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

NCT03370653 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2019-11-01

No results posted yet for this study

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.

Conditions

  • Mucopolysaccharidosis VI

Interventions

DRUG

Odiparcil

Investigational product: odiparcil 250 mg tablets

OTHER

Placebo

Comparator: placebo tablets similar to odiparcil 250 mg tablets

Sponsors & Collaborators

  • Inventiva Pharma

    lead INDUSTRY

Principal Investigators

  • Derralynn HUGHES, MD · Royal Free Hospital, London UK

  • Julia HENNERMANN, MD · Villa Metabolica, Mainz GERMANY

  • Nathalie GUFFON-FOUILHOUX, MD · Hôpital Femme-Mère-Enfant

  • Elisa LEAO-TELES, MD · Centro Hospitalar S. João, Porto, Portugal

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-12-30
Primary Completion
2019-09-24
Completion
2019-10-22

Countries

  • France
  • Germany
  • Portugal
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03370653 on ClinicalTrials.gov