Phase II clinical trial of PM00104 (Zalypsis®) in urothelial carcinoma patients progressing after first-line platinum-based regimen

Daniel E. Castellano, Joaquim Bellmunt, José Pablo Maroto, Albert Font-Pous, Rafael Morales-Barrera, Ismael Ghanem, Cristina Suarez, Cristina Martín Lorente, Olatz Etxaniz, Laia Capdevila, Cinthya Coronado, Vicente Alfaro, Mariano Siguero, Carlos Fernández-Teruel, Joan Carles

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    4 Citations (Scopus)


    Purpose: This exploratory phase II clinical trial evaluated the antitumor activity, safety profile and pharmacokinetics of PM00104 (Zalypsis®) 3 mg/m2 1 h every 3-week intravenous infusion in patients with advanced and/or metastatic urothelial carcinoma progressing after first-line platinum-based chemotherapy. Methods: The primary efficacy end point was the disease control rate (DCR), defined as the percentage of patients with confirmed objective response or progression-free at 3 months, according to the response evaluation criteria in solid tumors. Results: In a first stage (n = 19 patients evaluable for efficacy), only one patient achieved DCR (stable disease as best response and progression-free survival of 3.1 months). According to the 2-stage design used, the primary efficacy objective was unmet, and therefore, the trial was finalized without opening the second stage. The most common adverse events related to PM00104 were fatigue, anorexia, nausea, troponin I increase and neutropenia, which were transient and manageable with dose modifications or administration delays. Mean PK results (C max = 48.57 μg/l and area under the curve (AUC) = 154.97 h μg/l) were similar to those observed in a previous phase I trial evaluating the same dose and schedule. Few troponin I concentrations were higher than 0.10 ng/ml, and none of them were related to parameters of PM00104 exposure such as AUC or C max. Conclusions: No recommendation is given for further evaluation of PM00104 as single-agent treatment of patients with pretreated advanced and/or metastatic urothelial carcinoma. No new safety signals were observed. © 2014 Springer-Verlag Berlin Heidelberg.
    Original languageEnglish
    Pages (from-to)857-867
    JournalCancer Chemotherapy and Pharmacology
    Issue number4
    Publication statusPublished - 1 Jan 2014


    • Chemotherapy
    • Response
    • Transitional cell carcinoma


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