Complement 3 Glomerulopathy
Conditions
Keywords
Complement 3 Glomerulopathy, C3G, Iptacopan, Factor B inhibition, Alternative Complement Pathway, Early Access Program
Brief summary
This study aims to characterize the patient profiles and the clinical experience of iptacopan treatment in adult patients with C3G treated through an early access program (EAP) in Spain.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 100 Years
Healthy volunteers
No
Inclusion criteria
1. Adult patients (≥18 years). 2. Biopsy-confirmed C3G. 3. Patients who have received at least one dose of iptacopan through an EAP from October 2020 to June 2025.
Exclusion criteria
1\. Patients treated with iptacopan through an EAP for indications other than C3G.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Baseline Demographics | Baseline | — |
| Height | Baseline | — |
| Weight | Baseline | — |
| Blood Pressure | Baseline | Systolic and diastolic blood pressure. |
| Number and Percentage of Patients by Clinical Characteristic | Baseline | Clinical characteristics include: * Kidney status * History of dialysis * Presence of edema * Comorbidities * C3G histology subtype * Previous medication * Vaccination status * Receipt of antibiotic prophylaxis in addition to vaccination * History of severe infections |
| Duration of Antibiotic Prophylaxis | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number of Hospitalizations due to Severe Infection Before Iptacopan Initiation | Baseline | — |
| Age at C3G Diagnosis | Baseline | — |
| Number and Percentage of Patients by Clinical Features at First C3G Diagnosis | Baseline | Clinical features include: * Clinical presentation (nephrotic, nephritic, isolated non-nephrotic proteinuria, asymptomatic urinary abnormalities) * Presence of pathogenic variants in complement-related genes * Presence of autoantibodies against complement components |
| Age at Last Kidney Transplantation Before Iptacopan Initiation | Baseline | — |
| Number of Kidney Transplants Before Iptacopan Initiation | Baseline | — |
| Number and Percentage of Patients by Clinical Features of Last Kidney Transplant Before Iptacopan Initiation | Baseline | Clinical features include: * Previous recurrence with kidney failure * Kidney donor type * Highly sensitized (yes/no) * Immediate graft function * Type of induction therapy * Type of maintenance immunosuppressive (IS) therapy * Kidney transplant rejection episode and type of rejection |
| Duration of Cold Ischemia for the Last Kidney Transplant Before Iptacopan Initiation | Baseline | — |
| Post-Kidney Transplant Estimated Glomerular Filtration Rate (eGFR) | Baseline | — |
| Number and Percentage of Patients by Type of Biopsy Performed to Confirm Recurrence of C3G After Kidney Transplant | Baseline | — |
| Duration Between Last Kidney Transplant and Biopsy Confirmed C3G Recurrence | Baseline | — |
| eGFR at Last Post-transplant C3G Recurrence | Baseline | — |
| Serum Albumin Concentration at Last Post-transplant C3G Recurrence | Baseline | — |
| Urine Protein-Creatine Ratio (UPCR) From First-Morning Void (FMV) at Last Post-transplant C3G Recurrence | Baseline | — |
| Proteinuria-24h at Last Post-transplant C3G Recurrence | Baseline | Total protein excretion from 24-hour collection. |
| Serum Complement Component 3 (C3) Concentration at Last Post-transplant C3G Recurrence | Baseline | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| UPCR FMV During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| UPCR-24h During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | UPCR from 24-hour collection. |
| Proteinuria-24h During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | Total protein excretion from 24-hour collection. |
| Urine Albumin-to-Creatinine Ratio (UACR) FMV During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| UACR-24h During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | UACR from 24-hour collection. |
| Albuminuria-24h During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | Total albumin excretion from 24-hour collection. |
| Serum Albumin Concentration During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Serum Creatinine Concentration During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| eGFR During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Difference Between Post- and Pre-Iptacopan Treatment eGFR Slopes | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients With Kidney Failure | Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients With a Kidney Transplant During Follow-up | Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients Who Initiate Dialysis During Follow-up | Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Red Blood Cells (RBC) Count per High Power Field (HPF) During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients by Intensity of C3 Deposit in Direct Immunofluorescence (DIF) During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | DIF intensity: * 0 (negative) * +1 (mild) * +2 (moderate) * +3 (strong) |
| Number and Percentage of Patients by Type of Antibody Used for DIF at Each Biopsy During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients by Presence of Globally Sclerotic Glomeruli and Glomeruli With Crescents at Each Biopsy During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Percentage of Globally Sclerotic Glomeruli and Glomeruli With Crescents at Each Biopsy During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients by Degree of Interstitial Fibrosis/Tubular Atrophy (IF/TA) at Each Biopsy During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | Degree of IF/TA categories: * None * Mild (\<25%) * Moderate (25-50%) * Severe (\>50%) |
| Number and Percentage of Patients by Presence of Arteriosclerosis or Arteriolosclerosis at Each Biopsy During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients by Light Microscopy Pattern at Each Biopsy During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | Light microscopy patterns: * Membranoproliferative glomerulonephritis (MPGN) * Diffuse endocapillary glomerulonephritis (GN) * Mesangial proliferative GN * Diffuse sclerosing GN |
| Number and Percentage of Patients by Presence of Double Contours at Each Biopsy During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients by Presence of Leukocyte Infiltration at Each Biopsy During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Serum C3 Concentration During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Serum Complement Component 4 (C4) Concentration During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Serum Soluble Membrane Attach Complex (sC5b-9) Concentration During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Total Complement Function Assay (CH50) Concentration During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Daily Iptacopan Dose During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number of Missed or Delayed Doses During Iptacopan Treatment | Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients With Missed or Delayed Doses During Iptacopan Treatment | Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients Who Discontinued Iptacopan Treatment | Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients by Reason for Iptacopan Treatment Discontinuation | Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Time to Iptacopan Treatment Discontinuation | Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients by Type of Concomitant Therapies | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Treatment Dose of Concomitant Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients on the Maximum Tolerated Dose of Concomitant Treatment: Renin-angiotensin System (RAS) Blockade | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients by Reason for Stopping Concomitant Treatment | Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients With Adverse Events of Special Interest (AESI) | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | AESIs include infections by encapsulated bacteria, thyroid abnormality, testicular abnormality, hypersensitivity reaction, malignancy, and thrombocytopenia. |
| Number and Percentage of Patients With Severe AESI | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | AESI include infections by encapsulated bacteria, thyroid abnormality, testicular abnormality, hypersensitivity reaction, malignancy, and thrombocytopenia. Infections are categorized as severe if present with any of the following: fever with shivers/chills; headache with fever; fever with rash; fever with chest pain and cough; fever with breathlessness/fast breathing; fever with high heart rate; headache with nausea/vomiting; headache with stiff neck/back; confusion; body aches with flu-like symptoms; clammy skin; eyes sensitive to light. |
| Number and Percentage of Patients Hospitalized due to an AESI | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | AESIs include infections by encapsulated bacteria, thyroid abnormality, testicular abnormality, hypersensitivity reaction, malignancy, and thrombocytopenia. |
| Length of Stay in Hospital | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
| Number and Percentage of Patients who Die During Iptacopan Treatment | Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36 | — |
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed