A Multicenter, Dose-Ranging Trial Evaluating the Safety, Tolerability and Efficacy of LP-10 in Subjects With Refractory Moderate to Severe Hemorrhagic Cystitis

Safety and Efficacy of LP-10

The purpose of this study is to assess the safety and tolerability of three doses of LP-10 (intravesical tacrolimus). Twelve subjects meeting the inclusion and exclusion criteria will be enrolled and treated in a prospective and multi-center trial with LP-10. The proposed trial will recruit 12 subjects in a dose-escalation trial where 4 subjects will be allocated into each one of three groups.

2020-10-01

2023-08-21

2023-09-29

Completed

Early phase I

13

Inclusion Criteria: Males and females, at least 18 years * History of sterile moderate to severe HC (Grade 2-4) for at least 3 months documented in the medical record with at least 1 episode of macroscopic hematuria with or without clot * Previous use of medications and/or treatment(s) for HC without success * Patients of child-bearing capability agree to use a reliable form of birth control (condoms and/or oral contraceptives) during the course of instillation therapy and for 1 week thereafter * Willing and capable of understanding and complying with all requirements of the protocol, including proper completion of the 3 day Hemorrhagic Cystitis Diary (HC Diary) and self-administered questionnaires Exclusion Criteria: * History of interstitial cystitis/painful bladder syndrome * HC due to infection (bacterial, viral or fungal) * Vesicoureteral reflux disease based on cystogram within past 12 months * Subject is currently or has previously participated in another therapeutic or device study within 3 months of screening and has not returned to baseline * Pregnant or lactating * History of bleeding diathesis or active bleeding peptic ulcer disease * Life expectancy less than 12 months * PSA \> 10.0 ng/dl (measured within the last 3 months) * Known allergy to liposomes and/or egg yolk and/or tacrolimus * Urinary retention requiring daily catheterization * Previous augmentation cystoplasty * Subjects currently taking prescribed treatment for HC will be able to continue the treatment throughout the course of the study. If the patient cannot be maintained on a stable dose of the medication(s) throughout the treatment and follow-up period they will be excluded * Subject with history of intravesical treatment(s) within 1 week prior to Study Visit 1 * Sacral and/or pudendal nerve neuromodulation device (Interstim) within the last 6 months. Subjects would not be excluded if they had Interstim greater than 6 months ago and is on a stable setting. * Evidence of renal impairment (creatinine \> two times the upper limit of normal at Visit 1), hepatic impairment (AST or ALT \> three times the upper limit of normal at Visit 1), clinically significant cardiovascular, respiratory, or psychiatric diseases per investigator's judgment * Post-void residual (PVR) urine volume of \> 150 mL at screening * The presence of any clinically significant systemic disease or condition that in the opinion of the investigator would make the patient unsuitable for the study

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2023-12-11