A Study of Pentosan Polysulfate Sodium and the Development of Pigmentary Maculopathy and Pigmentary Retinopathy

NCT05179460 | Completed

• 2 other identifiers: CR109142RWJ800077ICS4001
• Study Type: observational
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate incidence and prevalence rates of the study endpoints (pigmentary maculopathy \[PM\]/ pigmentary retinopathy \[PR\]/Any, PM/PR/ pentosan polysulfate sodium \[PPS\], and PM/PR/Non-PPS) in relation to PPS exposure, and in participants with interstitial cystitis (IC) but not exposed to PPS; changes in visual acuity (VA) over time; participant treatment journey leading to PPS treatment, and potential risk factors associated with the occurrence of PM/PR/PPS.

Conditions

Pigmentary Maculopathy; Pigmentary Retinopathy; Interstitial Cystitis

Outcome Measures

Primary Outcomes (24)

• Clean Cohort: Incidence Rate of Pigmentary Maculopathy (PM)/ Pigmentary Retinopathy (PR)/Any Cases

  Incidence rate is defined as number of incident cases per number of all participants at risk (that is, number of all participants exposed to pentosan polysulfate sodium \[PPS\]). Incidence rate is calculated by using formula: incidence rate (per 100 person-years)= number of incidence cases divided by number of person-years time at risk.

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Clean Cohort: Incidence Rate of PM/PR/PPS

  Incidence rate is defined as number of incident cases per number of all participants at risk (that is, number of all participants exposed to PPS). Incidence rate is calculated by using formula: incidence rate (per 100 person-years)= number of incidence cases divided by number of person-years time at risk.

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Clean Cohort: Incidence Rate of PM/PR/Non-PPS

  Incidence rate is defined as number of incident cases per number of all participants at risk (that is, number of all participants not exposed to PPS). Incidence rate is calculated by using formula: incidence rate (per 100 person-years)= number of incidence cases divided by number of person-years time at risk.

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Clean Cohort: Prevalence Rate of PM/PR/Any Cases

  Prevalence rate is defined as number of prevalent cases per number of target cohort assessed (example, number of participants exposed to PPS).

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Clean Cohort: Prevalence Rate of PM/PR/PPS

  Prevalence rate is defined as number of prevalent cases per number of target cohort assessed (example, number of participants exposed to PPS).

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Clean Cohort: Prevalence Rate of PM/PR/Non-PPS

  Prevalence rate is defined as number of prevalent cases per number of target cohort assessed (example, number of participants not exposed to PPS).

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Overall Cohort: Incidence Rate of PM/PR/Any Cases

  Incidence rate is defined as number of incident cases per number of all participants at risk (that is, number of all participants exposed to pentosan polysulfate sodium \[PPS\]). Incidence rate is calculated by using formula: incidence rate (per 100 person-years)= number of incidence cases divided by number of person-years time at risk.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• Overall Cohort: Prevalence Rate of PM/PR/Any Cases

  Prevalence rate is defined as number of prevalent cases per number of target cohort assessed (example, number of participants exposed to PPS).

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• Overall Cohort: Incidence Rate of PM/PR/PPS

  Incidence rate is defined as number of incident cases per number of all participants at risk (that is, number of all participants exposed to pentosan polysulfate sodium \[PPS\]). Incidence rate is calculated by using formula: incidence rate (per 100 person-years)= number of incidence cases divided by number of person-years time at risk.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• Overall Cohort: Prevalence Rate of PM/PR/PPS

  Prevalence rate is defined as number of prevalent cases per number of target cohort assessed (example, number of participants exposed to PPS).

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• Overall Cohort: Number of PM/PR/PPS Cases Among the PM/PR/Any Cases

  Number of PM/PR/PPS cases among the PM/PR/any cases will be reported.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• Clean Cohort: Change in Visual Acuity (VA) in Relation to PPS Dose

  Change in VA in relation to PPS dose will be reported. It will be assessed based on the following categories: a) No change (refers to less than \[\<\] 1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) greater than or equal to (\>=) 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Clean Cohort: Change in VA in Relation to PM/PR/Any Cases

  Change in VA in relation to study endpoint (PM/PR/Any) will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Clean Cohort: Change in VA in Relation to PM/PR/PPS

  Change in VA in relation to study endpoint (PM/PR/PPS) will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Clean Cohorts: Change in VA in Relation to PM/PR/Non-PPS

  Change in VA in relation to study endpoint (PM/PR/Non-PPS) will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined

