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NCT Number: NCT07598448

A Study of Complement 3 Glomerulopathy (C3G) Patients Treated With Iptacopan 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.

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Key information

Conditions

Complement 3 Glomerulopathy Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Glomerulonephritis Glomerulonephritis, Membranoproliferative Immune System Diseases Kidney Diseases Male Urogenital Diseases Nephritis Urogenital Diseases Urologic Diseases

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Observational

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Adult patients (≥18 years).
  • Biopsy-confirmed C3G.
  • Patients who have received at least one dose of iptacopan through an EAP from October 2020 to June 2025.

Exclusion criteria

  • Patients treated with iptacopan through an EAP for indications other than C3G.

Treatment and study plan

Primary outcomes

  1. Baseline Demographics

    Time frame: Baseline

  2. Height

    Time frame: Baseline

  3. Weight

    Time frame: Baseline

  4. Blood Pressure

    Time frame: Baseline

    Systolic and diastolic blood pressure.

  5. Number and Percentage of Patients by Clinical Characteristic

    Time frame: 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

  6. Duration of Antibiotic Prophylaxis

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  7. Number of Hospitalizations due to Severe Infection Before Iptacopan Initiation

    Time frame: Baseline

  8. Age at C3G Diagnosis

    Time frame: Baseline

  9. Number and Percentage of Patients by Clinical Features at First C3G Diagnosis

    Time frame: 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

  10. Age at Last Kidney Transplantation Before Iptacopan Initiation

    Time frame: Baseline

  11. Number of Kidney Transplants Before Iptacopan Initiation

    Time frame: Baseline

  12. Number and Percentage of Patients by Clinical Features of Last Kidney Transplant Before Iptacopan Initiation

    Time frame: 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

  13. Duration of Cold Ischemia for the Last Kidney Transplant Before Iptacopan Initiation

    Time frame: Baseline

  14. Post-Kidney Transplant Estimated Glomerular Filtration Rate (eGFR)

    Time frame: Baseline

  15. Number and Percentage of Patients by Type of Biopsy Performed to Confirm Recurrence of C3G After Kidney Transplant

    Time frame: Baseline

  16. Duration Between Last Kidney Transplant and Biopsy Confirmed C3G Recurrence

    Time frame: Baseline

  17. eGFR at Last Post-transplant C3G Recurrence

    Time frame: Baseline

  18. Serum Albumin Concentration at Last Post-transplant C3G Recurrence

    Time frame: Baseline

  19. Urine Protein-Creatine Ratio (UPCR) From First-Morning Void (FMV) at Last Post-transplant C3G Recurrence

    Time frame: Baseline

  20. Proteinuria-24h at Last Post-transplant C3G Recurrence

    Time frame: Baseline

    Total protein excretion from 24-hour collection.

  21. Serum Complement Component 3 (C3) Concentration at Last Post-transplant C3G Recurrence

    Time frame: Baseline

Secondary outcomes

  1. UPCR FMV During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  2. UPCR-24h During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

    UPCR from 24-hour collection.

  3. Proteinuria-24h During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

    Total protein excretion from 24-hour collection.

  4. Urine Albumin-to-Creatinine Ratio (UACR) FMV During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  5. UACR-24h During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

    UACR from 24-hour collection.

  6. Albuminuria-24h During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

    Total albumin excretion from 24-hour collection.

  7. Serum Albumin Concentration During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  8. Serum Creatinine Concentration During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  9. eGFR During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  10. Difference Between Post- and Pre-Iptacopan Treatment eGFR Slopes

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  11. Number and Percentage of Patients With Kidney Failure

    Time frame: Months 3, 6, 9, 12, 18, 24, 30, and 36

  12. Number and Percentage of Patients With a Kidney Transplant During Follow-up

    Time frame: Months 3, 6, 9, 12, 18, 24, 30, and 36

  13. Number and Percentage of Patients Who Initiate Dialysis During Follow-up

    Time frame: Months 3, 6, 9, 12, 18, 24, 30, and 36

  14. Red Blood Cells (RBC) Count per High Power Field (HPF) During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  15. Number and Percentage of Patients by Intensity of C3 Deposit in Direct Immunofluorescence (DIF) During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

    DIF intensity:

    • 0 (negative)
    • +1 (mild)
    • +2 (moderate)
    • +3 (strong)

  16. Number and Percentage of Patients by Type of Antibody Used for DIF at Each Biopsy During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  17. Number and Percentage of Patients by Presence of Globally Sclerotic Glomeruli and Glomeruli With Crescents at Each Biopsy During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  18. Percentage of Globally Sclerotic Glomeruli and Glomeruli With Crescents at Each Biopsy During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  19. Number and Percentage of Patients by Degree of Interstitial Fibrosis/Tubular Atrophy (IF/TA) at Each Biopsy During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

    Degree of IF/TA categories:

    • None
    • Mild (<25%)
    • Moderate (25-50%)
    • Severe (>50%)

  20. Number and Percentage of Patients by Presence of Arteriosclerosis or Arteriolosclerosis at Each Biopsy During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  21. Number and Percentage of Patients by Light Microscopy Pattern at Each Biopsy During Iptacopan Treatment

    Time frame: 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

  22. Number and Percentage of Patients by Presence of Double Contours at Each Biopsy During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  23. Number and Percentage of Patients by Presence of Leukocyte Infiltration at Each Biopsy During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  24. Serum C3 Concentration During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  25. Serum Complement Component 4 (C4) Concentration During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  26. Serum Soluble Membrane Attach Complex (sC5b-9) Concentration During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  27. Total Complement Function Assay (CH50) Concentration During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  28. Daily Iptacopan Dose During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  29. Number of Missed or Delayed Doses During Iptacopan Treatment

    Time frame: Months 3, 6, 9, 12, 18, 24, 30, and 36

  30. Number and Percentage of Patients With Missed or Delayed Doses During Iptacopan Treatment

    Time frame: Months 3, 6, 9, 12, 18, 24, 30, and 36

  31. Number and Percentage of Patients Who Discontinued Iptacopan Treatment

    Time frame: Months 3, 6, 9, 12, 18, 24, 30, and 36

  32. Number and Percentage of Patients by Reason for Iptacopan Treatment Discontinuation

    Time frame: Months 3, 6, 9, 12, 18, 24, 30, and 36

  33. Time to Iptacopan Treatment Discontinuation

    Time frame: Months 3, 6, 9, 12, 18, 24, 30, and 36

  34. Number and Percentage of Patients by Type of Concomitant Therapies

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  35. Treatment Dose of Concomitant Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  36. Number and Percentage of Patients on the Maximum Tolerated Dose of Concomitant Treatment: Renin-angiotensin System (RAS) Blockade

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  37. Number and Percentage of Patients by Reason for Stopping Concomitant Treatment

    Time frame: Months 3, 6, 9, 12, 18, 24, 30, and 36

  38. Number and Percentage of Patients With Adverse Events of Special Interest (AESI)

    Time frame: 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.

  39. Number and Percentage of Patients With Severe AESI

    Time frame: 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.

  40. Number and Percentage of Patients Hospitalized due to an AESI

    Time frame: 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.

  41. Length of Stay in Hospital

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

  42. Number and Percentage of Patients who Die During Iptacopan Treatment

    Time frame: Baseline, Months 3, 6, 9, 12, 18, 24, 30, and 36

Study contacts

Contact information is provided by the study sponsor or research team.

Novartis Pharmaceuticals

CONTACT

[email protected]

+41613241111

Novartis Pharmaceuticals

CONTACT

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Acronym: ROUTE-C3G

Important dates

Study start
2026
Primary completion
2026
Study completion
2026
First posted
May 20, 2026
Registry last updated
Jul 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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