Pentosan Polysulfate Sodium (PPS) in subjects with mucopolysaccharidosis type I (MPS I)

Phase 1

Recruiting

• Conditions: Mucopolysaccharidosis type I (MPS I); Metabolic and Endocrine - Metabolic disorders; Human Genetics and Inherited Disorders - Other human genetics and inherited disorders

• Registration Number: ACTRN12620000823976
• Lead Sponsor: Paradigm Biopharmaceuticals Pty Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-08-18
• Last Update Posted: 7 February 2022

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 10

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
11.Current or recent (within 90 days preceding Day 1) immunosuppressive or immune-modulative systemic therapy
12.Any acute illness within 2 weeks of baseline
13.History of untreated drug or alcohol abuse and/or dependence within the 12 months preceding screening
14.Participation in another clinical trial or administration of any investigational product or experimental product within 12 weeks or 5 half-lives (whichever is longer) preceding Day 1
15.History of significant hypersensitivity to PPS or drugs of a similar chemical or pharmacological class
16.Any clinically significant abnormalities (with the exception of abnormalities relating to MPS I) on clinical chemistry, haematology, urinalysis, physical examination, medical history, 12-lead ECG, or vital signs as judged by the investigator which may interfere with participation in study activities

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To evaluate the safety of PPS in subjects with MPS I based on incidence of treatment-emergent adverse events including serious adverse events and change from baseline in clinical laboratory data. Adverse events are rare and may include bleeding issues, assessed by observation and blood samples, cardiac events, monitored by ECG and vital signs, injection site reactions, assessed through observation, nausea through self-report[Safety parameters will be recorded every week for the first 12 weeks then every 2 weeks there-after through week 73.]