Mucopolysaccharidosis VI Clinical Trial
Official title:
A Phase IIa Study to Investigate Safety, Pharmacokinetics, and Efficacy of Odiparcil in Patients 16 Years and Above With Mucopolysaccharidosis (MPS) Type VI
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.
Study design: This phase IIa study consists of 2 parts performed sequentially: a preliminary
safety assessment followed by the core study with a double-blind, randomized, dose-ranged
cohort of patients receiving Enzyme Replacement Therapy (ERT) and an open-label cohort of
patients not receiving ERT.
Preliminary safety assessment (N=2): open-label, escalating dose (2 doses) study. If
acceptable safety profile is achieved, patients will be then included in the open-label arm
of the core study.
Core study
Core study will be conducted on 2 populations in parallel:
- A first cohort (N=18): MPS VI patients receiving ERT assigned in 3 arms:
- Placebo (N=6)
- Odiparcil 500 mg per day (250 mg BID) (N=6)
- Odiparcil 1000 mg per day (500 mg BID) (N=6).
- A second cohort (N=6): MPS VI patient not receiving ERT (odiparcil 1000 mg per day (500
mg BID)).
Study duration: The overall study duration will be 20 months, including the 10-month
enrolment period.
For each patient, the study duration will be:
- Preliminary safety assessment: 6 weeks including a 4-week run-in period followed by
2-week treatment period. Then, patients will go on treatment period in core study.
- Core study: 34 weeks including a 4-week run-in period followed by 26-week treatment
period and 4-week of follow-up.
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