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

Launched by AO GENERIUM · Jan 13, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called GNR-055 for patients with Mucopolysaccharidosis Type II, also known as Hunter syndrome. The goal is to find out how safe the treatment is, how it works in the body, and whether it effectively helps manage the symptoms of this condition. The study is currently looking for male participants of different ages who have been diagnosed with Hunter syndrome.

To be eligible for the trial, participants must provide signed consent, have a confirmed diagnosis of Hunter syndrome, and either be new to treatment or have previously received another standard treatment. They should also be able to safely undergo a procedure called a lumbar puncture, which involves collecting a small sample of fluid from the spine. During the trial, participants will follow specific procedures and receive close monitoring to ensure their safety. It’s important to note that individuals with certain medical history, such as previous stem cell transplants or specific metal implants, may not be able to join the study. If you or someone you know is interested in participating, this trial could provide valuable information about a potential new treatment for Hunter syndrome.

Gender

MALE

Eligibility 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.