What side effects are associated with this type of intervention?

Common treatments for Glomerulonephritis, such as plasmapheresis, cyclophosphamide, prednisone, and avacopan, have various side effects. Plasmapheresis can cause hypotension and allergic reactions. Cyclophosphamide is associated with bone marrow suppression, increased infection risk, and potential bladder toxicity. Prednisone can lead to weight gain, hypertension, diabetes, and Cushingoid features. Avacopan, used to reduce glucocorticoid use, may cause liver issues and should be avoided in patients with chronic liver disease. TRF-budesonide, used in IgA nephropathy, can cause glucocorticoid-related adverse events like Cushingoid features. Calcineurin inhibitors, such as cyclosporine and tacrolimus, may lead to nephrotoxicity and hypertension.

What prior FDA approvals exist?

FDA-approved treatments for Glomerulonephritis have included various immunosuppressive therapies such as corticosteroids (e.g., prednisone), cyclophosphamide, and rituximab. These treatments aim to reduce inflammation and immune response in the kidneys. Pegcetacoplan, a C3 inhibitor, is being studied for its potential to reduce proteinuria in conditions like C3 glomerulopathy and immune-complex membranoproliferative glomerulonephritis, which are forms of Glomerulonephritis. While Pegcetacoplan itself is still under investigation, its mechanism of action is similar to other complement inhibitors that target the immune system to prevent kidney damage.

What other types of research exist?

Promising novel alternatives to Pegcetacoplan for Glomerulonephritis patients include Rituximab and Mycophenolate Mofetil (MMF). These agents are considered for patients who cannot tolerate cyclophosphamide, although they are not first-line therapies due to insufficient evidence. Additionally, ongoing research and clinical trials may reveal new targeted therapies or biologics that could offer effective treatment options in the near future.

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Complement Inhibitor

Pegcetacoplan for Glomerulonephritis (VALIANT Trial)

Phase 3

Waitlist Available

Research Sponsored by Apellis Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

No more than 50% global glomerulosclerosis or interstitial fibrosis on the baseline biopsy for adult participants or adolescent participants providing a baseline biopsy.

A diagnosis of primary C3G or IC-MPGN (with or without previous renal transplant).

Must not have

Female participants who are pregnant or currently breastfeeding and unwilling to discontinue for the duration of the study and for at least 90 days after the final dose of study drug.

Previous exposure to pegcetacoplan.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing an injectable medication called pegcetacoplan for patients with certain kidney diseases that cause high protein levels in their urine. These diseases are C3 glomerulopathy (C3G) and immune-complex membranoproliferative glomerulonephritis (IC-MPGN). Pegcetacoplan works by reducing the immune system's attack on the kidneys, which helps lower the protein levels in the urine.

Who is the study for?

Adults and certain adolescents with primary C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis, who have significant protein in their urine, stable kidney disease treatment regimens, and specific signs of active renal disease. Excludes those previously treated with Pegcetacoplan, secondary conditions causing the disease, severe infections or immune deficiencies, weight over 100 kg, known allergies to study drug components.

What is being tested?

The trial is testing the effectiveness of Pegcetacoplan versus a placebo in reducing proteinuria (excess protein in urine) for patients with C3G or IC-MPGN. Participants will receive twice-weekly subcutaneous doses and be monitored for changes in their condition.

What are the potential side effects?

Potential side effects may include reactions at the injection site, increased risk of infections due to immune system suppression by Pegcetacoplan, allergic responses to medication ingredients, and possibly other unknown risks that are common when testing new treatments.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My kidney biopsy shows less than 50% damage.

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I have been diagnosed with primary C3G or IC-MPGN.

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I have active kidney disease confirmed by tests or a biopsy.

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I have active kidney disease confirmed by specific tests.

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My kidney function is sufficient according to specific medical calculations.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not pregnant or breastfeeding, or I am willing to stop breastfeeding for the study and 90 days after.

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I have been treated with pegcetacoplan before.

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I have or had HIV, HBV, or HCV.

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My weight is over 100 kg.

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I have or might have hereditary fructose intolerance.

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I have had meningococcal disease before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2020 Phase 3 trial • 80 Patients • NCT03500549

41%

Injection site erythema

15%

Injection site pruritus

13%

Headache

13%

Injection site swelling

13%

Diarrhoea

10%

Injection site reaction

Nausea

Pyrexia

Injection site pain

Fatigue

Injection site induration

Dyspnoea

Nasopharyngitis

Contusion

Dizziness

Chromaturia

Vaccination site pain

Pain in extremity

Back pain

Myalgia

Vaccination complication

Anxiety

Thrombocytopenia

Cough

Abdominal pain

Abdominal discomfort

Arthralgia

Injection site bruising

Sepsis

Oral herpes

Sinusitis

Haemolysis

Vomiting

Anaemia

Asthenia

Palpitations

Abdominal distension

Constipation

Urinary tract infection

100%

80%

60%

40%

20%

Study treatment Arm

Run-in Period: Pegcetacoplan + Eculizumab

Open-label Period: Pegcetacoplan

RCP: Eculizumab

RCP: Pegcetacoplan

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Group 1: Pegcetacoplan administrationExperimental Treatment1 Intervention

Subcutaneous infusion of 20mL (1080 mg), twice weekly (for adults or adolescents \>50kg), and the three other weight-based doses either of 10mL (540mg), 12mL (648mg), or 15mL (810mg)

Group II: Group 2: Placebo administrationPlacebo Group1 Intervention

Subcutaneous infusion of either 10mL, 12mL, 15mL, or 20mL, twice weekly

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pegcetacoplan

2015

Completed Phase 3

~400

0 / 11Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Glomerulonephritis, such as immunosuppressive therapies and complement inhibitors, aim to reduce inflammation and immune-mediated damage to the kidneys. Immunosuppressive drugs like cyclophosphamide and glucocorticoids suppress the immune system to prevent further kidney damage. Complement inhibitors, such as Pegcetacoplan, specifically target the complement system, which is part of the immune response that contributes to kidney inflammation and damage in diseases like C3G and IC-MPGN. By inhibiting the complement pathway, these treatments help reduce proteinuria and preserve kidney function, which is crucial for improving outcomes and quality of life for Glomerulonephritis patients.

Meta-Analysis of the Effectiveness and Safety of Glucocorticoid for the Treatment of IgA Kidney Disease.Efficacy and Safety of Agents in IgA Nephropathy: An Update Network Meta-Analysis.Antithrombotic drug therapy for IgA nephropathy: a meta analysis of randomized controlled trials.