A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexMale
AgeNot listed
SponsorAO GENERIUM
About this trial
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.
Eligibility criteria
Qualifiers
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;
Disqualifiers
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.
Trial design
Treatments tested in this trial
- GNR-055 1.0-2.0-3.0 mg/kg
- GNR-055 2.0 mg/kg
- GNR-055 3.0 mg/kg
Treatment groups
32 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov