Pegcetacoplan for Glomerulonephritis

(VALIANT Trial)

Recruiting at 122 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

Trial Summary

What is the purpose of this trial?

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.

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.

What data supports the effectiveness of the drug Pegcetacoplan for Glomerulonephritis?

Pegcetacoplan has been approved for treating paroxysmal nocturnal hemoglobinuria (PNH), a condition related to the immune system, by targeting a part of the immune system called complement component 3 (C3). It is also being studied for other immune-related diseases, which suggests it might help with glomerulonephritis, another condition involving the immune system. ^{1 2 3 4 5}

How is the drug Pegcetacoplan different from other treatments for glomerulonephritis?

Pegcetacoplan is unique because it targets the complement system, a part of the immune system, which is different from traditional treatments like glucocorticoids or immunosuppressive drugs that broadly suppress the immune response. This targeted approach may offer a novel way to manage glomerulonephritis by specifically addressing the underlying immune mechanisms involved in the disease. ^{6 7 8 9 10}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Pegcetacoplan: First Approval. [2021]

2. England pubmed.ncbi.nlm.nih.gov

Cost-utility analysis of certolizumab pegol versus alternative tumour necrosis factor inhibitors available for the treatment of moderate-to-severe active rheumatoid arthritis in Spain. [2023]

3. Italy pubmed.ncbi.nlm.nih.gov

Three-year clinical outcomes in patients with rheumatoid arthritis treated with certolizumab pegol: results from the observational ECLAIR study. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Long-term efficacy and safety of certolizumab pegol in Japanese rheumatoid arthritis patients with an inadequate response to methotrexate: 52-week results from an open-label extension of the J-RAPID study. [2022]

5. Italy pubmed.ncbi.nlm.nih.gov

Efficacy and safety of certolizumab pegol in combination with methotrexate in methotrexate-inadequate responder Chinese patients with active rheumatoid arthritis: 24-week results from a randomised, double-blind, placebo-controlled phase 3 study. [2019]

6. France pubmed.ncbi.nlm.nih.gov

[Treatment of proliferative glomerulonephritis of systemic lupus erythematosus. Recent development and current recommendations]. [2016]

7. Germany pubmed.ncbi.nlm.nih.gov

[Rapidly progressing glomerulonephritis. Spontaneous course and differential therapy with special reference to the infection-associated form]. [2019]

8. Russia (Federation) pubmed.ncbi.nlm.nih.gov

[Clinico-laboratory pattern and effectiveness of glucocorticoid therapy of patients with primary diffuse mesangioproliferative glomerulonephritis]. [2013]

9. Romania pubmed.ncbi.nlm.nih.gov

[The relationships of rapidly progressive glomerulonephritis (RPGN), crescentic glomerulonephritis and vasculitis: the clinical, histopathological and therapeutic considerations]. [2011]

10. Japan pubmed.ncbi.nlm.nih.gov

[A case of microscopic polyangitis with sepsis due to pyelonephritis]. [2011]