Pegcetacoplan for Glomerulonephritis

(VALIANT Trial)

Recruiting at 143 trial locations

Overseen By Apellis Clinical Trial Information Line

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Apellis Pharmaceuticals, Inc.

Must be taking: ACE inhibitors, ARBs, SGLT2 inhibitors

Must not be taking: Rituximab, Belimumab, Anticomplement therapy

Disqualifiers: HIV, Hepatitis B/C, Malignancy, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

124 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness and safety of pegcetacoplan, an experimental treatment, for individuals with kidney conditions known as C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN). The goal is to determine if it can reduce protein levels in urine, a common issue for these conditions. Participants will receive either pegcetacoplan or a placebo (a substance with no active medicine) via an injection under the skin twice a week. Suitable candidates for the trial have been diagnosed with C3G or IC-MPGN and experience high protein levels in their urine. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial requires that your current medications for C3G or IC-MPGN, such as certain blood pressure medications and immunosuppressants, be stable for at least 12 weeks before starting the study. If you're on prednisone or similar steroids, the dose must be stable and not exceed 20 mg/day for the same period.

Is there any evidence suggesting that pegcetacoplan is likely to be safe for humans?

Studies have shown that pegcetacoplan is generally safe for patients with certain kidney diseases. Previous research found that most side effects were mild to moderate. Pegcetacoplan has been tested in several kidney-related conditions, and patients typically responded well, indicating it usually doesn't cause serious problems.

Notably, the FDA has already approved pegcetacoplan for other uses, which adds confidence in its safety. Overall, current evidence suggests the treatment appears to be safe. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for glomerulonephritis focus on managing symptoms and slowing kidney damage with immunosuppressants like corticosteroids. However, pegcetacoplan is unique because it targets the complement system, a part of the immune system that can contribute to kidney damage in this condition. By inhibiting the complement pathway, pegcetacoplan offers a novel mechanism of action that specifically addresses the root cause rather than just managing symptoms. Researchers are excited about this approach because it has the potential to provide more effective protection for the kidneys, potentially slowing or even halting disease progression more effectively than current options.

What evidence suggests that pegcetacoplan might be an effective treatment for glomerulonephritis?

In this trial, participants will receive either pegcetacoplan or a placebo. Studies have shown that pegcetacoplan significantly reduces protein in the urine, an important goal in treating kidney inflammation. In patients with C3 glomerulopathy (C3G) and immune-complex membranoproliferative glomerulonephritis (IC-MPGN), pegcetacoplan reduced urine protein levels by 68% after 26 weeks compared to a placebo, and this reduction was maintained even after one year. The treatment has shown promise in improving kidney function by increasing levels of a protein called C3 in the blood and lowering other markers of disease activity. Overall, pegcetacoplan has consistently helped manage these conditions. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

My kidney biopsy shows less than 50% damage.

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

My treatment for C3G/IC-MPGN, including medications for protein in urine and possibly prednisone, has been stable.

Exclusion Criteria

You are allergic to pegcetacoplan or any of the ingredients in the medication.

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

I have cancer, except for certain types.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive twice-weekly subcutaneous doses of pegcetacoplan or placebo

24 weeks

Twice weekly visits for subcutaneous infusion

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Pegcetacoplan

Trial Overview 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.

How Is the Trial Designed?

Group I: Group 1: Pegcetacoplan administration Experimental Treatment 1 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 administration Placebo Group 1 Intervention

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

Pegcetacoplan is already approved in United States, European Union for the following indications:

Approved in United States as Empaveli for:

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Approved in European Union as Aspaveli for:

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Who Is Running the Clinical Trial?

Apellis Pharmaceuticals, Inc.

Lead Sponsor

Trials

26

Recruited

4,300+

Citations

1. investors.apellis.com investors.apellis.com/news-releases/news-release-details/new-one-year-data-reinforcing-robust-and-sustained-efficacy

Release Details Significant proteinuria reduction maintained at one year, regardless of immunosuppressant use or baseline proteinuria levels ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10658235/

Clinical Safety and Efficacy of Pegcetacoplan in a Phase 2 ... Pegcetacoplan may provide therapeutic benefit for C3G and has a favorable safety profile across the 4 glomerular diseases studied.

3. clinicaltrials.gov clinicaltrials.gov/study/nct05067127

nct05067127 | Phase III Study Assessing the Efficacy and ... This is a Phase 3 study to assess the efficacy and safety of twice-weekly subcutaneous (SC) doses of pegcetacoplan compared to placebo in patients with C3 ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2468024924019612

Efficacy and Safety of Pegcetacoplan in Kidney Transplant ... Twelve-month data for these patients reported eGFR outcomes with sustained serum C3 increases and plasma sC5b-9 decreases but did not discuss C3 staining or ...

5. checkrare.com checkrare.com/new-data-on-pegcetacoplan-in-patients-with-c3g-and-ic-mpgn/

New Data on Pegcetacoplan in Patients With C3G and IC- ... Pegcetacoplan demonstrated a statistically significant proteinuria reduction of 68% at week 26 versus placebo. This reduction was sustained at ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05067127

NCT05067127 | Phase III Study Assessing the Efficacy and ... This is a Phase 3 study to assess the efficacy and safety of twice-weekly subcutaneous (SC) doses of pegcetacoplan compared to placebo in patients with C3 ...

7. journals.lww.com journals.lww.com/jasn/fulltext/2024/10001/long_term_safety_and_efficacy_of_pegcetacoplan_in.3545.aspx

Long-Term Safety and Efficacy of Pegcetacoplan ... A phase 3 extension study designed to evaluate long-term efficacy and safety of PEG in C3G and primary IC-MPGN.