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

• Status: Recruiting
• Trial end date: 2025-03-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AO GENERIUM

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.