What is iptacopan studying for C3 glomerulopathy?

Iptacopan is being studied to see how it affects kidney function and protein levels in patients with C3 glomerulopathy. Researchers want to understand if iptacopan can stabilize kidney health and reduce disease markers compared to placebo. The study is sponsored by Novartis Pharmaceuticals and aims to provide important information for patients with this condition.

Who may be eligible for this C3 glomerulopathy clinical trial?

People aged 12 to 60 years with a confirmed diagnosis of C3 glomerulopathy may be eligible. Both males and females can participate. Participants must have stable kidney function and protein levels, be on certain blood pressure medications, and have received specific vaccinations. Please review eligibility criteria for more details.

Is there any cost to participate in this C3 glomerulopathy clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any travel or related costs.

Actively Recruiting

Phase 3

Age: 12Years - 60Years

All Genders

ID04817618

Phase 3 Study of Iptacopan Compared to Placebo for Treating C3 Glomerulopathy in Patients Aged 12 to 60 Years

Led by Novartis Pharmaceuticals · Updated on 2026-07-13

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the efficacy and safety of iptacopan compared to placebo and standard care in patients with native Complement 3 Glomerulopathy C3G. This Phase 3 pivotal trial aims to assess how iptacopan affects proteinuria levels, kidney function, and kidney tissue changes in both adult and adolescent participants. The study also examines improvements in serum C3 levels as a marker of treatment impact. Participants will be randomly assigned to receive either iptacopan 200 mg twice daily or a matching placebo. Treatment dosing differs slightly for adults and adolescents but follows a twice-daily schedule. The study includes a double-blind treatment period of 12 months, during which kidney biopsies will be performed in adults to evaluate tissue improvements. Vaccinations against certain bacteria are required prior to starting treatment. During the trial, participants will provide urine samples to measure protein levels and have kidney function assessed through estimated glomerular filtration rate eGFR. Safety evaluations include monitoring vital signs, ECGs, and laboratory tests. Fatigue levels will be assessed using a standardized questionnaire. Researchers will follow participants over 6 and 12 months to observe treatment effects and safety, with ongoing assessments during both double-blind and open-label phases.

CONDITIONS

Brief Title

Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.

Who Can Participate

Age: 12Years - 60Years

All Genders

Eligibility Criteria

You may qualify if you...

Male and female participants aged 12 to 60 years at screening
Diagnosis of C3 glomerulopathy confirmed by renal biopsy within 12 months for adults and within 3 years for adolescents
On a maximally recommended or tolerated dose of ACE inhibitors or angiotensin receptor blockers for at least 90 days prior to randomization
Stable doses of other antiproteinuric medications for at least 90 days prior to randomization
Reduced serum C3 levels below 0.85 times the lower limit of normal at screening
Urine protein-to-creatinine ratio (UPCR) of at least 1.0 g/g from first morning urine samples at specified times
Estimated or measured glomerular filtration rate (eGFR or GFR) of at least 30 ml/min/1.73m2 at screening and Day -15
Mandatory vaccination against Neisseria meningitidis and Streptococcus pneumoniae before starting study treatment
Vaccination against Haemophilus influenzae if needed at least 2 weeks before first study treatment, or prophylactic antibiotics if treatment starts sooner

You will not qualify if you...

History of any cell or organ transplantation, including kidney transplant
Rapidly progressive crescentic glomerulonephritis with at least 50% decline in eGFR within 3 months and biopsy showing crescent formation in at least 50% of glomeruli
Kidney biopsy showing more than 50% interstitial fibrosis or tubular atrophy
Monoclonal gammopathy of undetermined significance (MGUS) confirmed by serum free light chain testing or local standard of care
Active bacterial, viral, or fungal infection within 14 days before study treatment
Fever of 38°C (100.4°F) or higher within 7 days before study treatment
History of recurrent invasive infections caused by encapsulated bacteria like Neisseria meningitidis or Streptococcus pneumoniae
Use of complement inhibitors within 6 months before screening
Use of certain immunosuppressants, cyclophosphamide, or systemic corticosteroids above specified doses within 90 days before study drug
Acute post-infectious glomerulonephritis at screening based on investigator opinion

Research Team

Novartis Pharmaceuticals

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Phase 3 Study of Iptacopan Compared to Placebo for Treating C3 Glomerulopathy in Patients Aged 12 to 60 Years

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria