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

Interstitial Cystitis Clinical Trial

Official title:

Post-authorization Safety Study and Real-world Evaluation of the Use of Pentosan Polysulfate Sodium and the Development of Pigmentary Maculopathy and Pigmentary Retinopathy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05179460
• Other study ID #: CR109142
• Secondary ID: RWJ800077ICS4001
• Status: Completed
• Start date: October 26, 2021
• Est. completion date: May 20, 2022

Study information

• Verified date: December 2023
• Source: Janssen Research & Development, LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 2
• Est. completion date: May 20, 2022
• Est. primary completion date: May 20, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

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

Related Conditions & MeSH terms

• Cystitis
• Cystitis, Interstitial
• Interstitial Cystitis
• Macular Degeneration
• Pigmentary Retinopathy
• Retinal Diseases
• Retinitis Pigmentosa

Locations

Country	Name	City	State
United States	Janssen R&D, LLC	Titusville	New Jersey

Sponsors (1)

Lead Sponsor	Collaborator
Janssen Research & Development, LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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	Demographic characteristics of Cohorts: Age	Demographic characteristics of cohorts (age) will be reported.	Baseline
Secondary	Demographic characteristics of Cohorts: Sex	Demographic characteristics of cohorts (sex) will be reported.	Baseline
Secondary	Demographic characteristics of Cohorts: Race	Demographic characteristics of cohorts (race including Asian, black or African American, other, White or Caucasian) will be reported.	Baseline
Secondary	Demographic characteristics of Cohorts: Ethnicity	Demographic characteristics of cohorts (ethnicity including Hispanic and non-Hispanic) will be reported.	Baseline
Secondary	Number of Participants with Comorbidities	Number of participants with general comorbidities (diabetes, hypertension, hypercholesterolemia, vaginitis, urinary tract infection [UTI], detrusor instability, urge incontinence, and overactive bladder, autoimmune disease, Malignant tumor(s) of head and neck [plus documentation of radiation therapy] and Radiation cystitis) and ocular comorbidities (diabetic retinopathy, diabetic macular edema, optic neuropathy, glaucoma, glaucoma-related procedure, cataract [diagnosis], cataract [procedure]) will be reported.	Baseline
Secondary	Number of Participants who had Provider Characteristics	Number of participants who had provider characteristics (treating provider specialty [retina specialist, non-retina specialist, general ophthalmologist, optometrist]; rural or non-rural location of index practice [rural/non-rural; United States Department of Agriculture Economic research service 2010 classification]) will be reported.	Baseline
Secondary	Overall Cohort: Distribution of International Classification of Diseases (ICD)-9/10 Codes	ICD-9/10 codes are compared among the participants who are exposed to PPS will be reported.	Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined
Secondary	Participant's Journey to PPS	Participant's journey to PPS is defined as the sequence of medications and other interventions the participant received before and after receiving PPS.	Data analysed retrospectively from 01-Jan-2015 to 31-Mar-2021 will be examined

External Links

•   Redacted CSR synopsis-RWJ800077ICS4001