Radiation Cystitis Treated With Pentosan Polysulfate Sodium Trial

A Randomized, Controlled, Phase II/III Trial to Evaluate the Efficacy and Safety of U101 Oral Capsules in Radiation-induced Cystitis

The purpose of this study is to evaluate the efficacy and safety of pentosan polysulfate sodium versus placebo and in patients with radiation cystitis who have received radiation therapy in pelvic region.

Study Overview

• Status: Recruiting
• Conditions: Radiation Cystitis
• Intervention / Treatment: Drug: Pentosan Polysulfate Sodium 100 MG Oral Capsule; Drug: Placebo

Detailed Description

RCT-PPS is a phase II/III study evaluating the efficacy, safety, and tolerability of pentosan polysulfate sodium (PPS) versus placebo to patients with radiation cystitis who have received radiation therapy in pelvic region.

Approximately 72 patients were planned to be randomized in RCT-PPS trial in a 1:1 ratio to treatment with either PPS or placebo. Patients will receive oral treatment with 16 weeks (PPS 100mg three times a day for 8 weeks and then with PPS 100mg twice daily for another 8 weeks).

• Study Type: Interventional
• Enrollment (Anticipated): 72
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Chi-Shin Tseng, MD; Phone Number: +886223123456; Email: clifford1987tcs@gmail.com

Study Locations

• Taiwan
  • Taipei, Taiwan
    • Recruiting
    • National Taiwan University Hospital
    • Contact: Chao-Yuan Huang, phD

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:

1. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in the study protocol.
2. Patients must be ≥20 years of age
3. Patients must have received definitive radiation therapy for cancer in pelvic area.
4. Time from the end of radiation therapy to radiation cystitis must be longer than 6 months.
5. Radiation cystitis with lower urinary tract symptoms or hematuria.

Exclusion Criteria:

1. Abnormal liver function with indication of AST, ALT, or total bilirubin ≥ 1.5 times higher than normal
2. Abnormal renal function with serum creatinine ≥ 1.5 times higher than normal
3. Abnormal coagulation profile with PT/INR higher than normal
4. Thrombocytopenia with platelet counts < 100,000/μL
5. Pregnant or breastfeeding, expecting to pregnancy, or positive for pregnancy test
6. Patients who have systemic inflammatory symptoms during screening period (ie, fever up to 38℃ or WBC counts >12,000/μL)
7. Patients with known urinary tract infection within 6 months of randomization.
8. Any previous intravesical instillation within 6 months of randomization (ie, hyaluronic acid instillation)
9. Any previous hyperbaric oxygen therapy within 6 months of randomization
10. Any previous treatment with pentosan polysulfate sodium within 6 months of randomization
11. Has history of thrombocytopenia, hemophilia or bladder cancer
12. Has known history of Human Immunodeficiency Virus (HIV) or organ transplantation
13. Has known history of drug allergy to pentosan polysulfate sodium
14. Has hematuria caused by clinically active urolithiasis or urothelial carcinoma

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pentosan Polysulfate Sodium; U101 is available as a capsule containing 100 milligrams (mg) of pentosan polysulfate sodium. Subjects will be administered U101 at a dose of 300 milligrams (mg) three times daily (tid) for the initial 8 weeks and then 200 milligrams (mg) twice daily (bid) for another 8 weeks during the study.	Drug: Pentosan Polysulfate Sodium 100 MG Oral Capsule; Pentosan Polysulfate Sodium 100 mg three times daily (tid) for 0-8 weeks Pentosan Polysulfate Sodium 100 mg twice daily (bid) for 9-16 weeks; Other Names: Urosan; ELMIRON; PPS
Placebo Comparator: Placebo Control; Placebo to match U101 is available as a capsule in 100 milligrams (mg). Subjects will be administered placebo at a dose of 300 milligrams (mg) three times daily (tid) for the initial 8 weeks and then 200 milligrams (mg) twice daily (bid) for another 8 weeks during the study.	Drug: Placebo; Placebo 100 mg three times daily (tid) for 0-8 weeks Placebo 100 mg twice daily (bid) for 9-16 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of complete remission in lower urinary tract symptoms and hematuria	Number of participants without lower urinary tract symptoms and hematuria assessed by ESOU 2019 and CTCAE v5.0	From date of randomization throughout the treatment period up to 5 years
Time to complete remission	Time from randomization to complete remission of lower urinary tract syndromes and hematuria	From date of randomization to study completion up to 5 years
Time to hematuria recurrence	Time from randomization to recurrence of hematuria	From date of randomization to study completion up to 5 years
Assessment of quality of life	Functional Assessment of 36-Item Short Form Survey (SF-36)	From date of randomization to study completion up to 5 years
Assessment of symptoms	Functional Assessment of Expanded Prostate Cancer Index Composite-26 (EPIC-26)	From date of randomization to study completion up to 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Duration of hospitalization	Overall days of hospitalization due to radiation cystitis	From date of randomization to study completion up to 5 years
Number of invasive procedures	Percentage of patients underwent transurethral cauterization of bladder bleeders or transurethral clots evacuation.	From date of randomization to study completion up to 5 years
Number of adverse events	Percentage of patients with any adverse event (AE), adverse event (AE) leading to study drug discontinuation, serious adverse event (SAE), and adverse event (AE) related to study drug.	From date of randomization to study completion up to 5 years

Collaborators and Investigators

• Sponsor: National Taiwan University Hospital
• Collaborators: TCM Biotech International Corporation
• Investigators: Principal Investigator: Chao-Yuan Huang, MD, PhD, National Taiwan University Hospital

Study record dates

Study Major Dates

• Study Start (Actual): April 29, 2022
• Primary Completion (Anticipated): November 1, 2024
• Study Completion (Anticipated): November 1, 2029

Study Registration Dates

• First Submitted: January 23, 2022
• First Submitted That Met QC Criteria: February 16, 2022
• First Posted (Actual): February 18, 2022

Study Record Updates

• Last Update Posted (Actual): May 4, 2022
• Last Update Submitted That Met QC Criteria: May 2, 2022
• Last Verified: January 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Urinary Bladder Diseases; Cystitis; Anticoagulants; Pentosan Sulfuric Polyester
• Other Study ID Numbers: 202108161MIPD

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

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