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

NCT05208281 | Recruiting Phase 2 DMC

• 1 other identifier: IDB-MPS-II-III
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 5

Brief 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.

Conditions

Mucopolysaccharidosis Type II; Metabolic Diseases

Keywords

Mucopolysaccharidosis type II; Cognitive Dysfunction; Metabolic Diseases; Lysosomal Storage Diseases; Neurocognitive Disorders; Metabolism, Inborn; Genetic Diseases, Inborn; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Hunter syndrome; Iduronate-2-sulfatase; Modified I2S protein; Connective Tissue Diseases; Mental Disorders; Intellectual Disability; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Cognition Disorders; Mental Retardation, X-Linked

Outcome Measures

Primary Outcomes (2)

• Incidence of Adverse events (AEs) and Serious Adverse Events (SAEs)

  Safety assessment will be performed based on the subjective complaints, physical examination, assessment of vital signs, laboratory tests, and 12-lead ECG; Incidence of allergic and infusion-related reactions; Incidence of Anti-Drug Antibodies (ADAs) against GNR-055 and their neutralizing activity.

  Baseline to Week 56

• Urine GAG excretion

  Changes in levels of urine GAG excretion after multiple-dose administration of GNR-055

  Baseline to Week 4, 8, 10, 26, and 52

Secondary Outcomes (12)

• Serum concentration of the GNR-055

  Week 52

• GAG level in CerebroSpinal Fluid (CSF)

  Baseline to Week 6, 10, 26, and 52

• Serum GAG level

  Baseline to Week 4, 8, 10, 26, and 52

• Large joint range of motion

  Week 8, 10, 26, and 52

• Liver and spleen volumes (MRI)

  Baseline to Week 8, 10, 26, and 52

Study Arms (3)

Adult: GNR-055

EXPERIMENTAL

GNR-055: 1.0-2.0-3.0 mg/kg

Drug: GNR-055 1.0-2.0-3.0 mg/kg

Paediatric: GNR-055 2.0 mg/kg

EXPERIMENTAL

GNR-055 2.0 mg/kg

Drug: GNR-055 2.0 mg/kg

Paediatric: GNR-055 3.0 mg/kg

EXPERIMENTAL

GNR-055 3.0 mg/kg

Drug: GNR-055 3.0 mg/kg

Interventions

GNR-055 1.0-2.0-3.0 mg/kg DRUG

Weekly IV infusion (lyophilized powder) 1.0-2.0-3.0 mg/kg

Also known as: GNR-055

Adult: GNR-055

GNR-055 2.0 mg/kg DRUG

Weekly IV infusion (lyophilized powder) 2.0 mg/kg

Also known as: GNR-055

Paediatric: GNR-055 2.0 mg/kg

GNR-055 3.0 mg/kg DRUG

Weekly IV infusion (lyophilized powder) 3.0 mg/kg

Also known as: GNR-055

Paediatric: GNR-055 3.0 mg/kg

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed inform consent;
• Verified diagnosis of MPS II (Hunter syndrome);
• Naïve patients or patients who have received standard ERT whit idursulfase products;
• No contraindications for lumbar puncture as judged by the Investigator;
• Willingness and ability to follow study procedures.

You may not qualify if:

• Clinically pronounced hypersensitivity to ID2S or any other component of the drug product;
• History of hematopoietic stem cell transplantation (HSCT) or bone marrow transplantation;
• Implanted or external non-removable metal devices, a cardiac pacemaker, or other objects sensitive to the magnetic field that may pose a danger to both the wearer and the correct operation of magnetic resonance imaging (MRI) equipment;
• Concomitant diseases and conditions that, in the Investigator's opinion, can put at risk the patient's safety during his/her participation in the study, or which will influence the safety data analysis in case of the disease/condition exacerbation during the study.

Sponsors & Collaborators

• AO GENERIUM: lead

Study Sites (5)

Federal State-Funded Healthcare Institution Central Clinical Hospital of the Russian Academy of Sciences (Research Institute of Pediatrics and Child Health Protection of the Central Clinical Hospital of the Russian Academy of Sciences)

Moscow, 119333, Russia

NOT YET RECRUITING

Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University" of the Ministry of Health of the Russian Federation

Saint Petersburg, 194100, Russia

NOT YET RECRUITING

V.I. Vernadsky Crimean Federal University

Simferopol, 295007, Russia

RECRUITING

State Budgetary Healthcare Institution Republican Medical Genetic Center

Ufa, 450076, Russia

RECRUITING

State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital

Yekaterinburg, 620149, Russia

RECRUITING

MeSH Terms

Conditions

Mucopolysaccharidosis II; Metabolic Diseases; Cognitive Dysfunction; Lysosomal Storage Diseases; Neurocognitive Disorders; Genetic Diseases, Inborn; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Sudden Infant Death; Connective Tissue Diseases; Mental Disorders; Intellectual Disability; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Cognition Disorders; X-Linked Intellectual Disability

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Mucinoses; Skin and Connective Tissue Diseases; Nutritional and Metabolic Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Death, Sudden; Death; Pathologic Processes; Infant Death; Neurodevelopmental Disorders; Neurodegenerative Diseases

Study Officials

• Oksana A. Markova, MD, MSc

  AO GENERIUM

  STUDY DIRECTOR

Central Study Contacts

Svetlana B. Korotkova, MD, PhD

CONTACT

+7(495) 988 47 94; sbkorotkova@generium.ru

Oksana A. Markova, MD, MSc

CONTACT

+7(495) 988 47 94; oamarkova@generium.ru

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Sequential Assignment

Study Record Dates

• First Submitted: October 14, 2021
• First Posted: January 26, 2022
• Study Start: November 30, 2021
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): March 1, 2028
• Last Updated: July 30, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

RU (5)