Clinical Trial Details
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NCT03370653 : A Study in MPS VI to Assess Safety and Efficacy of Odiparcil
PhasePhase 2
AgesMin: 16 Years Max: N/A
Main criteria for inclusion: Inclusion and exclusion criteria are similar for both the
preliminary safety assessment and the core study. Patients enrolled in the preliminary
safety assessment will satisfy criteria of Not ERT treated group.

Inclusion criteria for the entire cohort:

1. Male or female gender.

2. Age ?16 years.

3. Diagnosis of MPS VI, demonstrated by a reduced Arylsulfatase B (ARSB) activity
relative to the normal range of the laboratory performing the assay in either white
blood cells or fibroblast culture or confirmation of two known disease causing
mutations in the ARSB gene.

4. Urine GAG above upper limit of normal (ULN) at screening visit.

5. Willing and able to provide written, dated, signed informed consent, or in the case of
subjects age < 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.

6. Able to comply with all study procedures.

7. Women with childbearing potential must agree to use a highly effective method of birth
control during the study and at least 10 weeks after last administration.

Women with childbearing potential are required to have a confirmed negative blood pregnancy
test before starting medication administration at baseline (V0) and repeatedly during the

Inclusion criteria for ERT treated group:

1. Patients with MPS Type VI receiving enzyme replacement therapy (Naglazyme) for at least
6 months on the licensed dosage or as per local guidelines.

Inclusion criteria for not ERT treated group:

Patients with MPS Type VI not receiving enzyme replacement therapy for the following

1. Patients previously treated with ERT but have discontinued for more than 3 months
either due to medical decision or personal choice

2. Patients allergic to ERT therapy

3. Patients that have had a previous hematopoietic stem cell transplant (HSCT)

4. Patients not treated with ERT i.e. treatment naïve

Exclusion criteria:

Exclusion criteria for the entire cohort:

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

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

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

Exclusion criteria for ERT treated group:

1. Previous hematopoietic stem cell transplant (HSCT)
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