Clinical trial NCT06851845: Clinical Outcomes of C3 Glomerulopathy and IC-MPGN in Russia: Hybrid Retrospective - Prospective Study
Official title: Clinical Outcomes of C3 Glomerulopathy and IC-MPGN in Russia: Hybrid Retrospective - Prospective Study
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Kidney Diseases
Keywords: glomerulopathy
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2025-02-28
Enrollment: 100
Sex: ALL
Minimum age (years): 0
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The study will recruit adults (aged 18 years and older) and pediatric patients (legally acceptable representatives) with biopsy-confirmed diagnosis of primary C3G or idiopathic IC-MPGN in the period from March 2024 to February 2027, who will meet the inclusion criteria and none of the exclusion criteria.

Brief summary: The study is planned to study specificity of the clinical course and treatment outcomes of C3 glomerulopathy (C3G) and Immune-complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged under 18 years and 18 years and older in the Russian population in 2025-2028. The primary objective of the study is to estimate the frequency of complete or partial remission 12 months after morphological verification of the diagnosis. Assessment of demographic, clinical and laboratory, morphological characteristics at diagnosis and their relationship with the disease outcomes, primarily the disease progression and development of chronic renal failure, will allow assessing the efficacy of treatment used in real clinical practice, disease prognosis and factors associated with unfavourable outcomes.


Eligibility criteria: Inclusion Criteria:

1. Willing to sign informed consent form. If the patient is under 18 years of age, the parent or legally acceptable representative of the child/adolescent who can participate in this study also signs the consent form.
2. Diagnosis of primary C3G or IC-MPGN confirmed by the results of morphological and clinical studies.
3. The results of serum creatinine level, proteinuria determination obtained not earlier than 4 weeks before the kidney biopsy
4. Index date (kidney biopsy) not earlier than March 2024 and not later than 11 months before signing the ICF
5. The estimated baseline GFR (using the CKD-EPI formula for persons aged over 18 years and the modified Schwarz formula for children) or the measured GFR is ≥30 mL/min per 1.73 m2.

Exclusion Criteria:

1. Patients participating in a clinical trial with the investigational product.
2. Patients with monoclonal gammopathies (myeloma, B-cell lymphoma/lymphocytic leukemia, lymphoplasmacytoma)
3. Patients with systemic autoimmune diseases and vasculitis (systemic lupus erythematosus, systemic sclerosis, dermatomyositis, mixed connective tissue disease, Sjogren syndrome, Henoch-Schönlein purpura, ANCA vasculitis, cryoglobulinemia)
4. Patients with current or recent infections (viral hepatitis B, viral hepatitis C, infective endocarditis or sepsis, infected ventriculoatrial shunts, abscesses, meningococcal and other bacterial infections, protozoan infections)
5. Patients with any clinically significant acute disease within 30 days prior to kidney biopsy
6. Clinically significant concomitant kidney disease
7. Treatment with pegcetacoplan

Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Number and proportion of patients with overall (complete + partial) remission following the standard therapy in Russia
timeFrame: 12 months
Secondary outcomes:
measure: Number and proportion of patients with complete remission
timeFrame: 6 months; 12 months
measure: Number and proportion of patients with partial remission
timeFrame: 6 months; 12 months
measure: Number and proportion of patients with relapse
timeFrame: 6 months; 12 months
measure: Number and proportion of patients with progression
timeFrame: 6 months; 12 months
measure: Number and proportion of patients with progression to end-stage kidney disease
timeFrame: 6 months; 12 months
measure: Number and proportion of died patients
timeFrame: 6 months; 12 months
measure: Time to start of maintenance dialysis
timeFrame: 6 months; 12 months
measure: Time from the diagnosis date to the start date of dialysis
timeFrame: 6 months; 12 months
measure: Time from the start date of treatment to the start date of dialysis
timeFrame: 6 months; 12 months
measure: Time from the diagnosis date to transplantation
timeFrame: 6 months; 12 months
measure: Time from the diagnosis date to the date of complete remission
timeFrame: 6 months; 12 months
measure: Time from the diagnosis date to the date of partial remission
timeFrame: 6 months; 12 months
measure: Time from the diagnosis date to the date of overall remission (complete and partial remission)
timeFrame: 6 months; 12 months
measure: Time to progression
timeFrame: 6 months; 12 months
measure: Number and proportion of patients with a) an increase in eGFR by ≥15% from the baseline; b) decrease in eGFR by ≥15% from the baseline; c) stable eGFR (change up to 15%)
timeFrame: 6 months; 12 months
measure: Time to 30%, 40% and 50% decline in eGFR
timeFrame: 6 months; 12 months
measure: Number and proportion of patients with proteinuria >1 g/24 h and eGFR ≥30 mL/min per 1.73 m2
timeFrame: 6 months; 12 months
measure: Number and proportion of patients with nephrotic syndrome
timeFrame: 6 months; 12 months
measure: Demographic and clinical characteristics of patients with primary C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: Baseline
measure: Number of patients by morphologically verified disease form in patients with primary C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: Baseline
measure: Number of patients with presence of serum complement markers, serum factor H, rare genetic variants
timeFrame: Baseline
measure: Number and proportion of patients stratified by treatment sequence and specific regimen of treatment
timeFrame: Baseline; 6 months; 12 months
measure: Number and proportion of patients that discontinued a specific regimen of treatment at each line of therapy at any time during follow-up and reason for discontinuation for each cohort
timeFrame: Baseline; 6 months; 12 months
measure: Number of exposed doses for non-continuous treatments (e.g., pulse therapy, rituximab infusion) in each cohort
timeFrame: Baseline; 6 months; 12 months
measure: Number of patients by qualitative, semi-quantitative and quantitative scores of active and chronic lesions
timeFrame: Baseline
measure: Number of patients with signs of thrombotic microangiopathy in the biopsy specimen
timeFrame: Baseline
measure: Number of patients with acute tubular necrosis
timeFrame: Baseline
measure: Number of patients with presence of C1q, IgA, IgM, IgG, C3, kappa chain, lambda chain deposits
timeFrame: Baseline
measure: Patient-reported outcomes for C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 12 months
measure: Number of patients (%) with adverse events/serious adverse events by treatment option
timeFrame: 12 months
measure: Change from baseline of systolic and diastolic blood pressure (BP) in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline serum creatinine level (µmol/L) in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline estimated glomerular filtration rate (eGFR) in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline eGFR rate of change over time (eGFR slope) in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline chronic kidney disease (CKD) stage (number of patients, %) in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline red blood cell count in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline RBC casts in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline daily proteinuria (proteinuria in g/24 hours) in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline proteinuria >1 g/24 h in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline eGFR ≥30 mL/min per 1.73 m2 in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
measure: Change from baseline number of patients with nephrotic syndrome (%) in patients with C3G (cohort 1), IC-MPGN (cohort 2), and overall
timeFrame: 6 months; 12 months
Other outcomes:
Locations:
Study officials:
References: