Comparison of baseline patient characteristics in phase 1 and phase 2/3 clinical trials for anticancer treatments
Abstract Background Characteristics of patients significantly differ between registrational clinical trials (CTs) and Italian real-world practice, with older median age, higher elderly (≥ 65) rate and worse performance status (PS) in the latter, without imbalance in female rate. We compared the same...
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BMC
2025-05-01
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| Series: | Journal of Translational Medicine |
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| Online Access: | https://doi.org/10.1186/s12967-025-06509-z |
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| author | Maria Lucia Iacovino Simone Talucci Laura Arenare Andrea Caglio Andrea Canciello Flavio Salerno Sergio Facchini Adriano Gravina Clorinda Schettino Maria Carmela Piccirillo Pier Paolo Olimpieri Massimo Di Maio Francesco Perrone |
| author_facet | Maria Lucia Iacovino Simone Talucci Laura Arenare Andrea Caglio Andrea Canciello Flavio Salerno Sergio Facchini Adriano Gravina Clorinda Schettino Maria Carmela Piccirillo Pier Paolo Olimpieri Massimo Di Maio Francesco Perrone |
| author_sort | Maria Lucia Iacovino |
| collection | DOAJ |
| description | Abstract Background Characteristics of patients significantly differ between registrational clinical trials (CTs) and Italian real-world practice, with older median age, higher elderly (≥ 65) rate and worse performance status (PS) in the latter, without imbalance in female rate. We compared the same characteristics between registrational phase 2/3 and phase 1 CTs. Methods Data on age, sex and PS were extracted from European Public Assessment Reports of European Medicines Agency. Weighted means and standard deviations were calculated in both groups and differences were described overall, by cancer type and drug class. Results We collected 103 phase 2/3 and 111 phase 1 CTs, supporting 97 therapeutic indications. Age and sex were compared in 59 indications. Mean median age (SD) was 60.7 (5.1) years in phase 2/3 and 59.7 (5.6) years in phase 1 (p = 0.051). Age difference was greater for skin and breast cancer; no heterogeneity emerged among drug classes. Mean female rate was not statistically significantly lower in phase 2/3 than phase 1 CTs overall, (mean difference − 4.9%, p = 0.999); difference was greater for skin and upper-gastrointestinal cancers and for cytotoxic agents. Mean PS > 1 rate, compared in 47 indications, was similar in phase 2/3 [2.3% (4.7)] and phase 1 [1.8% (3.5)] (p = 0.374); difference was greater for colorectal cancer and cytotoxic agents. Conclusions We found no statistically significant difference in age, sex and PS between patients in phase 2/3 and corresponding phase 1 CTs for anticancer treatments. Therefore, patient selection in phase 1 trials appears crucial, considering its potential impact in later development phases. |
| format | Article |
| id | doaj-art-c85cab2da4eb40a6bb07e855dba4fd96 |
| institution | OA Journals |
| issn | 1479-5876 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | BMC |
| record_format | Article |
| series | Journal of Translational Medicine |
| spelling | doaj-art-c85cab2da4eb40a6bb07e855dba4fd962025-08-20T01:51:28ZengBMCJournal of Translational Medicine1479-58762025-05-012311810.1186/s12967-025-06509-zComparison of baseline patient characteristics in phase 1 and phase 2/3 clinical trials for anticancer treatmentsMaria Lucia Iacovino0Simone Talucci1Laura Arenare2Andrea Caglio3Andrea Canciello4Flavio Salerno5Sergio Facchini6Adriano Gravina7Clorinda Schettino8Maria Carmela Piccirillo9Pier Paolo Olimpieri10Massimo Di Maio11Francesco Perrone12National Cancer Institute IRCCS Fondazione G.PascaleMenarini Ricerche SpANational Cancer Institute IRCCS Fondazione G.PascaleDepartment of Oncology, University of Turin, Ordine Mauriziano Hospital Umberto INational Cancer Institute IRCCS Fondazione G.PascaleDepartment of Oncology, University of Turin, Ordine Mauriziano Hospital Umberto INational Cancer Institute IRCCS Fondazione G.PascaleNational Cancer Institute IRCCS Fondazione G.PascaleNational Cancer Institute IRCCS Fondazione G.PascaleNational Cancer Institute IRCCS Fondazione G.PascaleItalian Medicines AgencyDepartment of Oncology, University of Turin, AOU Città della Salute e della Scienza di TorinoNational Cancer Institute IRCCS Fondazione G.PascaleAbstract Background Characteristics of patients significantly differ between registrational clinical trials (CTs) and Italian real-world practice, with older median age, higher elderly (≥ 65) rate and worse performance status (PS) in the latter, without imbalance in female rate. We compared the same characteristics between registrational phase 2/3 and phase 1 CTs. Methods Data on age, sex and PS were extracted from European Public Assessment Reports of European Medicines Agency. Weighted means and standard deviations were calculated in both groups and differences were described overall, by cancer type and drug class. Results We collected 103 phase 2/3 and 111 phase 1 CTs, supporting 97 therapeutic indications. Age and sex were compared in 59 indications. Mean median age (SD) was 60.7 (5.1) years in phase 2/3 and 59.7 (5.6) years in phase 1 (p = 0.051). Age difference was greater for skin and breast cancer; no heterogeneity emerged among drug classes. Mean female rate was not statistically significantly lower in phase 2/3 than phase 1 CTs overall, (mean difference − 4.9%, p = 0.999); difference was greater for skin and upper-gastrointestinal cancers and for cytotoxic agents. Mean PS > 1 rate, compared in 47 indications, was similar in phase 2/3 [2.3% (4.7)] and phase 1 [1.8% (3.5)] (p = 0.374); difference was greater for colorectal cancer and cytotoxic agents. Conclusions We found no statistically significant difference in age, sex and PS between patients in phase 2/3 and corresponding phase 1 CTs for anticancer treatments. Therefore, patient selection in phase 1 trials appears crucial, considering its potential impact in later development phases.https://doi.org/10.1186/s12967-025-06509-zClinical trialsPhase 1AgeSexPerformance status |
| spellingShingle | Maria Lucia Iacovino Simone Talucci Laura Arenare Andrea Caglio Andrea Canciello Flavio Salerno Sergio Facchini Adriano Gravina Clorinda Schettino Maria Carmela Piccirillo Pier Paolo Olimpieri Massimo Di Maio Francesco Perrone Comparison of baseline patient characteristics in phase 1 and phase 2/3 clinical trials for anticancer treatments Journal of Translational Medicine Clinical trials Phase 1 Age Sex Performance status |
| title | Comparison of baseline patient characteristics in phase 1 and phase 2/3 clinical trials for anticancer treatments |
| title_full | Comparison of baseline patient characteristics in phase 1 and phase 2/3 clinical trials for anticancer treatments |
| title_fullStr | Comparison of baseline patient characteristics in phase 1 and phase 2/3 clinical trials for anticancer treatments |
| title_full_unstemmed | Comparison of baseline patient characteristics in phase 1 and phase 2/3 clinical trials for anticancer treatments |
| title_short | Comparison of baseline patient characteristics in phase 1 and phase 2/3 clinical trials for anticancer treatments |
| title_sort | comparison of baseline patient characteristics in phase 1 and phase 2 3 clinical trials for anticancer treatments |
| topic | Clinical trials Phase 1 Age Sex Performance status |
| url | https://doi.org/10.1186/s12967-025-06509-z |
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