Clinical Trials Logo

Clinical Trial 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.


Clinical Trial Description

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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03370653
Study type Interventional
Source Inventiva Pharma
Contact
Status Completed
Phase Phase 2
Start date December 30, 2017
Completion date October 22, 2019

See also
  Status Clinical Trial Phase
Terminated NCT01675674 - Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics N/A
Completed NCT00067470 - Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI Phase 3
Completed NCT00048620 - Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI Phase 1
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Completed NCT01707433 - Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease N/A
Active, not recruiting NCT03632213 - Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI Phase 2
Terminated NCT00748969 - Clinical Trial of Growth Hormone in MPS I, II, and VI Phase 2/Phase 3
Recruiting NCT05619900 - Registry of Patients Diagnosed With Lysosomal Storage Diseases
Completed NCT00299000 - A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI Phase 4
Completed NCT00176917 - Stem Cell Transplantation for Hurler Phase 2
Enrolling by invitation NCT05368038 - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Completed NCT00048711 - Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI Phase 2
Active, not recruiting NCT03153319 - Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI Phase 1/Phase 2
Completed NCT00104234 - Study of rhASB in Patients With Mucopolysaccharidosis VI Phase 3
Recruiting NCT06036693 - MPS (RaDiCo Cohort) (RaDiCo-MPS)
Active, not recruiting NCT00005900 - Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation N/A
Completed NCT01586455 - Human Placental-Derived Stem Cell Transplantation Phase 1