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

NCT03129126 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: Safety and Efficacy of LP-10
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 9

Brief 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.

Conditions

Hemorrhagic Cystitis

Outcome Measures

Primary Outcomes (1)

• Patient Reported Mean episodes of visible blood

  Pre-post changes in mean episodes of visible blood in urine (or blood clots) on 3-day bladder diaries at baseline and primary endpoint

  At every patient visit, up to 2 weeks following initial treatment

Secondary Outcomes (12)

• Urine Dipstick Mean episodes of Visible Blood

  At every patient visit, up to 2 weeks following initial treatment

• Mean urine hemoglobin concentration

  At every patient visit, up to 2 weeks following initial treatment

• Urine analysis with microscopy

  At every patient visit, up to 2 weeks following initial treatment

• Whole blood Add to dictionary levels

  At every patient visit, up to 2 weeks following initial treatment

• Blood chemistry and liver function test

  At every patient visit, up to 2 weeks following initial treatment

Study Arms (3)

LP-10 2mg

EXPERIMENTAL

LP-10 (intravesical tacrolimus), 2mg reconstituted in sterile water for injection, intravesical instillations, up to two instillations, instillations will occur greater than 3 days but less than 7 days apart as needed.

Drug: LP-10

LP-10 4mg

EXPERIMENTAL

LP-10 (intravesical tacrolimus), 4mg reconstituted in sterile water for injection, intravesical instillations, up to two instillations, instillations will occur greater than 3 days but less than 7 days apart as needed.

Drug: LP-10

LP-10 8mg

EXPERIMENTAL

LP-10 (intravesical tacrolimus), 8mg reconstituted in sterile water for injection, intravesical instillations, up to two instillations, instillations will occur greater than 3 days but less than 7 days apart as needed.

Drug: LP-10

Interventions

LP-10 DRUG

Intravesical tacrolimus

LP-10 2mg; LP-10 4mg; LP-10 8mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Lipella Pharmaceuticals, Inc.: lead

Study Sites (9)

University of Arizona

Tucson, Arizona, 85724, United States

Location

University of California San Francisco

San Francisco, California, 94122, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Michigan Institute of Urology

Troy, Michigan, 48084, United States

Location

Premier Medical Group

Poughkeepsie, New York, 12603, United States

Location

Temple University

Philadelphia, Pennsylvania, 19140, United States

Location

Allegheny Health Network Research Institute

Pittsburgh, Pennsylvania, 15212, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Cystitis, Hemorrhagic

Condition Hierarchy (Ancestors)

Cystitis; Urinary Bladder Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 17, 2017
• First Posted: April 26, 2017
• Study Start: October 1, 2020
• Primary Completion: August 21, 2023
• Study Completion: September 29, 2023
• Last Updated: December 11, 2023

Record last verified: 2023-03

Locations

US (9)