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Pigmentary MaculopathyNCT05179460

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

An observational study of Pigmentary Maculopathy and Pigmentary Retinopathy, run by Janssen Research & Development, LLC at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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… Read more about this study

Who is it for?
Ages 18 years and older
How many people
2 participants
Where
1 siteUnited States
Study dates
26 Oct 2021to 20 May 2022
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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.

Who may be able to join

  • 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

Who cannot join

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

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Janssen R&D, LLC

    Titusville, New Jersey, 08560

Other trials in United States

About this study

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

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.

Medical subject headings
Retinitis Pigmentosa, Cystitis, Interstitial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT05179460
Study groupRoleWhat they receive
Clean CohortClean 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 CohortOverall 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) CohortIC 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).

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

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

  2. 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.

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

  3. 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.

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

  4. 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).

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

  5. 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).

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

  6. 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).

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

  7. 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.

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

  8. 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).

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

  9. 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.

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

  10. 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).

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

  11. 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.

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

  12. 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.

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

  13. 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.

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

  14. 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.

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

  15. 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.

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

  16. 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.

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

  17. 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.

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

  18. 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.

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

  19. 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.

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

  20. 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.

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

  21. 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.

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

  22. 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.

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

  23. 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.

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

  24. 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.

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

Secondary outcomes (8)
  1. Demographic characteristics of Cohorts: Age

    Demographic characteristics of cohorts (age) will be reported.

    Measured: Baseline

  2. Demographic characteristics of Cohorts: Sex

    Demographic characteristics of cohorts (sex) will be reported.

    Measured: Baseline

  3. Demographic characteristics of Cohorts: Race

    Demographic characteristics of cohorts (race including Asian, black or African American, other, White or Caucasian) will be reported.

    Measured: Baseline

  4. Demographic characteristics of Cohorts: Ethnicity

    Demographic characteristics of cohorts (ethnicity including Hispanic and non-Hispanic) will be reported.

    Measured: Baseline

  5. 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.

    Measured: Baseline

  6. 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.

    Measured: Baseline

  7. 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.

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

  8. 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.

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Jan 2022
  2. Study start26 Oct 2021
  3. Primary completion20 May 2022
  4. Study completion20 May 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Retrospective
Enrolment
2 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05179460
Sponsor's protocol ID
CR109142
Other IDs
RWJ800077ICS4001
Status last verified
Jun 2025
First submitted
10 Dec 2021
Last updated
26 Jun 2025
Results posted
No
Data monitoring committee
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Yuan Z, Vaughan S, Jeffcoat C, Hu P, Yuson R, Fife D, Borkar D. Retrospective study of incidence/prevalence of pigmentary maculopathy and retinopathy in patients receiving pentosan polysulfate sodium. PLoS One. 2025 Jan 9;20(1):e0313497. doi: 10.1371/journal.pone.0313497. eCollection 2025.

    PubMed 39787060

Questions about this trial

What is this clinical trial studying?

An observational study of Pigmentary Maculopathy and Pigmentary Retinopathy, run by Janssen Research & Development, LLC at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 26 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Clean Cohort: Incidence Rate of Pigmentary Maculopathy (PM)/ Pigmentary Retinopathy (PR)/Any Cases (time frame: Data analysed retrospectively from 22-May-2018 to 31-Mar-2021 will be examined). The study lists 24 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05179460, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2025.

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