Pentosan Polysulfate Sodium for Osteoarthritis, Knee

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Osteoarthritis, Knee+1 More
Pentosan Polysulfate Sodium - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This trial will test the effectiveness of pentosan polysulfate sodium injections compared to placebo injections in reducing knee pain and improving function in people with osteoarthritis.

Eligible Conditions
  • Osteoarthritis, Knee

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 36 Secondary · Reporting Duration: Stage 1: Day 1, 15 and 39 - pre-dose and 2, 4, and 6 hours. Pre-dose on Days 4, 8, 11, 18, 22, 25, 29, 32 and 36

Baseline to Day 56
Key secondary: Change from baseline at Day 56 in function as assessed by the average functional subscale score of the WOMAC NRS 3.1 Index
Day 84
Stage 1 only: Change from baseline at Day 56 and 84 in knee pain as assessed by the average pain subscale score of the WOMAC NRS 3.1 Index for PPS 1.5 mg/kg twice weekly, PPS 2.0mg/kg once weekly, and PPS fixed dose once weekly
Stage 1 only: Improvement in function of ≥ 30% and ≥ 50% as assessed by the average functional subscale score of the WOMAC NRS 3.1 Index at Days 56 and 84 for PPS 1.5mg/kg twice weekly, PPS 2.0mg/kg once weekly, and PPS fixed dose once weekly
Stage 1 only: PGIC scores at Days 56 and 84 for PPS 1.5mg/kg twice weekly, PPS 2.0mg/kg once weekly, and PPS fixed dose once weekly
Stage 1 only: Reduction in knee pain of ≥ 30% and ≥ 50% as assessed by the average pain subscale score of the WOMAC NRS 3.1 Index at Days 56 and 84 for PPS 1.5mg/kg twice weekly, PPS 2.0mg/kg once weekly, and PPS fixed dose once weekly
Stage 1 only: Stage 1 only: Change from baseline at Day 56 and 84 in function as assessed by the average functional subscale score of the WOMAC NRS 3.1 Index for PPS 1.5 mg/kg twice weekly, PPS 2.0mg/kg once weekly, and PPS fixed dose once weekly
Baseline to Day 84
Key secondary: Change from baseline at Day 84 in function as assessed by the average functional subscale score of the WOMAC NRS 3.1 Index
Key secondary: Change from baseline at Day 84 in knee pain as assessed by the average pain subscale score of the WOMAC NRS 3.1 Index
Day 168
Incidence of Treatment-emergent clinical laboratory abnormalities upto end of study
Incidence of treatment-emergent Adverse Event (TEAEs), including serious AEs (SAEs) upto end of study
Number of days of rescue medication used from Day 1 to Day 168
Stage 1 and 2: Incidence of treatment-emergent Adverse Event (TEAEs), including serious AEs (SAEs)
Stage 1 and 2: Number of days of rescue medication used from Day 1 to Day 168
Stage 1 and 2: Treatment-emergent clinical laboratory abnormalities
Day 39
Incidence of clinically significant changes in electrocardiograms (ECG) compared with baseline at Day 15 and 39
Day 39
Stage 1 and 2: Clinically significant changes in electrocardiograms (ECG) compared with baseline (pharmacokinetic [PK] subset only)
Baseline, Day 56
Knee
Day 56
Change from baseline at Days 56, 84, 112, 140, and 168 in Work Productivity and Activity Impairment (WPAI) questionnaire score
Stage 1 and 2: Change from baseline at Days 56, 84, 112, 140, and 168 in Work Productivity and Activity Impairment (WPAI) questionnaire score
Day 11
Change from baseline in knee pain of >=25% and >=50% as assessed by the average pain sub-scale score of the WOMAC® NRS 3.1 Index at Days 11, 25, 39, 56, 84, 112, 140, and 168
Stage 1 and 2: Change from baseline at Days 11, 25, 39, 112, 140 and 168 in function as assessed by the average functional sub-scale score of the WOMAC® Index.
Stage 1 and 2: Change from baseline at Days 11, 25, 39, 112, 140 and 168 in knee pain as assessed by the average pain sub-scale score of the WOMAC® NRS 3.1 Index.
Stage 1 and 2: Change from baseline in knee pain of >=30% and >=50% as assessed by the average pain sub-scale score of the WOMAC® NRS 3.1 Index at Days 11, 25, 39, 112, 140, and 168
Day 11
Change from baseline in function of >=25% and >=50% as assessed by the average functional sub-scale score of the WOMAC® NRS 3.1 Index from baseline at Days 11, 25, 39, 112, 140, and 168
Stage 1 and 2: Improvement in function of >=30% and >=50% as assessed by the average functional sub-scale score of the WOMAC® NRS 3.1 Index from baseline at Days 11, 25, 39, 112, 140, and 168
Day 11
Exporatory: Number of participants with an Anti-Drug-Antibody (ADA) response after treatment
Number of participants with an Anti-Drug-Antibody (ADA) response at Days 11, 25, 39, 56 and 84
Day 11
Change from baseline at Days 11, 25, 39. 56, 84, 112, 140 and 168 in function as assessed by the average functional sub-scale score of the WOMAC® Index.
Day 11
Change from baseline at Days 11, 25, 39, 56, 84, 112, 140, and 168 in Quality of Life (QoL) as assessed by Short Form-36 General Health Survey (SF-36)
Change from baseline at Days 11, 25, 39, 56, 84, 112, 140, and 168 in knee stiffness as assessed by the average stiffness sub-scale score of the WOMAC® NRS 3.1 Index
Change from baseline at Days 11, 25, 39, 56, 84, 112, 140, and 168 overall as assessed by the overall score of WOMAC® NRS 3.1 Index
PGIC scores at Days 39, 56, 84 112, 140, and 168
Stage 1 and 2: Change from baseline at Days 11, 25, 39, 56, 84, 112, 140, and 168 in Quality of Life (QoL) as assessed by Short Form-36 General Health Survey (SF-36)
Stage 1 and 2: Change from baseline at Days 11, 25, 39, 56, 84, 112, 140, and 168 in knee stiffness as assessed by the average stiffness sub-scale score of the WOMAC® NRS 3.1 Index
Stage 1 and 2: Change from baseline at Days 11, 25, 39, 56, 84, 112, 140, and 168 overall as assessed by the overall score of WOMAC® NRS 3.1 Index
Day 11
Exploratory: Correlation of Anti-Drug-Antibody (ADA) response with clinical events
Day 11
Change from baseline at Days 11, 25, 39, 84, 112, 140 and 168 in knee pain as assessed by the average pain sub-scale score of the WOMAC® NRS 3.1 Index.
Day 39
Stage 1 and 2: PGIC scores at Days 39, 112, 140, and 168
Day 39
Percentage of patients meeting Outcomes Measures in Arthritis Clinical Trials-Osteoarthritis Research Society International - Osteoarthritis Research Society International (OMERACT-OARSI) Responder Index at Days 39, 56, 84, 112, 140, and 168
Stage 1 and 2: Outcomes Measures in Rheumatology - Osteoarthritis Research Society International (OMERACT-OARSI) Responder Index response rate at Days 39, 56, 84, 112, 140, and 168
Day 4
Exploratory: PPS PK profile of single and multiple doses based on sparse blood sampling
Stage 1: Change in PPS plasma concentration over time of single and multiple doses at Days 1, 15 and 39
Day 4
Stage 2: Change in PPS plasma concentration over time of single and multiple doses at Days 1, 15 and 39
screening, Day 168
Exploratory: Change in bone shape on MRI from baseline at Day 168
Magnetic Resonance Imaging
Exploratory: Change in joint space width on MRI from baseline at Day 168
Exploratory: Change in subchondral BML area and volume on MRI from baseline at Day 168
Exploratory: Changes in joint synovitis/effusion volume on MRI from baseline on Day 168
Bone and Bones
Exploratory: The effect of PPS on cartilage volume changes on MRI and whether these correlate with clinical outcomes
Exploratory: The effect of PPS on joint space width changes and whether these correlate with clinical outcomes
Exploratory: The effect of PPS on joint synovitis/effusion volume on MRI and whether these correlate with clinical outcomes
Exploratory: The effect of PPS on subchondral BML volume and area on MRI and whether these changes correlate with clinical outcomes

