Clinical Trials Logo
  1. Home
  2. Metabolic Diseases
  3. NCT05208281

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

Metabolic Diseases Clinical Trial

Official title:
Multicenter, Open-Label, Multi-cohort Study to Evaluate Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Drug Product GNR 055 (JSC "GENERIUM", Russia) in Patients With Mucopolysaccharidosis Type II

Clinical Trial Summary

This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.

Clinical Trial Description

GNR-055 is intended for ERT in patient with Mucopolysaccharidosis type II (MPS II), or Hunter syndrome. MPS II is a recessive X-linked inheritance lysosomal storage disease, which is characterized by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (ID2S), caused by a mutation in the ID2S gene. Enzyme deficiency leads to the accumulation of Glycosaminoglycans (GAG) (mainly of heparan and dermatan sulfates) in lysosomes of almost all types of cells of various tissues and organs. The disease is manifested by growth retardation, damage of many organs and systems, severe deformations of bones and joints, gross facial features, pathology of the respiratory and cardiovascular systems, damage to parenchymal organs (hepatosplenomegaly), and hearing impairment. A severe form of the disease occurs with the involvement of the nervous system in the pathological process, including mental retardation, behavior anomalies, and impaired motor function. GNR-055 is a recombinant modified ID2S capable to penetrate the blood-brain barrier and thus expected to prevent neurodegenerative consequences and the cognitive deficit and to attain a significant improvement in the life quality and expectancy of patients with MPS II. Study IDB-MPS-II-III is a multicenter, open-label, multi-cohort study to assess safety, PK and PD, and efficacy of GNR-055 in patients of different age groups with MPS II (Hunter syndrome). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05208281
Study type Interventional
Source AO GENERIUM
Contact Svetlana B. Korotkova, MD, PhD
Phone +7(495) 988 47 94
Email sbkorotkova@generium.ru
Status Recruiting
Phase Phase 2/Phase 3
Start date November 30, 2021
Completion date March 2028
View Details
See also
  Status Clinical Trial Phase
Completed NCT02380846 - The Metabolic Effects of Consuming Carbohydrate With Different Protein Types N/A
Not yet recruiting NCT01916603 - Diet, Physical Activity and Breastfeeding Intervention on Maternal Nutrition, Offspring Growth and Development N/A
Completed NCT02402985 - Comparison of a Plant Protein Diet to a Animal Protein Diet Emphasized in Type 2 Diabetics N/A
Completed NCT01616082 - Effect of Caloric Restriction on Fat Oxidation in Obese Men and Women (Magellan II) N/A
Completed NCT01737164 - Effect of Age on Glucose and Lipid Metabolism N/A
Terminated NCT00935766 - Effect of Fish Oil (Omega-3 Fatty Acids) on Arteries Phase 3
Enrolling by invitation NCT00628199 - Study to Create Potential Cell-Based Therapies to Treat Human Disease and Disability
Recruiting NCT04958109 - Evaluation of Kisspeptin Glucose-Stimulated Insulin Secretion With Oral Glucose Tolerance Test Phase 1
Completed NCT02275429 - Metabolic Disorders in Ultramarathon Runners During and After the Reunion Island Madmen's Diagonal N/A
Completed NCT01968720 - Pilot Study To Assess CAT-2003 in Patients With Severe Hypertriglyceridemia Phase 2
Completed NCT01719913 - The Effect of Gluten on Gut Microbiome and Metabolic Health. N/A
Completed NCT01705782 - The Effects of Amino Acid Supplement During Acute Inflammation. N/A
Completed NCT01708681 - Lean Seafood Intake and Postprandial Metabolism N/A
Completed NCT01435382 - A Pharmacokinetic and Pharmacodynamic Study of PF-04950615 (RN316) in Subjects With Hypercholesterolemia Phase 1
Recruiting NCT02629926 - Transitional Growth Hormone (GH) Use in Growth Hormone Deficient (GHD) Cancer Survivors Phase 4
Completed NCT00707746 - Safety and Efficacy Study of ISIS 301012 (Mipomersen) Administration in High Risk Statin Intolerant Subjects Phase 2
Completed NCT00550706 - Drug Utilization Prevalence in a Pediatric Care Medical Center N/A
Completed NCT03941392 - Nutritional Study in Spanish Pediatric Population
Terminated NCT02440945 - Use of the DBS for the Follow-up of Blood Clinical Parameters of Old People N/A
Not yet recruiting NCT06000605 - Brain Substrate Switch to Ketones and Lactate N/A