An open-label single-centre pilot study to evaluate the safety and tolerability of pentosan polysulfate sodium in subjects with mucopolysaccharidosis type I (MPS I)

Paradigm Biopharmaceuticals Pty Ltd0 sites10 target enrollmentAugust 18, 2020

ConditionsMucopolysaccharidosis type I (MPS I)Metabolic and Endocrine - Metabolic disorders Human Genetics and Inherited Disorders - Other human genetics and inherited disorders

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Mucopolysaccharidosis type I (MPS I)
Sponsor: Paradigm Biopharmaceuticals Pty Ltd
Enrollment: 10
Status: Recruiting
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

August 18, 2020

End Date

TBD

Last Updated

4 years ago

Study Type

Interventional

Sex

All

Investigators

Sponsor

Paradigm Biopharmaceuticals Pty Ltd

Eligibility Criteria

Inclusion Criteria

•1\.Males and females aged greater than or equal to 5 years
•2\.Documented diagnosis of MPS I
•3\.Must have received primary therapy for MPS I (HSCT (Haemopoietic stem cell transplantation) \+/\- ERT (enzyme replacement therapy))
•4\.For subjects receiving ERT, ERT must have been administered at a stable dose
•5\.Able to walk independently with or without use of an assistive device
•6\.Subjects must be willing and able to comply with all scheduled visits, treatment plan, laboratory tests and other study procedures
•Additional inclusion/exclusion criteria apply

Exclusion Criteria

•1\.Documented or reported history of increased bleeding tendency in the presence or absence of anticoagulant or antiplatelet drugs
•2\.History of idiopathic or immune\-mediated (including heparin\-induced) thrombocytopenia
•3\.Current treatment with anticoagulants or antiplatelet drugs, excluding aspirin less than or equal to 100 mg/day
•4\.Use of opioids or medications on the Prohibited Medications list within 2 weeks of Day 1 and throughout the duration of the study
•5\.Currently active or recent history (within preceding 12 months) of a gastric or duodenal ulcer, or suspicion of GI tract bleeding
•6\.Coagulation parameters (activated partial thromboplastin time \[aPTT]), outside laboratory reference range, platelets \<160,000/µl, or liver function tests (aspartate transaminase \[AST], alanine transaminase \[ALT]) greater than or equal to 1\.5x upper limit of normal range (ULNR) at Screening
•7\.History or evidence of chondrocalcinosis or fibromyalgia
•8\.History or evidence of human immunodeficiency virus (HIV), hepatitis B or hepatitis C
•9\.Major surgery within 12 weeks preceding Day 1 or anticipated surgery in the study period
•10\.Medical history or evidence of any clinically significant active or chronic condition (with the exception of signs and symptoms relating to MPS I) including autoimmune disease involving musculoskeletal system which in the opinion of the Investigator or Sponsor may impact assessment of safety or efficacy parameters or the validity of study results