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 个研究点目标入组 10 人开始时间: 2020年8月18日最近更新: 2022年2月7日

适应症

试验速览

阶段: 1 期
状态: 招募中
发起方
入组人数: 10

研究概览研究设计入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional
分配方式: Non-randomised trial
主要目的: Treatment

入排标准

年龄范围: 5 Years 至 o limit（—）
性别: All

入选标准

•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

排除标准

•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

研究者

发起方

Paradigm Biopharmaceuticals Pty Ltd

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