Safety and tolerability study to evaluate of Pentosan Polysulfate Sodium in treating subjects with Mucopolysaccharidosis type VI
- Conditions
- Mucopolysaccharidosis VIOther mucopolysaccharidoses
- Registration Number
- RBR-2tz9dky
- Lead Sponsor
- Paradigm Biopharmaceuticals Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- Not specified
Male and female subjects greater than or equal to 5 years of age; Confirmed diagnosis by genetic testing and/or enzyme activity of MPS type VI (N-acetylgalactosamine-4-sulfatase deficiency and a second normal sulfatase enzyme); Must be on enzyme replacement therapy (ERT) for greater than or equal to 1 year; Must be on at stable dose of ERT for 3 months prior to baseline; Must have a mean pain score of a minimum of 3 and a maximum of 9 on the Faces Pain Scale – Revised (FPS-R) taken from the first 5 consecutive measurements at screening; Able to walk 30 metres with or without use of an assistive device; 6MWT less than or equal to 70% predicted of normative mean value for age; Subject demonstrates an impairment of ROM in at least one shoulder; Subject or parent, or legally acceptable representative sufficiently able to understand the purposes and risks of the study and able to provide written informed consent or in the case of subjects age less then 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures or study assessment; If applicable, subjects must be willing to comply with the medically acceptable contraceptive requirements of the study from screening to at least 28 days after the last investigational product (IP) administration. Subjects should not be pregnant or breastfeeding at the time of study entry; Subjects must be willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.
Documented or reported history of increased bleeding tendency in the presence or absence of anticoagulant or antiplatelet drugs; History of heparin induced thrombocytopenia; Current treatment with anticoagulants or antiplatelet drugs, excluding aspirin less than or equal to 100 mg per day; Absence of limitation in upper extremity fine motor skills; Absence of shoulder joint ROM limitations; Subject taking opioids within 2 weeks of Day 1 or unwilling to stop the treatment opioids throughout the study; Parenteral iloprost from 12 weeks before Day 1 and throughout the study; Parenteral bisphosphonates from 12 months before Day 1 and throughout the study; Currently active or recent history (within preceding 12 months) of a gastric or duodenal ulcer, or suspicion of gastrointestinal (GI) tract bleeding; Coagulation parameters or platelets outside laboratory reference range, liver function tests greater than or equal to 1.5 × upper limit of normal (ULN), or estimated glomerular filtration rate (eGFR) less then 30 mL per min at Screening; Hepatic synthetic insufficiency (including Gilbert’s Syndrome); History of bone marrow transplant or haematopoietic stem cell transplant; History or evidence of chondrocalcinosis or fibromyalgia; History or evidence of HIV, hepatitis B or hepatitis C; Major surgery within 12 weeks preceding Day 1 or anticipated surgery in the study period; Medical history or evidence of any clinically significant active or chronic condition involving the musculoskeletal system, which in the opinion of the Investigator may impact assessment of safety or efficacy parameters or the validity of study results; Current or recent immunosuppressive or immunomodulatory systemic therapy; Currently hospitalised; Any acute illness within 2 weeks of baseline; A history of drug or alcohol abuse and/or dependence as documented by subject/caregiver reporting within the 12 months preceding screening; Participation in another clinical trial or administration of any IP or experimental product within 12 weeks or five half-lives (whichever is longer) preceding Day 1; History of significant hypersensitivity to PPS or drugs of a similar chemical or pharmacological class; Any clinically significant abnormalities as judged by the Investigator at screening; History of or current clinically significant GI, hepatic, renal, cardiovascular, respiratory, endocrine, oncological, immunological, neurological, ophthalmological (including existing pigmentary maculopathy), haematological or psychiatric disorder or any other condition (with the exception of signs and symptoms relating to MPS VI), which in the opinion of the Investigator or Sponsor would jeopardize the safety of the subject or the validity of the study results; An employee of the Sponsor or research site personnel directly affiliated with this study or their immediate family members defined as a spouse, parent, sibling, or child, whether biological or legally adopted;
Study & Design
- Study Type
- Intervention
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1. To evaluate the incidence of treatment emergent adverse events (TEAEs) including serious adverse events (SAEs). The number and percentage of participants experiencing any TEAEs (Treatment part only), SAEs and AEs overall will be tabulated.;2. To evaluate treatment-emergent clinical laboratory abnormalities. Values for vital signs, hematology and clinical chemistry parameters will be tabulated.;3. To evaluate safety and tolerability assess assessed by clinically relevant changes in electrocardiogram (ECG) compared to baseline and funduscopic retinal examination compared to baseline.
- Secondary Outcome Measures
Name Time Method