Pegcetacoplan for Complement 3 Glomerulopathy

(VALE Trial)

Recruiting at 47 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: Pegcetacoplan

Disqualifiers: Pregnancy, Breastfeeding, Drug abuse, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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: Long-term

100 Participants Needed

Trial Summary

What is the purpose of this trial?

This study is designed as a long-term extension to Study APL2-C3G-310, and is being conducted to establish the long-term safety and efficacy of pegcetacoplan in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN).

Do I need to stop my current medications to join the trial?

The trial requires you to stay on a stable treatment plan for your condition, so you likely won't need to stop your current medications.

What data supports the effectiveness of the drug Pegcetacoplan for treating Complement 3 Glomerulopathy?

Pegcetacoplan has shown effectiveness in improving blood-related outcomes in patients with paroxysmal nocturnal haemoglobinuria, a condition involving the complement system, which is also implicated in Complement 3 Glomerulopathy. This suggests potential benefits for similar complement-related conditions. ^{1 2 3 4 5}

What makes the drug pegcetacoplan unique for treating C3 glomerulopathy?

Pegcetacoplan is unique because it targets the complement protein C3, which plays a crucial role in the complement system involved in C3 glomerulopathy. Unlike other treatments, it is a pegylated peptide that specifically inhibits C3, potentially offering a new approach for a condition with no approved treatments. ^{1 2 5 6 7}

Eligibility Criteria

This trial is for patients who have certain kidney diseases like C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis and were part of a previous study (APL2-C3G-310). They should have seen benefits from the drug pegcetacoplan, be vaccinated against specific infections, and agree to use contraception. It's not open to new patients; only those continuing from the prior study.

Inclusion Criteria

Completed participation in Study APL2-C3G-310 through the week 52 visit requirements

I can self-administer my medication or have someone to help me.

I benefited from pegcetacoplan treatment in a past trial, according to my doctor.

Exclusion Criteria

I am not pregnant, planning to become pregnant, or breastfeeding during the study and for 90 days after.

Inability or unwillingness to cooperate with the requirements of the protocol

Any condition that, in the opinion of the investigator, creates an undue risk for the participant by participating in the study or is likely to confound interpretation of the study results

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive pegcetacoplan administered subcutaneously twice weekly

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Continuation of treatment to evaluate long-term safety and efficacy

Long-term

Treatment Details

Interventions

• Pegcetacoplan

Trial Overview The trial is testing the long-term safety and effectiveness of a medication called pegcetacoplan in people with specific kidney conditions. This extension study follows up on earlier research where participants already received this treatment for about one year.

Participant Groups

Group I: Pegcetacoplan administered subcutaneously Experimental Treatment 1 Intervention

Pegcetacoplan administered subcutaneously twice weekly according to protocol defined dosing regimen

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+

Findings from Research

Iptacopan, a selective inhibitor of factor B in the alternative complement pathway, is being evaluated in a Phase III study involving 68 adults with complement 3 glomerulopathy (C3G) to assess its efficacy and safety in reducing proteinuria and improving kidney function.

The study aims to show that iptacopan can significantly reduce protein levels in urine (measured by urine protein:creatinine ratio) compared to a placebo, potentially offering a new treatment option for patients with this rare kidney disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Alternative Complement Pathway Inhibition With Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial. Bomback, AS., Kavanagh, D., Vivarelli, M., et al.[2022]

C3 glomerulopathy is a complex kidney disease affecting a diverse group of patients, with a mean diagnosis age of 40.4 years, and is characterized by significant proteinuria and hematuria, indicating kidney damage.

The disease shows a variable response to treatment, with most patients receiving corticosteroids and immunosuppressive drugs; however, 9.2% of patients without monoclonal immunoglobulin progressed to end-stage renal disease within a median follow-up of 22.3 months.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

C3 Glomerulopathy: Ten Years' Experience at Mayo Clinic. Ravindran, A., Fervenza, FC., Smith, RJH., et al.[2020]

Avacopan, a C5aR antagonist, was safely administered to a child with C3 glomerulonephritis (C3GN) in a Phase 2 clinical trial, demonstrating good tolerability and safety.

The patient was able to discontinue the use of mycophenolate mofetil (MMF) while maintaining remission from C3GN, suggesting that avacopan may be an effective treatment option for this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Successful treatment with avacopan (CCX168) in a pediatric patient with C3 glomerulonephritis. Zotta, F., Diomedi-Camassei, F., Gargiulo, A., et al.[2023]

References

1. England pubmed.ncbi.nlm.nih.gov

Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Alternative Complement Pathway Inhibition With Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial. [2022]

3. England pubmed.ncbi.nlm.nih.gov

C3 Glomerulopathy: Ten Years' Experience at Mayo Clinic. [2020]

4. United States pubmed.ncbi.nlm.nih.gov

C3 glomerulopathy. [2023]

5. Germany pubmed.ncbi.nlm.nih.gov

Successful treatment with avacopan (CCX168) in a pediatric patient with C3 glomerulonephritis. [2023]

6. Italy pubmed.ncbi.nlm.nih.gov

Novel targeted C3 inhibitor pegcetacoplan for paroxysmal nocturnal hemoglobinuria. [2023]

7. United States pubmed.ncbi.nlm.nih.gov

Injection Site Reactions with Long-Term Pegcetacoplan Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Brief Report. [2023]