LBA01-11 PHASE 2A MULTICENTER DOSE ESCALATION STUDY OF LP-10 FOR THE TREATMENT OF REFRACTORY MODERATE TO SEVERE HEMORRHAGIC CYSTITIS

Abstract

You have accessJournal of UrologyCME1 Apr 2023LBA01-11 PHASE 2A MULTICENTER DOSE ESCALATION STUDY OF LP-10 FOR THE TREATMENT OF REFRACTORY MODERATE TO SEVERE HEMORRHAGIC CYSTITIS Jason Hafron, Benjamin Bryer, Shreyas Joshi, Christopher Smith, Melissa Kaufman, Janet Okonski, and Michael Chancellor Jason HafronJason Hafron More articles by this author , Benjamin BryerBenjamin Bryer More articles by this author , Shreyas JoshiShreyas Joshi More articles by this author , Christopher SmithChristopher Smith More articles by this author , Melissa KaufmanMelissa Kaufman More articles by this author , Janet OkonskiJanet Okonski More articles by this author , and Michael ChancellorMichael Chancellor More articles by this author View All Author Informationhttps://doi.org/10.1097/JU.0000000000003360.11AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: Radiation used to treat prostate, colon, uterine, cervical and other pelvic cancers can cause chronic, painful urinary inflammation and hemorrhagic cystitis (HC). The blood loss associated with hemorrhagic cystitis can lead to surgery and can be fatal. Lipella Pharmaceuticals received orphan disease designation for HC and recently completed a phase 2a clinical trial of LP-10 for the treatment of HC. METHODS: The LP-10 Phase 2a clinical trial was a multi-center, dose-escalation study of intravesical instillation of liposomal formulation of tacrolimus at 2mg, 4 mg and 8mg. The study recruited 13 subjects with moderate to severe refractory hemorrhagic cystitis. These subjects were treated with up to two courses of LP-10 bladder instillations (clinicaltrials.gov: NCT01393223). RESULTS: Fifteen subjects were screened (14 male and 1 female) and 13 enrolled. All enrolled subjects were male with mean age 67 (range 25-89 years old) with a history of prostate cancer (n=9), bladder cancer (n=2) and lymphoma (n=2). Mean duration of HC was 4 years and ranged from 1-14 years. All subjects tolerated LP-10 instillations and completed the study without report of product related serious adverse events. LP-10 pharmacokinetic analysis demonstrated short duration of systemic uptake. A dose response was observed. Responder analysis evaluating cystoscopy, hematuria, and symptoms noted complete response in 3 subjects, partial response in 7 subjects and no response in 3 subjects. (NR=“no response”, PR=“partial response”, CR=“complete response”) (normalized rank, 0 to 9) (Figure 1). CONCLUSIONS: The results of the Phase 2a study demonstrated safety and a signal of efficacy with dose response of LP-10 for the treatment of refractory moderate to severe HC. This is the first successfully completed trial for cancer survivors with HC and more research is needed to develop a drug treatment for hemorrhagic cystitis, a disease with great unmet need. Source of Funding: Lipella Pharmaceuticals, Inc. © 2023 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 209Issue Supplement 4April 2023Page: e1180 Advertisement Copyright & Permissions© 2023 by American Urological Association Education and Research, Inc.MetricsAuthor Information Jason Hafron More articles by this author Benjamin Bryer More articles by this author Shreyas Joshi More articles by this author Christopher Smith More articles by this author Melissa Kaufman More articles by this author Janet Okonski More articles by this author Michael Chancellor More articles by this author Expand All Advertisement PDF downloadLoading ...