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

Recruiting now · Phase 2/Phase 3

Conditions studied: Mucopolysaccharidosis Type II, Metabolic Diseases

In brief

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.

Key facts

Study ID

NCT05208281

Run by

AO GENERIUM

People needed

32

Starts

2021-11-30

Expected to finish

2028-03-01

Last updated by the study team

2025-07-30

Who can join

Age: any. Sex: male. Healthy volunteers: not accepted.

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.

Where it is running

• V.I. Vernadsky Crimean Federal University — Simferopol, Russia (enrolling)
• State Budgetary Healthcare Institution Republican Medical Genetic Center — Ufa, Russia (enrolling)
• State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital — Yekaterinburg, Russia (enrolling)
• 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, Russia
• 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, Russia

Full record on ClinicalTrials.gov

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