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

NCT ID: NCT03129126

Last Updated: 2023-12-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-10-01

Study Completion Date

2023-09-29

Brief Summary

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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.

Detailed Description

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This is a multi-center, dose-ranging study including male and female subjects with refractory moderate to severe hemorrhagic cystitis as determined by a physician. A total of up to 12 subjects are anticipated and will be enrolled in study sites in the United States. Enrollment is expected to be completed within one year of initiating the study. 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

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Hemorrhagic Cystitis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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LP-10 2mg

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.

Group Type EXPERIMENTAL

LP-10

Intervention Type DRUG

Intravesical tacrolimus

LP-10 4mg

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.

Group Type EXPERIMENTAL

LP-10

Intervention Type DRUG

Intravesical tacrolimus

LP-10 8mg

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.

Group Type EXPERIMENTAL

LP-10

Intervention Type DRUG

Intravesical tacrolimus

Interventions

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LP-10

Intravesical tacrolimus

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Lipella Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Arizona

Tucson, Arizona, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Michigan Institute of Urology

Troy, Michigan, United States

Site Status

Premier Medical Group

Poughkeepsie, New York, United States

Site Status

Temple University

Philadelphia, Pennsylvania, United States

Site Status

Allegheny Health Network Research Institute

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

Countries

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United States

Other Identifiers

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Safety and Efficacy of LP-10

Identifier Type: -

Identifier Source: org_study_id