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

ConditionsMucopolysaccharidosis Type IIMetabolic Diseases
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

This trial does not accept healthy volunteers

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

Design model

Sequential

Treatments tested in this trial

  • GNR-055 1.0-2.0-3.0 mg/kg

    Drug

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

  • GNR-055 2.0 mg/kg

    Drug

    Weekly IV infusion (lyophilized powder) 2.0 mg/kg

  • GNR-055 3.0 mg/kg

    Drug

    Weekly IV infusion (lyophilized powder) 3.0 mg/kg

Treatment groups

32 Participants
are divided into 3 treatment groups
Group A: Adult: GNR-055Experimental treatment 1 intervention
Group B: Paediatric: GNR-055 2.0 mg/kgExperimental treatment 1 intervention
Group C: Paediatric: GNR-055 3.0 mg/kgExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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.

Time frame
Baseline to Week 56
2

Urine GAG excretion

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

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

Secondary outcomes

1

Serum concentration of the GNR-055

Assessment of the serum concentration of GNR-055 and calculation of Cmax, AUC, T1/2, Cl et other parameters after multiple-dose administration

Time frame
Week 52
2

GAG level in CerebroSpinal Fluid (CSF)

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

Time frame
Baseline to Week 6, 10, 26, and 52
3

Serum GAG level

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

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

Large joint range of motion

Changes over time in the large joint range of motion after multiple-dose administration of GNR-055

Time frame
Week 8, 10, 26, and 52

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

AO GENERIUM

Lead sponsor

Source ClinicalTrials.gov
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