Hemorrhagic CystitisPhase 2NCT03129126
The Safety, Tolerability and Efficacy of LP-10 in Subjects With Refractory Moderate to Severe Hemorrhagic Cystitis
A Phase 2 study testing LP-10 in people with Hemorrhagic Cystitis, run by Lipella Pharmaceuticals, Inc. at 9 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemorrhagic Cystitis
- What is being tested?
- LP-10
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 9 sitesUnited States
- Study dates
- 1 Oct 2020to 29 Sep 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 17Show fewer
- 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
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Emory University
Atlanta, Georgia, 30322
Baylor College of Medicine
Houston, Texas, 77030
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Temple University
Philadelphia, Pennsylvania, 19140
Allegheny Health Network Research Institute
Pittsburgh, Pennsylvania, 15212
Premier Medical Group
Poughkeepsie, New York, 12603
University of California San Francisco
San Francisco, California, 94122
Michigan Institute of Urology
Troy, Michigan, 48084
University of Arizona
Tucson, Arizona, 85724
About this study
Official title: A Multicenter, Dose-Ranging Trial Evaluating the Safety, Tolerability and Efficacy of LP-10 in Subjects With Refractory Moderate to Severe Hemorrhagic Cystitis
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.
Read the detailed description
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
- Hemorrhagic Cystitis
- Being tested
- LP-10
- Medical subject headings
- Cystitis, Hemorrhagic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LP-10Drug
Intravesical tacrolimus
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Drug: LP-10 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: At every patient visit, up to 2 weeks following initial treatment
Secondary outcomes (12)
Urine Dipstick Mean episodes of Visible Blood
Mean episodes of visible blood in urine (or blood clots) and urine dipstick for quantitative grading of microscopic hematuria on bladder diaries
Measured: At every patient visit, up to 2 weeks following initial treatment
Mean urine hemoglobin concentration
Mean urine hemoglobin concentration
Measured: At every patient visit, up to 2 weeks following initial treatment
Urine analysis with microscopy
Urine analysis with microscopy including red blood cells per high power field test
Measured: At every patient visit, up to 2 weeks following initial treatment
Whole blood Add to dictionary levels
Whole blood tacrolimus levels
Measured: At every patient visit, up to 2 weeks following initial treatment
Blood chemistry and liver function test
Blood chemistry and liver function test
Measured: At every patient visit, up to 2 weeks following initial treatment
Patient Reported Global Response Assessment Survey Score
Changes in Global Response Assessment (GRA)
Measured: At every patient visit, up to 2 weeks following initial treatment
Patient Reported Urinary frequency
Changes in urinary frequency and incontinence measured on diaries
Measured: At every patient visit, up to 2 weeks following initial treatment
Bladder Cystoscopy
Cystoscopic changes in bladder
Measured: At initial treatment and on final patient visit, up to 2 weeks following initial treatment
Patient Reported Health Related Quality of Life Survey Score
Changes in Health Related Quality of Life (HRQOL) scores
Measured: At every patient visit, up to 2 weeks following initial treatment
Post void residual urine volume
Post void residual urine volume
Measured: At every patient visit, up to 2 weeks following initial treatment
Patient Reported Pain and Urgency
Change in pain and urgency 10 cm visual analog scales (VAS)
Measured: At every patient visit, up to 2 weeks following initial treatment
Incidence of Treatment-Emergent Adverse Events
Safety data will be collected by ongoing monitoring of adverse events, during the entire duration of the study, including need for blood transfusion, bladder irrigation, emergency room visit, hospitalization, urinary catheterization, and/or surgery in addition to patient reporting of changes in urinary frequency, hematuria/ clots, incontinence, spasm or discomfort.
Measured: At every patient visit, up to 2 weeks following initial treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2017
- Study start1 Oct 2020
- Primary completion21 Aug 2023
- Study completion29 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemorrhagic CystitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lipella Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03129126
- Sponsor's protocol ID
- Safety and Efficacy of LP-10
- Status last verified
- Mar 2023
- First submitted
- 17 Apr 2017
- Last updated
- 11 Dec 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Dose-Ranging Trial Evaluating the Safety, Tolerability and Efficacy of LP-10 in Subjects With Refractory Moderate to Severe Hemorrhagic Cystitis
- ClinicalTrials.gov ID
- NCT03129126
- Acronym / sponsor ID
- Safety and Efficacy of LP-10
- Conditions
- Hemorrhagic Cystitis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Lipella Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Oct 2020 (actual)
- Primary completion
- 21 Aug 2023 (actual)
- Study completion
- 29 Sep 2023 (actual)
- First posted
- 26 Apr 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| LP-10 2mg | Experimental | Drug: LP-10LP-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. |
| LP-10 4mg | Experimental | Drug: LP-10LP-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. |
| LP-10 8mg | Experimental | Drug: LP-10LP-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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (17 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LP-10Intravesical tacrolimus | Drug | LP-10 2mg, LP-10 4mg, LP-10 8mg |
Primary outcome
Patient Reported Mean episodes of visible blood
Time frame: At every patient visit, up to 2 weeks following initial treatment
Pre-post changes in mean episodes of visible blood in urine (or blood clots) on 3-day bladder diaries at baseline and primary endpoint
12 secondary outcomes
Urine Dipstick Mean episodes of Visible Blood
Time frame: At every patient visit, up to 2 weeks following initial treatment
Mean urine hemoglobin concentration
Time frame: At every patient visit, up to 2 weeks following initial treatment
Urine analysis with microscopy
Time frame: At every patient visit, up to 2 weeks following initial treatment
Whole blood Add to dictionary levels
Time frame: At every patient visit, up to 2 weeks following initial treatment
Blood chemistry and liver function test
Time frame: At every patient visit, up to 2 weeks following initial treatment
Patient Reported Global Response Assessment Survey Score
Time frame: At every patient visit, up to 2 weeks following initial treatment
Patient Reported Urinary frequency
Time frame: At every patient visit, up to 2 weeks following initial treatment
Bladder Cystoscopy
Time frame: At initial treatment and on final patient visit, up to 2 weeks following initial treatment
Patient Reported Health Related Quality of Life Survey Score
Time frame: At every patient visit, up to 2 weeks following initial treatment
Post void residual urine volume
Time frame: At every patient visit, up to 2 weeks following initial treatment
Patient Reported Pain and Urgency
Time frame: At every patient visit, up to 2 weeks following initial treatment
Incidence of Treatment-Emergent Adverse Events
Time frame: At every patient visit, up to 2 weeks following initial treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2023.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LP-10 in people with Hemorrhagic Cystitis, run by Lipella Pharmaceuticals, Inc. at 9 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Patient Reported Mean episodes of visible blood (time frame: At every patient visit, up to 2 weeks following initial treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Dec 2023.