ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 2 for:    lipella
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03129126
Recruitment Status : Not yet recruiting
First Posted : April 26, 2017
Last Update Posted : April 26, 2017
Sponsor:
Information provided by (Responsible Party):
Lipella Pharmaceuticals, Inc.

Brief Summary:
The purpose of this study is to assess the safety and tolerability of three doses of LP-10 (intravesical tacrolimus) compared to placebo. Eighteen subjects meeting the inclusion and exclusion criteria will be enrolled and treated in a prospective, doubleblind, randomized, placebo controlled, and multicenter trial with LP-10. The proposed trial will recruit 18 subjects in a dose-escalation trial where 6 subjects will be allocated into each one of three groups.

Condition or disease Intervention/treatment Phase
Hemorrhagic Cystitis Drug: Placebo Drug: LP-10 Phase 2

Detailed Description:
This is a multi-center, dose-ranging, placebo-controlled, double-blind, randomized study including male and female subjects with refractory moderate to severe hemorrhagic cystitis as determined by a physician. A total of up to 18 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 18 subjects in a dose-escalation trial where 6 subjects will be allocated into each one of three groups.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Multicenter, Double-blind, Randomized, Dose-Ranging, Placebo Controlled Trial Comparing the Safety, Tolerability and Efficacy of LP-10 With Placebo in Subjects With Refractory Moderate to Severe Hemorrhagic Cystitis
Estimated Study Start Date : December 2017
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : February 2019

Arm Intervention/treatment
Experimental: LP-10 2mg
LP-10 (intravesical tacrolimus), 2mg reconstituted in sterile water for injection, intravesical instillations, up to four instillations, instillations will occur greater than 1 day but less than 7 days apart as needed.
Drug: LP-10
Intravesical tacrolimus

Experimental: LP-10 4mg
LP-10 (intravesical tacrolimus), 4mg reconstituted in sterile water for injection, intravesical instillations, up to four instillations, instillations will occur greater than 1 day but less than 7 days apart as needed.
Drug: LP-10
Intravesical tacrolimus

Experimental: LP-10 8mg
LP-10 (intravesical tacrolimus), 8mg reconstituted in sterile water for injection, intravesical instillations, up to four instillations, instillations will occur greater than 1 day but less than 7 days apart as needed.
Drug: LP-10
Intravesical tacrolimus

Placebo Comparator: Placebo
Normal saline, intravesical instillations, up to four instillation.
Drug: Placebo
Placebo, normal saline




Primary Outcome Measures :
  1. Patient Reported Mean episodes of visible blood [ Time Frame: At every patient visit, up to 10 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


Secondary Outcome Measures :
  1. Urine Dipstick Mean episodes of Visible Blood [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Mean episodes of visible blood in urine (or blood clots) and urine dipstick for quantitative grading of microscopic hematuria on bladder diaries

  2. Mean urine hemoglobin concentration [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Mean urine hemoglobin concentration

  3. Urine analysis with microscopy [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Urine analysis with microscopy including red blood cells per high power field test

  4. Whole blood Add to dictionary levels [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Whole blood tacrolimus levels

  5. Blood chemistry and liver function test [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Blood chemistry and liver function test

  6. Patient Reported Global Response Assessment Survey Score [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Changes in Global Response Assessment (GRA)

  7. Patient Reported Urinary frequency [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Changes in urinary frequency and incontinence measured on diaries

  8. Bladder Cystoscopy [ Time Frame: At initial treatment and on final patient visit, up to 10 weeks following initial treatment ]
    Cystoscopic changes in bladder

  9. Patient Reported Health Related Quality of Life Survey Score [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Changes in Health Related Quality of Life (HRQOL) scores

  10. Post void residual urine volume [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Post void residual urine volume

  11. Patient Reported Pain and Urgency [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    Change in pain and urgency 10 cm visual analog scales (VAS)

  12. Incidence of Treatment-Emergent Adverse Events [ Time Frame: At every patient visit, up to 10 weeks following initial treatment ]
    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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03129126


Contacts
Contact: Michele Gruber, BS 412-894-1853 michele.anthony@lipella.com

Sponsors and Collaborators
Lipella Pharmaceuticals, Inc.

Responsible Party: Lipella Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03129126     History of Changes
Other Study ID Numbers: Safety and Efficacy of LP-10 1
First Posted: April 26, 2017    Key Record Dates
Last Update Posted: April 26, 2017
Last Verified: April 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Cystitis
Urinary Bladder Diseases
Urologic Diseases
Tacrolimus
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Calcineurin Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action