Intravesical Liposomes for Ulcerative Cystitis

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).

Study Overview

• Status: Completed
• Conditions: Interstitial Cystitis
• Intervention / Treatment: Drug: Liposomes

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.

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Michigan
    • Royal Oak, Michigan, United States, 48073
      • William Beaumont Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Liposomes; Intravesical instillation of Liposomes in sterile water totally 40 cc at four weekly treatments.	Drug: Liposomes; Intravesical instillation; Other Names: Bladder instillation

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Baseline to 12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Baseline to 12 Weeks

Collaborators and Investigators

• Sponsor: Kenneth Peters, MD
• Investigators: Principal Investigator: Kenneth M Peters, MD, William Beaumont Hospitals

Study record dates

Study Major Dates

• Study Start: August 1, 2010
• Primary Completion (Actual): October 1, 2010
• Study Completion (Actual): October 1, 2010

Study Registration Dates

• First Submitted: March 8, 2010
• First Submitted That Met QC Criteria: March 9, 2010
• First Posted (Estimate): March 10, 2010

Study Record Updates

• Last Update Posted (Estimate): February 8, 2017
• Last Update Submitted That Met QC Criteria: December 14, 2016
• Last Verified: December 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Urinary Bladder Diseases; Cystitis; Cystitis, Interstitial
• Other Study ID Numbers: 2010-021

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: No plan to make individual participant data available

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No