The Safety, Tolerability and Efficacy of LP-10 in Subjects With Refractory Moderate to Severe Hemorrhagic Cystitis

• Status: Recruiting
• Trial end date: 2022-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Lipella Pharmaceuticals, Inc.
• Treatments: Tacrolimus

Criteria

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