Radiation Cystitis Treated With Pentosan Polysulfate Sodium Trial

Phase 2

Recruiting

Interventions: Drug: Pentosan Polysulfate Sodium 100 MG Oral Capsule
Drug: Placebo

Brief Summary: 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.

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).

Recruitment & Eligibility

Inclusion Criteria

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.
Patients must be ≥20 years of age
Patients must have received definitive radiation therapy for cancer in pelvic area.
Time from the end of radiation therapy to radiation cystitis must be longer than 6 months.
Radiation cystitis with lower urinary tract symptoms or hematuria.

Exclusion Criteria

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

Arm && Interventions

Group	Intervention	Description
Pentosan Polysulfate Sodium	Pentosan Polysulfate Sodium 100 MG Oral Capsule	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.
Placebo Control	Placebo	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.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Duration of hospitalization	From date of randomization to study completion up to 5 years	Overall days of hospitalization due to radiation cystitis
Number of invasive procedures	From date of randomization to study completion up to 5 years	Percentage of patients underwent transurethral cauterization of bladder bleeders or transurethral clots evacuation.
Number of adverse events	From date of randomization to study completion up to 5 years	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.