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

4 Treatment Groups

PPS Twice Weekly
1 of 4
PPS Once Weekly
1 of 4
PPS Fixed Dose Once Weekly
1 of 4
Placebo
1 of 4

Experimental Treatment

Non-Treatment Group

938 Total Participants · 4 Treatment Groups

Primary Treatment: Pentosan Polysulfate Sodium · Has Placebo Group · Phase 2 & 3

PPS Twice WeeklyExperimental Group · 2 Interventions: Pentosan Polysulfate Sodium, Pentosan Polysulfate Sodium twice weekly · Intervention Types: Drug, Drug
PPS Once WeeklyExperimental Group · 3 Interventions: Pentosan Polysulfate Sodium once weekly, Pentosan Polysulfate Sodium, Placebo (Sodium Chloride Injection, 0.9%) · Intervention Types: Drug, Drug, Drug
PPS Fixed Dose Once WeeklyExperimental Group · 2 Interventions: Pentosan Polysulfate Sodium Fixed Dose, Placebo (Sodium Chloride Injection, 0.9%) · Intervention Types: Drug, Drug
Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo (Sodium Chloride Injection, 0.9%) · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: stage 1: day 1, 15 and 39 - pre-dose and 2, 4, and 6 hours. pre-dose on days 4, 8, 11, 18, 22, 25, 29, 32 and 36

Who is running the clinical trial?

Paradigm Biopharmaceuticals USA (INC)Lead Sponsor
1 Previous Clinical Trials
938 Total Patients Enrolled
1 Trials studying Osteoarthritis, Knee
938 Patients Enrolled for Osteoarthritis, Knee
Thomas SchnitzerPrincipal InvestigatorNorthwestern University Feinberg School of Medicine
1 Previous Clinical Trials
938 Total Patients Enrolled
1 Trials studying Osteoarthritis, Knee
938 Patients Enrolled for Osteoarthritis, Knee

Eligibility Criteria

Age 18+ · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a clinical diagnosis of OA in the index knee by ACR criteria 1986.
You are female of childbearing potential and you must agree to comply with the protocol specified contraceptive requirements.
You have a minimum pain score of 4 on either of the individual WOMAC NRS 3.1 Index questions of pain on walking on a flat surface or pain on climbing stairs at Screening AND Day 1.
You have an average WOMAC NRS score of 4 to 10 in the index knee at Screening and Day 1.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 31st, 2021

Last Reviewed: November 20th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

Who else is applying?

What state do they live in?
Louisiana33.3%
Florida16.7%
Indiana16.7%
Other33.3%
How old are they?
65+66.7%
18 - 6533.3%
What site did they apply to?
Northwestern University Feinberg School of Medicine25.0%
Fiel Family and Sports Medicine25.0%
Tandem Clinical Research50.0%
What portion of applicants met pre-screening criteria?
Did not meet criteria16.7%
Met criteria83.3%

How responsive is this trial?

Typically responds via
Phone Call100.0%
Most responsive sites:
  1. Fiel Family and Sports Medicine: < 24 hours
Average response time
  • < 1 Day