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2023 ACMG Annual Clinical Genetics Meeting Digital ...
Results of an Open-label, Single-Center, Clinical ...
Results of an Open-label, Single-Center, Clinical Study Evaluating the Safety and Tolerability of Pentosan Polysulfate Sodium in Mucopolysaccharidosis I Subjects
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A phase 2 clinical trial investigated the therapeutic effects of Pentosan Polysulfate Sodium (PPS) on knee osteoarthritis. The trial aimed to assess the safety and tolerability of PPS and explore its efficacy in reducing pain, inflammation, and promoting cartilage protection. The study included physical assessments of mobility and dexterity such as the 2-minute walk test, 6-minute walk test, and 9-hole peg test. Patient-reported outcomes were measured using various forms of the Patient-Reported Outcomes Measurement Information System (PROMIS) and the Patient Global Impression of Change (PGIC). Safety assessments included monitoring treatment-emergent adverse events and changes in clinical laboratory data. Four subjects with MPS I-Hurler, both male and female, were enrolled in the study.<br /><br />The results showed that PPS was well tolerated at doses of 0.75mg/kg or 1.5mg/kg over 48 weeks of dosing. Subjects reported improved mobility and function based on the physical assessments. Patient-reported outcomes indicated improvements in pain and quality of life. The study also highlighted the mechanisms of action of PPS in reducing inflammation, pain, and preserving tissue integrity. The authors concluded that PPS shows promise in improving pain and function issues related to MPS I.<br /><br />Mucopolysaccharidosis I (MPS I) is a rare genetic disorder caused by a deficiency of the enzyme α-L-iduronidase, leading to the accumulation of glycosaminoglycans (GAGs) and resulting in multiorgan dysfunction. The current treatment approaches for MPS I include enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT). PPS, a polysulfated semi-synthetic non-biologic, has shown potential therapeutic effects in reducing pain and inflammation in osteoarthritis and may provide beneficial effects in MPS I.<br /><br />The study was funded by Paradigm Biopharmaceuticals Ltd. PPS was found to be safe and well-tolerated, with no serious adverse events reported. PPS treatment showed improvements in pain and physical function in patients with MPS I. The authors acknowledged the contributions of other researchers and the funding support of Paradigm Biopharmaceuticals Ltd.
Asset Subtitle
Co-Author - Curtis Gravance, PhD; Co-Author - Drago Bratkovic, MD MBBS FRACP; Co-Author - David Ketteridge, MD; Presenting Author - Michael Imperiale, MD; Co-Author - Ravi Krishnan, PhD; Co-Author - Divya Navuru, BPharm, CCDM;
Meta Tag
Enzyme Replacement Therapy
Lysosomal Diseases
Therapy
Co-Author
Curtis Gravance, PhD
Co-Author
Drago Bratkovic, MD MBBS FRACP
Co-Author
David Ketteridge, MD
Co-Author
Ravi Krishnan, PhD
Co-Author
Divya Navuru, BPharm, CCDM
Presenting Author
Michael Imperiale, MD
Keywords
Pentosan Polysulfate Sodium
PPS
knee osteoarthritis
therapeutic effects
safety and tolerability
pain reduction
inflammation reduction
cartilage protection
physical assessments
patient-reported outcomes
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