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A Clinical Trial with KJ103 in Anti-GBM Disease

Phase 2
Not yet recruiting
Conditions
Anti-Glomerular Basement Membrane Disease
Interventions
Drug: KJ103 for Injection
Drug: Glucocorticoids
Procedure: Plasma exchange (PE)
Registration Number
NCT06607016
Lead Sponsor
Shanghai Bao Pharmaceuticals Co., Ltd.
Brief Summary

An open-label, single-arm Phase II study to evaluate the preliminary efficacy, safety, pharmacokinetics, pharmacodynamics and immunogenicity of KJ103 in patients with anti-GBM disease.

Detailed Description

Anti-glomerular basement membrane (GBM) disease is a severe, rare autoimmune disorder with an internationally reported incidence of 0.5-1/1 million. It is defined as a vasculitis in which anti-GBM antibodies affect glomerular capillaries, pulmonary capillaries or both. Pulmonary involvement leads to pulmonary haemorrhage and renal involvement can lead to glomerulonephritis with necrosis and crescents.

Anti-GBM disease is a severe autoimmune disorder characterised by rapidly progressive glomerulonephritis and positive anti-GBM antibodies. Antibodies can be found in the circulation and deposited in the lungs and kidneys, mediating renal injury through complement activation and recruitment of inflammatory cells. If left untreated, the vast majority of patients will progress to end-stage renal disease (ESRD) or die from pulmonary haemorrhage. Early detection and measures to reduce anti-GBM antibody levels have the potential to alter prognosis and protect renal function. Unfortunately, many patients with anti-GBM disease are diagnosed late and renal function is not restored even with an aggressive treatment regimen of plasma exchange (PE) combined with immunosuppression. Current KDIGO (Kidney Disease Improving Global Prognosis Organisation) guidelines state that the clinical treatment of anti-GBM disease is a combination of glucocorticoids, cyclophosphamide and PE. Despite treatment, patients continue to produce anti-GBM antibodies in their bodies. Rebound anti-GBM antibodies are usually indicative of adverse renal outcomes and PE must be initiated to remove the rebound antibodies.PE is an effective means of removing circulating anti-GBM antibodies, but only removes about 1/3 of the percentage per treatment, so multiple treatments are required to achieve complete removal.

This is a single-arm Phase II study designed to evaluate the preliminary efficacy, safety, pharmacokinetics, pharmacodynamics and immunogenicity of KJ103 in patients with anti-GBM disease. The trial is expected to enrol 9 to 12 subjects who will receive KJ103 treatment.

Recruitment & Eligibility

Status
NOT_YET_RECRUITING
Sex
All
Target Recruitment
12
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Experimental GroupKJ103 for InjectionKJ103 +SOC(SoC consists of a standardized combination of PE, Cyclophosphamide, and glucocorticoids)
Experimental GroupCyclophosphamideKJ103 +SOC(SoC consists of a standardized combination of PE, Cyclophosphamide, and glucocorticoids)
Experimental GroupGlucocorticoidsKJ103 +SOC(SoC consists of a standardized combination of PE, Cyclophosphamide, and glucocorticoids)
Experimental GroupPlasma exchange (PE)KJ103 +SOC(SoC consists of a standardized combination of PE, Cyclophosphamide, and glucocorticoids)
Primary Outcome Measures
NameTimeMethod
Renal functionday 90, day 180

Proportion of subjects with renal function after KJ103 administration.

Secondary Outcome Measures
NameTimeMethod
Adverse eventsday 180

Assessing the incidence and severity of adverse events (AEs) and serious adverse events (SAEs) via the Common Terminology Criteria for Adverse Events (CTCAE) v5.0.

Proportion and number of subjects requiring PEday 180

Proportion and number of subjects requiring PE after KJ103 administration.

Anti-GBM antibodiesday 180

Number of days positive for anti-GBM antibodies after KJ103 administration.

Immunogenicityday 180

Immunogenicity of KJ103 (Anti-KJ103 antibody) in patients with anti-GBM disease.

eGFR and change from baseline.Day28, Day60, Day90, Day120, Day150, Day180

Estimated glomerular filtration rate (eGFR). Glomerular filtration rate is an estimate of the amount of ultrafiltrate produced by each side of the kidney per unit of time and is an indicator of kidney function.

Pharmacokinetics of KJ103 (Cmax)day 7

Maximum observed serum concentration of KJ103 following dosing (Cmax)

Pharmacokinetics of KJ103 (AUC)day 7

Area under the serum concentration versustime curve (AUC)

Pharmacokinetics of KJ103 (t1/2)day 7

Half-life of KJ103

Pharmacokinetics of KJ103 (CL)day 7

Clearance(CL) is a measure of the ability of the body to clear KJ103

Pharmacokinetics of KJ103 (Vz)day 7

Vz = Volume of distribution during the elimination phase

Pharmacodynamic profile (Serum IgG levels)day 180

Serum IgG levels after KJ103 dosing

Pharmacodynamics-Anti neutrophil cytoplasmic antibodies (ANCA)day 180

Serum Anti neutrophil cytoplasmic antibodies (ANCA) levels after KJ103 dosing

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