Interstitial Cystitis
Conditions
Brief summary
The objective is to instill liposomes reconstituted with sterile water into the bladder as a compassionate use treatment in one patient with ulcerative interstitial cystitis (IC).
Detailed description
A physician sponsored Investigation New Drug (IN) has been acquired from the FDA for compassionate use of this non-approved treatment in one patient from Dr. Peters' private practice. Since this treatment has not been used previously in the US, we propose to evaluate the safety and efficacy of the treatment in this one patient by comparing pre and post treatment symptom changes. Prior to treatment (visit 1) baseline measures will be assessed: vital signs,pelvic/bladder pain on a visual analog scale (VAS; 0 being none and 10 being severe), concomitant medications, Serum pregnancy test and Antiphospholipid Antibody Panel, Urine cytology, urinalysis (dipstick), and culture. At visits 2-5 (4 treatment visits) questionnaires will be completed and vital signs, pain VAS, post void residual (measured by bladder scan or catheter), unanticipated events, and voiding history (voiding diary) will also be assessed. Additionally, cystoscopy will be done prior to the first treatment to view the bladder lining. At each treatment visit, the patients will have a solution of liposomes instilled in the bladder with a catheter, retained for 30 minutes, and be carefully monitored for at least 1 hour after the liposomes are drained. After treatment, visits 6 and 7 (follow up) will include vital signs with VAS pain assessment, post void residual (PVR), urinalysis (dipstick), unanticipated events assessment, questionnaire completion and review of voiding diary. Visit 8 (study exit) will include the same parameters measured at follow up visits 6 and 7 with the addition of cystoscopy and Antiphospholipid Antibody Panel testing.
Interventions
DRUGLiposomes
Intravesical instillation
Sponsors
Kenneth Peters, MD
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
20 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Ulcerative IC for at least 6 months documented, * Negative urine cytology, * Able to independently complete self administered questionnaires and voiding diaries.
Exclusion criteria
* Pregnant or lactating, * History of bleeding diathesis, * On anticoagulant therapy, * Active peptic ulcer disease, * Obvious neurological impairment, * Known allergy to liposomes.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Symptom and Problem Severity | Baseline to 12 weeks | The primary objective is to determine the impact of 4 weekly bladder instillations of liposomes on symptoms in one patient with ulcerative interstitial cystitis (IC). The primary endpoint will be assessed at the end of the study, 8 weeks after the last bladder instillation, and will be measured by the O'Leary-Sant IC Symptom and Problem Indices (ICSI-PI) questionnaire. The ICSI is composed of 4 questions that address the occurrence of IC symptoms, specifically urinary urgency, frequency, nocturia, and bladder pain. Scores range from 0 (Not at All) to 5 (Almost Always). The IC Problem Indices questionnaire is also composed of 4 questions. Each question asks the patient to indicate how big a problem each of the 4 symptoms are to them. Scores range from 0 (No Problem) to 4 (Big Problem). Responses to all 8 questions are added together to create a total ICSI-PI score. The total ICSI-PI scores ranges from 0 to 36. A lower score indicates less IC symptoms and/or related problem |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Bladder Appearance | Baseline to 12 Weeks | The secondary objective to assess treatment efficacy will compare the number of bladder ulcers pre-treatment to the number of ulcers visualized at 12 weeks, the end of the study. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Liposomes Intravesical instillation of Liposomes in sterile water totally 40 cc at four weekly treatments. | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | Liposomes |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants |
| Gender Female | 1 Participants |
| Gender Male | 0 Participants |
| Region of Enrollment United States | 1 participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 1 |
| serious Total, serious adverse events | 0 / 1 |
Outcome results
Primary
Change in Symptom and Problem Severity
The primary objective is to determine the impact of 4 weekly bladder instillations of liposomes on symptoms in one patient with ulcerative interstitial cystitis (IC). The primary endpoint will be assessed at the end of the study, 8 weeks after the last bladder instillation, and will be measured by the O'Leary-Sant IC Symptom and Problem Indices (ICSI-PI) questionnaire. The ICSI is composed of 4 questions that address the occurrence of IC symptoms, specifically urinary urgency, frequency, nocturia, and bladder pain. Scores range from 0 (Not at All) to 5 (Almost Always). The IC Problem Indices questionnaire is also composed of 4 questions. Each question asks the patient to indicate how big a problem each of the 4 symptoms are to them. Scores range from 0 (No Problem) to 4 (Big Problem). Responses to all 8 questions are added together to create a total ICSI-PI score. The total ICSI-PI scores ranges from 0 to 36. A lower score indicates less IC symptoms and/or related problem
Time frame: Baseline to 12 weeks
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Liposomes | Change in Symptom and Problem Severity | 12 Week Total ICSI-PI Score | 13 units on a scale |
| Liposomes | Change in Symptom and Problem Severity | Baseline Total ICSI-PI Score | 22 units on a scale |
Secondary
Bladder Appearance
The secondary objective to assess treatment efficacy will compare the number of bladder ulcers pre-treatment to the number of ulcers visualized at 12 weeks, the end of the study.
Time frame: Baseline to 12 Weeks
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Liposomes | Bladder Appearance | Total Number of Ulcers at Baseline | 3 Ulcers |
| Liposomes | Bladder Appearance | Total Number of Ulcers at 12 Weeks | 0 Ulcers |