Interstitial Cystitis, Pelvic Pain
Conditions
Brief summary
Interstitial cystitis/painful bladder syndrome (IC/PBS) is a poorly understood chronic disorder of unknown etiology consisting of irritative bladder symptoms and pelvic pain that dramatically affects quality of life. Preclinical study data (obtained by using an IC/PBS model in Sprague-Dawley female rats) have demonstrated normalization of urinary frequency indicating that LP may be a potent protectant of the bladder mucosa against inflammation and irritation. Intravesical LP has so far demonstrated an excellent safety profile and minimal toxicity at concentrations of 2 mg/ml. Thus, we hypothesize that intravesical instillation of LP may form a molecular film on bladder ulcer surfaces in patients with IC and provide a safe, effective, and minimally invasive treatment option to alleviating symptoms.
Detailed description
Liposomes (LP), the treatment article in this proposal, were discovered in the process of exploring a control compound when measuring the efficacy of liposomally encapsulated capsaicin for the intravesical treatment of interstitial cystitis. Liposomes are lipid vesicles composed of concentric phospholipid bilayers, which enclose an aqueous interior. Liposomes have the ability to form a molecular film on cell and tissue surfaces and are currently being tested as possible therapeutic agents to promote wound healing. Application of liposomes at the wound surface provides a moist protective film over the wound and augments wound healing without chronic inflammatory reactions in the neodermal layer.
Interventions
BIOLOGICALLiposomes
Intravesical instillation of liposomes.
Sponsors
Corewell Health East
Kenneth Peters, MD
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* • Written informed consent has been obtained * Males and females, at least 18 years of age * History of IC/PBS for at least 6 months documented in the medical record * Recurring IC/PBS symptoms * An average of 8 or more urine voids over a 3-day period, confirmed by the baseline voiding diary * Bladder pain score \> 4 in the last 24 hours (assessed at screening visit) * Previous use of medications and/or treatment(s) for symptom relief * Females of child-bearing capability agree to use a reliable form of birth control (condoms and/or oral contraceptives (birth control pills)) during the 4 week course of therapy and 1 week thereafter * Willing and capable of understanding and complying with all requirements of the protocol, including proper completion of the voiding diaries and self-administered questionnaires
Exclusion criteria
* • Subjects currently taking prescribed medications for IC/PBS will be able to continue the medications 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. * Subjects must not have had intravesical treatment(s)/bladder installations of the following medications: dimethylsulfoxide (DMSO), lidocaine and/or heparin within 1 month prior to study visit 1 * Pregnant or lactating * History of bleeding diathesis * Currently on anticoagulant therapy (e.g. warfarin, clopidogrel) * Active bleeding peptic ulcer disease * Obvious neurological impairment which may be affecting bladder function * Known allergy to liposomes and/or egg yolk * Current or previous participation in another therapeutic or device study within 6 months of the screening visit * 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Symptom Severity at 4 and 8 Weeks Post-Treatment as Measured by the Total O'Leary-Sant IC Symptom and Problem Index (ICSI/ICPI) Score | 4 and 8 weeks post-treatment | The O'Leary-Sant IC Symptom Index (ICS-I) total score ranges from 0 to 20 and the Problem Index (ICP-I) total score ranges from 0 to 16. Each index has 4 questions and lower scores represent a better outcome. A total ICSI/ICPI score is obtained by adding the total scores from both indices. The combined ICSI/ICPI total score ranges from 0 to 36. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Pain Scores at 4 and 8 Weeks Post-Treatment as Measured by the Visual Analog Scale (VAS) | 4 and 8 weeks post-treatment | Patients utilized the Visual Analog Scale (VAS) to describe their pain. The scale ranges from 0:No pain to 10: Pain as bad as it could possibly be. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Liposomes Liposomes
Liposomes: Intravesical instillation of liposomes. | 16 |
| Total | 16 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Did not meet study criteria | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Liposomes |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 4 Participants |
| Age, Categorical Between 18 and 65 years | 12 Participants |
| Gender Female | 13 Participants |
| Gender Male | 3 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 15 Participants |
| Region of Enrollment United States | 16 participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 4 / 14 |
| serious Total, serious adverse events | 0 / 14 |
Outcome results
Primary
Change in Symptom Severity at 4 and 8 Weeks Post-Treatment as Measured by the Total O'Leary-Sant IC Symptom and Problem Index (ICSI/ICPI) Score
The O'Leary-Sant IC Symptom Index (ICS-I) total score ranges from 0 to 20 and the Problem Index (ICP-I) total score ranges from 0 to 16. Each index has 4 questions and lower scores represent a better outcome. A total ICSI/ICPI score is obtained by adding the total scores from both indices. The combined ICSI/ICPI total score ranges from 0 to 36.
Time frame: 4 and 8 weeks post-treatment
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Liposomes | Change in Symptom Severity at 4 and 8 Weeks Post-Treatment as Measured by the Total O'Leary-Sant IC Symptom and Problem Index (ICSI/ICPI) Score | Baseline to 8 Weeks Post Treatment | -4 units on a scale |
| Liposomes | Change in Symptom Severity at 4 and 8 Weeks Post-Treatment as Measured by the Total O'Leary-Sant IC Symptom and Problem Index (ICSI/ICPI) Score | Baseline to 4 Weeks Post Treatment | -4 units on a scale |
Secondary
Change in Pain Scores at 4 and 8 Weeks Post-Treatment as Measured by the Visual Analog Scale (VAS)
Patients utilized the Visual Analog Scale (VAS) to describe their pain. The scale ranges from 0:No pain to 10: Pain as bad as it could possibly be.
Time frame: 4 and 8 weeks post-treatment
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Liposomes | Change in Pain Scores at 4 and 8 Weeks Post-Treatment as Measured by the Visual Analog Scale (VAS) | Baseline to 4 Weeks Post | -2 units on a scale |
| Liposomes | Change in Pain Scores at 4 and 8 Weeks Post-Treatment as Measured by the Visual Analog Scale (VAS) | Baseline to 8 Weeks Post-Treatment | -1.14 units on a scale |