A Study of Complement 3 Glomerulopathy (C3G) Patients Treated With Iptacopan Through an Early Access Program in Spain (ROUTE-C3G)

Clinical Experience With Iptacopan in Adult Patients With Complement 3 Glomerulopathy (C3G): A Multicenter, Observational, Retrospective Study of Patients Treated Through an Early Access Program in Spain

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Complement 3 Glomerulopathy

• Study Type: Observational
• Enrollment (Estimated): 35

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients with a diagnosis of C3G who have received at least one dose of iptacopan through an EAP from 01 October 2020 through 30 June 2025.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Iptacopan Cohort; Adult patients with C3G who received iptacopan through an EAP in Spain.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Height		Baseline
Weight		Baseline
Blood Pressure	Systolic and diastolic blood pressure.	Baseline
Baseline Demographics		Baseline
Number and Percentage of Patients by Clinical Characteristic	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	Baseline
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	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	Baseline
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	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	Baseline
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	Total protein excretion from 24-hour collection.	Baseline
Serum Complement Component 3 (C3) Concentration at Last Post-transplant C3G Recurrence		Baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
UPCR FMV During Iptacopan Treatment		Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
UPCR-24h During Iptacopan Treatment	UPCR from 24-hour collection.	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
Proteinuria-24h During Iptacopan Treatment	Total protein excretion from 24-hour collection.	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
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	UACR from 24-hour collection.	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
Albuminuria-24h During Iptacopan Treatment	Total albumin excretion from 24-hour collection.	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
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	DIF intensity: 0 (negative); +1 (mild); +2 (moderate); +3 (strong)	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
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	Degree of IF/TA categories: None; Mild (<25%); Moderate (25-50%); Severe (>50%)	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
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	Light microscopy patterns: Membranoproliferative glomerulonephritis (MPGN); Diffuse endocapillary glomerulonephritis (GN); Mesangial proliferative GN; Diffuse sclerosing GN	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
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)	AESIs include infections by encapsulated bacteria, thyroid abnormality, testicular abnormality, hypersensitivity reaction, malignancy, and thrombocytopenia.	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
Number and Percentage of Patients With Severe AESI	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.	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
Number and Percentage of Patients Hospitalized due to an AESI	AESIs include infections by encapsulated bacteria, thyroid abnormality, testicular abnormality, hypersensitivity reaction, malignancy, and thrombocytopenia.	Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36
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

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Estimated): May 16, 2026
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): September 30, 2026

Study Registration Dates

• First Submitted: May 14, 2026
• First Submitted That Met QC Criteria: May 14, 2026
• First Posted (Actual): May 20, 2026

Study Record Updates

• Last Update Posted (Actual): May 20, 2026
• Last Update Submitted That Met QC Criteria: May 14, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Iptacopan; C3G; Complement 3 Glomerulopathy; Early Access Program; Factor B inhibition; Alternative Complement Pathway
• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Immune System Diseases; Glomerulonephritis; Nephritis; Glomerulonephritis, Membranoproliferative
• Other Study ID Numbers: CLNP023B1ES01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO