Clinical Trials for C3 Glomerulopathy
There are currently 4 clinical trials studying treatments for C3 glomerulopathy, a rare kidney disease characterized by abnormal protein deposits in the kidney’s filtering units. These trials are testing various medications aimed at reducing kidney inflammation and protecting kidney function, with studies taking place across multiple European countries.
Clinical trial locations
- Austria
- Study on Long-term Safety and Efficacy of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Study on the Effectiveness and Safety of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Belgium
- Czechia
- France
- Germany
- Study on Long-Term Effects of Iptacopan in Patients with C3 Glomerulopathy or Idiopathic Immune-Complex Membranoproliferative Glomerulonephritis
- Study on Long-term Safety and Efficacy of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Study on the Effectiveness and Safety of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Greece
- Italy
- Study on Long-Term Effects of Iptacopan in Patients with C3 Glomerulopathy or Idiopathic Immune-Complex Membranoproliferative Glomerulonephritis
- Study on Long-term Safety and Efficacy of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Study on the Effectiveness and Safety of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Netherlands
- Study on Long-Term Effects of Iptacopan in Patients with C3 Glomerulopathy or Idiopathic Immune-Complex Membranoproliferative Glomerulonephritis
- Study on Long-term Safety and Efficacy of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Study on the Effectiveness and Safety of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Spain
- Sweden
Study Comparing Aliskiren and Enalapril for Patients with C3 Glomerulopathy
This study, taking place in Sweden, is comparing two different medications for treating C3 glomerulopathy: aliskiren and enalapril. The trial uses a cross-over design, meaning participants will receive both medications at different times during the study.
Who can participate: Children aged 6 years and older, as well as adults, may join this study. Participants must have a confirmed diagnosis of Dense Deposit Disease or C3 glomerulonephritis, verified by a kidney biopsy performed within the past two years. Patients may already be on treatment with aliskiren, ACE inhibitors, ARBs, or immune suppressive medications, or they may be starting treatment for the first time. Women of childbearing age must agree to use effective birth control and undergo regular pregnancy testing throughout the study.
Who cannot participate: The study excludes patients who do not have a confirmed diagnosis of C3 glomerulopathy, those outside the specified age range, vulnerable populations requiring special protection, and individuals unable to follow study procedures. Pregnant or breastfeeding women, those with allergies to the study medications, and people currently enrolled in other clinical trials are also excluded.
Study focus: The main goal is to evaluate whether aliskiren is more effective than enalapril at reducing kidney inflammation and improving kidney function. Researchers will monitor changes in serum C3 levels and complement protein deposits in the kidneys, which are markers of disease activity. The study will track kidney function over time to determine which medication provides better protection against disease progression.
Investigational drugs: Aliskiren is a direct renin inhibitor that works by blocking a protein involved in blood pressure regulation and may help reduce complement activation in the kidneys. It is taken orally as a 300 mg tablet once daily. Enalapril is an ACE inhibitor commonly used to treat high blood pressure and protect the kidneys. It is taken orally as a 5 mg tablet once daily.
Study on Long-Term Effects of Iptacopan in Patients with C3 Glomerulopathy or Idiopathic Immune-Complex Membranoproliferative Glomerulonephritis
This long-term study is evaluating iptacopan for patients with C3 glomerulopathy or a related condition called idiopathic immune-complex membranoproliferative glomerulonephritis. The trial is being conducted across seven European countries: Italy, France, Netherlands, Spain, Germany, Czechia, and Greece.
Who can participate: Patients of any age or gender who have completed the treatment period of specific previous studies (CLNP023X2202, CLNP023B12301, or CLNP023B12302) are eligible. This includes vulnerable populations who may need special care.
Who cannot participate: The study excludes individuals who were not diagnosed with C3 glomerulopathy or immune-complex membranoproliferative glomerulonephritis, those who were not part of the specified previous clinical trials, and people outside the designated study groups.
Study focus: The primary aim is to assess the long-term safety, tolerability, and effectiveness of iptacopan over an extended period, with a maximum treatment duration of 66 weeks. Researchers will monitor kidney function and measure protein levels in urine to determine if iptacopan can maintain or improve kidney health over time. For participants with transplanted kidneys, the study will evaluate changes in C3 deposit scores through kidney biopsies.
Investigational drug: Iptacopan is taken orally as hard gelatin capsules. It works by inhibiting a specific protein in the complement system, which is part of the immune system, to reduce inflammation and damage in the kidneys. The medication is classified as a complement inhibitor.
Study on Long-term Safety and Efficacy of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
This study is examining the long-term safety and effectiveness of pegcetacoplan for patients with C3 glomerulopathy or immune-complex membranoproliferative glomerulonephritis. The trial is taking place in eight European countries: Italy, Netherlands, Czechia, Spain, France, Austria, Belgium, and Germany.
Who can participate: Eligible participants must have completed a previous study called APL2-C3G-310 through the Week 52 visit and experienced clinical benefit from pegcetacoplan according to their doctor’s assessment. Participants must remain on a stable treatment plan and have received required vaccinations against certain bacterial infections. Both men and women must agree to use effective birth control during the study and for 90 days after the last dose. Participants or their caregivers must be willing and able to self-administer the medication through injections.
Who cannot participate: The study excludes patients with severe allergic reactions to the study medication, active infections requiring treatment, those currently in another clinical trial, people with recent drug or alcohol abuse, pregnant or breastfeeding women, kidney transplant recipients, and those who recently received certain immune system medications.
Study focus: The trial aims to evaluate the long-term safety and effectiveness of pegcetacoplan in protecting kidney function and reducing protein in the urine. Regular monitoring will include blood tests, urine tests, and assessments of kidney health. The study will track changes over several years to provide comprehensive data on long-term treatment outcomes.
Investigational drug: Pegcetacoplan is administered as an injection under the skin using a special device. It works by targeting the complement system, specifically the C3 protein, which plays a role in inflammation and kidney damage. By controlling this part of the immune system, pegcetacoplan aims to reduce kidney damage and improve function over time.
Study on the Effectiveness and Safety of Pegcetacoplan for Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
This double-blind, placebo-controlled study is testing pegcetacoplan for patients with C3 glomerulopathy or immune-complex membranoproliferative glomerulonephritis. The trial is being conducted in Austria, Netherlands, Italy, and Germany.
Who can participate: Adults aged 18 and older, as well as adolescents aged 12-17 who weigh at least 30 kg, can join the study. Participants must have a confirmed diagnosis of primary C3 glomerulopathy or immune-complex membranoproliferative glomerulonephritis, with or without a previous kidney transplant, and show signs of active disease through kidney biopsy or blood and urine tests. Eligible participants must have specific levels of protein in their urine and maintain stable kidney function. They must have been on a stable treatment plan for at least 12 weeks before joining and have received required vaccinations. Women of childbearing age must use effective birth control, and men must agree to use contraception and not donate semen during the study and for 90 days afterward.
Who cannot participate: The study excludes patients with other kidney diseases unrelated to C3 glomerulopathy or immune-complex membranoproliferative glomerulonephritis, those outside the specified age range, people unable to follow study procedures, pregnant or breastfeeding women, individuals with severe allergic reactions to the medication, current participants in other trials, people with serious medical conditions that could interfere with results, kidney transplant recipients, and those with a history of drug or alcohol abuse or active infections.
Study focus: The primary goal is to determine if pegcetacoplan can effectively reduce proteinuria, a key indicator of kidney damage, over a 26-week treatment period. The study uses a double-blind design, meaning neither participants nor researchers know who receives the actual medication or placebo, to ensure unbiased results. Regular assessments will monitor changes in kidney function and protein levels in urine.
Investigational drug: Pegcetacoplan is given as an injection under the skin twice a week at a dose of 1,080 mg. It works by inhibiting the complement pathway, specifically targeting the C3 protein involved in inflammation and tissue damage in the kidneys.
Summary
The four clinical trials for C3 glomerulopathy reflect a focused research effort on immune system-targeted therapies. Most trials are concentrated in Western European countries, with Germany, Italy, Netherlands, and Austria hosting multiple studies. Three of the four trials are testing pegcetacoplan, a complement inhibitor administered through subcutaneous injections, suggesting significant research interest in this particular medication. One trial is exploring iptacopan, an oral complement inhibitor, and another is comparing traditional medications (aliskiren versus enalapril) in a unique Swedish study.
The trials vary in their approach, with some focusing on long-term safety and effectiveness in patients who have already participated in earlier studies, while others are recruiting new participants to evaluate initial effectiveness. Most studies require participants to have received specific vaccinations and maintain stable treatment regimens. The trials typically monitor kidney function through regular assessments of protein levels in urine and blood tests, with some including kidney biopsies to directly evaluate disease activity.
These studies offer hope for patients with C3 glomerulopathy, a rare and serious kidney disease with limited treatment options. The concentration on complement inhibitors reflects current understanding that the disease involves dysregulation of the immune system’s complement pathway, and that targeting this pathway may help protect kidney function and slow disease progression.