• Overall Cohort: Change in Visual Acuity (VA) in Relation to PPS Dose

  Change in VA in relation to PPS dose will be reported. It will be assessed based on the following categories: a) No change (refers to less than \[\<\] 1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) greater than or equal to (\>=) 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• Overall Cohort: Change in VA in Relation to PM/PR/Any Cases

  Change in VA in relation to study endpoint (PM/PR/Any) will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• Overall Cohort: Change in VA in Relation to PM/PR/PPS

  Change in VA in relation to study endpoint (PM/PR/PPS) will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• Overall Cohort: Change in VA in Relation to PM/PR/Non-PPS

  Change in VA in relation to study endpoint (PM/PR/Non-PPS) will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• Interstitial Cystitis (IC) Cohort: Incidence Rate of PM/PR/Any Cases Among the Participants with IC and No-Exposure to PPS

  Incidence rate is defined as number of incident cases per number of all participants at risk (that is, number of all participants not exposed to PPS). Incidence rate is calculated by using formula: incidence rate (per 100 person-years)= number of incidence cases per number of person-years time at risk.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• IC Cohort: Change in VA in Relation to PM/PR/Any Cases

  Change in VA in relation to study endpoint (PM/PR/Any) will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• IC Cohort: Change in VA Based on Age

  Change in VA based on age among participants exposed to PPS and without exposure to PPS will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• IC Cohort: Change in VA Based on Sex

  Change in VA based on sex among participants exposed to PPS and without exposure to PPS will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

• IC Cohort: Change in VA Based on Time Between the First and Last VA Measurement in Matched Cohorts

  Change in VA based on time between the first and last VA measurement in matched cohorts among participants exposed to PPS and without exposure to PPS will be reported. It will be assessed based on the following categories: a) No change (refers to \<1 line of worsening or improvement, considered not clinically meaningful); b) 1 to \<3 lines of worsening; c) \>= 3 lines of worsening; d) 1 to \<3 lines of improvement; e) \>= 3 lines of improvement.

  Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

Secondary Outcomes (8)

• Demographic characteristics of Cohorts: Age

  Baseline

• Demographic characteristics of Cohorts: Sex

  Baseline

• Demographic characteristics of Cohorts: Race

  Baseline

• Demographic characteristics of Cohorts: Ethnicity

  Baseline

• Number of Participants with Comorbidities

  Baseline

Study Arms (3)

Clean Cohort

Clean cohort refers to cohort of participants who had their first documented exposure to pentosan polysulfate sodium (PPS; Elmiron) on or after 22 May 2018 and who are assumed to have had shorter exposure (the earliest available data based on the linked database between the IRIS registry and Komodo database in this study).

Overall Cohort

Overall cohort refers to cohort of participants who had their first documented exposure to PPS (Elmiron) any time beginning 01 January 2015 and who are assumed to have relatively longer exposure (the earliest available data based on the linked database between the intelligent research in sight (IRIS) registry and Komodo database in this study).

Interstitial Cystitis (IC) Cohort

IC cohort refers to cohort of participants who had at least one IC diagnosis beginning 01 January 2015 and had no documented exposure to PPS based on the records from the Komodo database (the earliest available data based on the linked database between the IRIS registry and Komodo database in this study).

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population is derived from the United States-based electronic databases, including the Intelligent Research in Sight (IRIS) Registry, American Urological Association Quality (AQUA) Registry, and Komodo Health claims. The selection of the study participants and cohort creation will be determined by the inclusion and exclusion criteria.

You may qualify if:

• Participants must have at least 6 months baseline information prior to index date (this may apply to the relevant databases, if the study participants are identified and the outcomes are ascertained via multiple linked data source) For the pentosan polysulfate sodium (PPS) Cohort
• Participants must have records in both the intelligent research in sight (IRIS) database and the closed claims portion of the Komodo claims database and have at least one record of PPS dispensing For the interstitial cystitis (IC) Cohort not exposed to PPS
• Participants must have records in both the IRIS database and the closed claims portion of the claims database; have at least one diagnosis of IC; and have no record of PPS dispensing

You may not qualify if:

• \- Evaluated based on the Komodo database. Participants will be excluded from the study if they have no information on age or sex (or both)

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (1)

Janssen R&D, LLC

Titusville, New Jersey, 08560, United States

Location

Related Links

• Redacted CSR synopsis-RWJ800077ICS4001

MeSH Terms

Conditions

Retinitis Pigmentosa; Cystitis, Interstitial

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cystitis; Urinary Bladder Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 10, 2021
• First Posted: January 5, 2022
• Study Start: October 26, 2021
• Primary Completion: May 20, 2022
• Study Completion: May 20, 2022
• Last Updated: June 26, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)