A Study in MPS VI to Assess Safety and Efficacy of Odiparcil

Exclusion Criteria

• •1. Use of any investigational product or investigational medical device within 30 days prior to screening. This will include product bought over the counter specifically compounds like genistein and pentosane polysulphate which may not be considered as investigational products by patients and some health care professionals.
• •2. Concurrent disease or condition that would interfere with study participation or pose a safety concern for example patient with: severe cardiac insufficiency as define NYHA class \> II, and severe restrictive chronic respiratory insufficiency as reflected by serum \[HCO3-\] ≥28 mEq/L.
• •3. Subjects who had surgery within 3 months before study starts, or for whom surgery is planned during study period.
• •4. Patient with spinal cord compression requiring surgical intervention.
• •5. Subjects with the following liver test anomalies: any ALT, AST \> 3xULN or bilirubin \>1.5xULN (except if Gilbert syndrome) at screening visit.
• •6. Evidence of an immunosuppressive state, including known HIV infection, agammaglubilinemias, T-Cell deficiencies.
• •7. Subjects with history of chronic infections, including but not limited to subjects with past history of viral hepatitis C, or B, with recent history of serious or life-threatening infection or any current signs or symptoms that may indicate infection at visit V-1 of study as per investigators clinical judgement.
• •8. History of malignant cancer except of cervical carcinoma in situ, basal cell carcinoma, dermatological squamous cell carcinoma.
• •9. Subjects with significant haematologic abnormalities, such as haemoglobin \<8 g/dL, or WBC\<2000 /mm3 or absolute neutrophil count \<1300 /mm3, or platelet \<30.000 /mm3.
• •10. International Normalized Ratio (INR), activated partial thromboplastin time (aPTT) or thrombin time (TT) values above the laboratory reference range at screening. For patients on anti-coagulants, they should be within their target effect on INR and be stable.
• •11. Any history of bleeding diathesis
• •12. Patient with coexistence of corneal pathologies other than corneal clouding (e.g. exposure keratopathy)
• •13. An unwillingness on the part of male patients to abstain from sexual intercourse with pregnant or lactating women; or an unwillingness to use highly effective form of birth control if engaging in sexual intercourse with a woman who could become pregnant from the time of the first dose of study medication until completion of follow-up procedures.
• •14. An unwillingness on the part of female patients to use highly effective form of birth control2 if engaging in sexual intercourse and to have a monthly pregnancy test during treatment and until completion of follow-up procedures.
• •15. Pregnant or lactating women.
• •16. Have a known hypersensitivity to any of the ingredients or excipients of the IMP including: Microcrystalline Cellulose, Povidone, Sodium starch glycolate (type A), Magnesium stearate, Opadry™ II 85F18422
• •1\. Previous hematopoietic stem cell transplant (HSCT)