and strengthens face validity of pooled outcomes; linkage to QC and production metrics adds methodological transparency uncommon in this literature; however, retrospective observational design, patient heterogeneity, variable routes/products, and uncontrolled concomitant therapies preclude causal inference, particularly for the antibiotic synergy signal. | Level 4—Retrospective observational cohort | Uncontrolled—structured |
| Kim et al. (2024)[39] | uUTI due to E. coli with history of drug-resistant UTI | Randomized open-label dosing study; ITT n = 39 | Intraurethral + IV LBP-EC01 + TMP-SMX; multiple IV dose regimens tested | No serious AEs; 46% with AEs (more with higher IV dosing); rapid E. coli reduction and symptom resolution by day 10 in evaluable patients | Engineered phage with robust PK characterization and randomized dose-ranging design represents a methodological advancement over compassionate-use studies in the field; tolerability boundaries identified at higher systemic exposure provide actionable safety information for future trials; however, open-label design introduces observer bias risk for symptom resolution endpoints; absence of placebo or standard-of-care comparator limits efficacy inference; co-administration of TMP-SMX confounds attribution of microbiological response to phage activity specifically. | Level 2—Randomized uncontrolled phase II dose-ranging trial | Randomized uncontrolled—dose-ranging |
| Chan et al. (2025)[41] | CF adults with MDR/PDR P. aeruginosa | Compassionate cohort; n = 9 | Nebulized single phage or cocktails; receptor/trade-off selection strategy | Median sputum Pseudomonas decrease (104 CFU/mL); ppFEV1 improvement signal; no adverse events; microbiome not altered | Evolutionary trade-off phage selection and pre-specified multi-endpoint measurement (sputum density, ppFEV1, microbiome) represent methodological advances over earlier case series; however, compassionate-use design, small n (9), salvage population, and patient-specific regimens preclude efficacy inference. | Level 4—Compassionate-use case series | Uncontrolled, structured |
| Petersen et al. (2026)[7] | Gut E. coli colonization in healthy volunteers (decolonization rationale for HSCT risk reduction) | Randomized, placebo-controlled, double-blind; n = 36 | Oral BID × 7 days; escalating doses (108–1012 PFU/dose) | No grade 3–4 AEs; no SAEs in treated groups; phage recovered in stool; microbiome stable; 78% reduction in E. coli vs placebo at day 14 (NS) | Randomized placebo-controlled double-blind design with healthy-volunteer cohort and pre-specified safety, PK, and microbiome endpoints represents the most methodologically rigorous phage therapy trial design to date in the field; CRISPR-Cas3-armed engineered phage with structured PK characterization across five orders of magnitude in dose offers an unusually complete biological profile for a first-in-human study; however, the 78% reduction in E. coli at day 14 did not reach statistical significance, limiting interpretation of pharmacodynamic activity; healthy-volunteer design does not characterize drug behaviour in the immunocompromised target population (HSCT recipients), and the gap between asymptomatic colonisation reduction and clinical infection prevention requires dedicated efficacy trials. | Level 2—Randomized placebo-controlled double-blind phase 1 dose-escalation trial | Controlled, exploratory |
| Gorodnichev et al. (2026)[8] | ICU VAP with MDR K. pneumoniae | Prospective open-label non-randomized with comparator groups; n = 21 (3 × 7) | Inhaled phage cocktail BID ×14 days (targeted vs non-targeted vs no phage) + antibiotics | Eradication at day 14: 86% targeted vs 57% antibiotics-only vs 0% non-targeted; no therapy-related AEs | Inclusion of a non-targeted phage control arm is rare in phage therapy literature and provides a methodological control for non-specific effects of phage administration that pure phage-vs-no-phage designs cannot; comparative design with concurrent controls represents a meaningful step up from single-arm case series; however, non-randomized allocation introduces selection bias risk between arms; very small per-arm sample size (n = 7) produces wide confidence intervals around the headline percentages, and the 0% eradication in the non-targeted arm cannot be cleanly attributed to phage mismatch without controlling for between-arm differences; open-label design adds observer bias risk. | Level 3—Prospective non-randomised controlled study with concurrent comparator arms | Controlled, exploratory |
| Paul et al. (2021)[61] | Abdominal infection with vancomycin-resistant E. faecium | Case report | IV magistral preparation with two enterococcal phages | Clinical improvement correlated with CRP reduction and no associated clinical AEs | Illustrates the feasibility of magistral pharmacy-prepared phage products as a regulatory pathway for individualized therapy in paediatric salvage settings; however, single-patient observation with no comparator cannot distinguish phage-attributable improvement from antibiotic effect, surgical management, supportive care, or natural disease trajectory; generalisability to other patients is precluded by the inherent limitations of single-case evidence. | Level 5—Single case report | Uncontrolled, exploratory |
| Rodriguez et al. (2022)[62] | Refractory MRSA chronic rhinosinusitis | Case report | Systemic + intranasal phage + antibiotics | Reported successful treatment of refractory CRS using combined systemic and topical phage therapy administered alongside antibiotics | Illustrates the use of compartment-targeted dual delivery (systemic + intranasal) for an anatomically defined infection site, demonstrating the feasibility of combined delivery strategies in difficult-to-treat upper airway infections; however, single-patient observation with concomitant oritavancin administration cannot distinguish phage-attributable improvement from antibiotic effect or natural disease fluctuation. | Level 5—Single case report | Uncontrolled, exploratory |
| Tan et al. (2021)[63] | MDR lung infection due to carbapenem-resistant A. baumannii | Case report | Nebulized personalized phage + antibiotics | Reported clinical improvement | Documents nebulised delivery of a personalised single-phage preparation matched to patient isolate in a critically complex patient profile (elderly, ventilator-dependent, multiple comorbidities); reports phage-resistance evolutionary trade-off in recovered isolates; however, dual concomitant antibiotic administration (tigecycline + polymyxin E) confounds attribution; single-phage (non-cocktail) approach carries elevated resistance risk; high endotoxin level in the preparation, acknowledged by the authors, raises preparation-quality concerns. | Level 5—Single case report | Uncontrolled, exploratory |
Evidence levels in this table follow the Oxford Centre for Evidence-Based Medicine (CEBM) Levels of Evidence (https://www.cebm.ox.ac.uk/resources/levels-of-evidence/ocebm-levels-of-evidence). Strength of evidence is graded within each tier based on the presence of a comparator arm (‘controlled’ vs ‘uncontrolled’), randomization, and the degree of methodological structure of the study (pre-specified outcomes, protocol-driven enrolment, regulatory framework, and quality verification of the intervention). ‘Structured’ studies have pre-specified outcomes and formal protocols; ‘exploratory’ studies do not. The body of evidence summarized below is predominantly hypothesis-generating; routine clinical adoption should await confirmatory randomized trials. MDR: multidrug-resistant; PDR: pandrug-resistant; GMP: Good Manufacturing Practice; QC quality control; PK/PD: pharmacokinetics and pharmacodynamics.