A Study in MPS VI to Assess Safety and Efficacy of Odiparcil (NCT03370653) | Clinical Trial Compass
CompletedPhase 2
A Study in MPS VI to Assess Safety and Efficacy of Odiparcil
France, Germany, Portugal20 participantsStarted 2017-12-30
Plain-language summary
Mucopolysaccharidoses (MPS) are a group of rare inherited disorders characterized by a deficiency of lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs). Medical need for treatment of MPS is still very high due to the poor penetration of the recombinant enzymes into the blood brain barrier as well as the ocular barriers and into tissues that are poorly vascularized, such as cartilages and bones. Odiparcil is an orally active compound that allows the synthesis of soluble glycosaminoglycans (GAGs), mainly chondroitin sulfate (CS) and dermatane sulfate (DS). The neosynthesized solubles GAGs are then excreted in urine. By diverting endogenous GAG synthesis to the synthesis of soluble odiparcil linked GAGs, odiparcil should decrease the intracellular pool of GAGs and consequently decrease the lysosomal GAG accumulation.
The primary objective of the study is to assess the safety and efficacy of two doses of odiparcil in MPS VI patients and to provide evidence to enable the selection of the relevant dose of odiparcil for phase III study. The secondary objective of this study is to characterize the dose response, PK and PD of odiparcil.
Who can participate
Age range16 Years
SexALL
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Inclusion criteria
✓. Patients previously treated with ERT but have discontinued for more than 3 months either due to medical decision or personal choice
✓. Patients allergic to ERT therapy
✓. Patients that have had a previous hematopoietic stem cell transplant (HSCT)
✓. Patients not treated with ERT i.e. treatment naïve
Exclusion criteria
✕. 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.
✕. 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.
✕. Subjects who had surgery within 3 months before study starts, or for whom surgery is planned during study period.
What they're measuring
1
Number of patients with modified clinical signs
Timeframe: 26 weeks
2
Number of patients with modified biological values
. Patient with spinal cord compression requiring surgical intervention.
✕. Subjects with the following liver test anomalies: any ALT, AST \> 3xULN or bilirubin \>1.5xULN (except if Gilbert syndrome) at screening visit.
✕. Evidence of an immunosuppressive state, including known HIV infection, agammaglubilinemias, T-Cell deficiencies.
✕. 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.
✕. History of malignant cancer except of cervical carcinoma in situ, basal cell carcinoma, dermatological squamous cell carcinoma.