Odiparcil in patients with mucopolysaccharidosis (MPS) type VI
Research type
Research Study
Full title
A phase IIa study to investigate safety, Pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI.
IRAS ID
232348
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Inventiva S.A.
Eudract number
2017-002158-35
Duration of Study in the UK
1 years, 6 months, days
Research summary
‘Mucopolysaccharidoses (MPS) are a group of rare inherited disorders characterised by a lack of important enzymes responsible for the normal breakdown of sugars called glycosaminoglycans (GAGs) in the body. Medical need for treatment of MPS is still very high. Odiparcil is an orally active compound that allows the production of soluble glycosaminoglycans (GAGs) which results in excretion out the body in urine. This mechanism should lead to a decrease in the build up of GAGs in the body which cause the symptoms that MPS VI patients experience. The safety of odiparcil has been assessed in 700 healthy volunteers in several phase I studies for period of time up to 14 days and in approximately 1100 adult subjects with thrombosis risk in the phase II program for a period of time of up to 16 weeks. Odiparcil was well tolerated in adult subjects.\n24 participants will take part in this study from 2 sites in the UK and Germany. The study consists of 2 parts performed sequentially: first a preliminary safety assessment followed by the core study. 2 patients not treated with Enzyme Replacement Therapy (ERT), will be enrolled in the preliminary safety assessment divided into a screening period (4 weeks), followed by a treatment period (2 weeks) of escalating dose (500 mg and 1000 mg). If odiparcil 1000 mg is well tolerated, those patients will go on into the core study and 22 additional patients will be directly included in core study for 40 weeks, comprising of a screening period (4 weeks), followed by a treatment period (26 weeks) and then a follow-up period (10 weeks), if the participant is found to be eligible. Procedures involve physical examinations, ECGs, blood samples, completion of efficacy assessments and questionnaires.\n\nThis study is sponsored by Inventiva S.A.
REC name
London - Hampstead Research Ethics Committee
REC reference
17/LO/1726
Date of REC Opinion
16 Nov 2017
REC opinion
Further Information Favourable Opinion