Pegcetacoplan for Recurrent Kidney Disease

(NOBLE Trial)

Not currently recruiting at 27 trial locations

Overseen By Apellis Clinical Trial Information Line

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Apellis Pharmaceuticals, Inc.

Disqualifiers: HIV, Hepatitis B, Hepatitis C, others

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

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the safety and effectiveness of pegcetacoplan for individuals who have experienced a recurrence of specific kidney diseases (C3G or IC-MPGN) after a kidney transplant. The study includes two groups: one will start treatment immediately, while the other will begin after 12 weeks. Individuals living with these kidney conditions who have had a transplant may be suitable for the trial, particularly if their condition has not improved recently. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

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

The trial protocol does not specify if you need to stop your current medications. However, it mentions that your treatment for recurrent C3G/IC-MPGN should be stable for at least 4 weeks before the screening and until randomization.

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

Research has shown that pegcetacoplan, the treatment under study, is generally safe. Studies have found that most patients tolerate it well, with side effects usually mild or moderate. Importantly, recent research has not identified any new safety concerns. Pegcetacoplan is being studied for its potential to help with kidney problems like C3G and IC-MPGN, and results so far suggest it could be a safe option for these conditions. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Pegcetacoplan is unique because it targets the complement system, specifically inhibiting C3, which plays a crucial role in the immune response that can lead to kidney damage. Unlike traditional treatments for recurrent kidney disease that may focus on managing symptoms or using immunosuppressants, pegcetacoplan directly interferes with the underlying inflammatory processes. This approach not only offers a novel mechanism of action but also has the potential to more effectively prevent disease progression and preserve kidney function. Researchers are excited about pegcetacoplan because it represents a promising new way to treat kidney disease at its source, rather than just alleviating symptoms.

What evidence suggests that pegcetacoplan might be an effective treatment for recurrent kidney disease?

Research has shown that pegcetacoplan may help treat certain kidney diseases. In this trial, participants in Group 1 will receive pegcetacoplan treatment throughout the study. Studies have found that pegcetacoplan can reduce protein in urine by 68%, an important indicator of kidney disease improvement. Additionally, pegcetacoplan helps maintain stable kidney function over time. Patients with similar conditions have experienced improvements in kidney health markers like eGFR, which measures how well the kidneys filter blood. Overall, pegcetacoplan appears to have a positive effect for those with recurring kidney disease after a transplant. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for adults who've had a kidney transplant and are now facing a recurrence of certain kidney diseases (C3G or IC-MPGN). Participants should have stable or worsening disease, adequate kidney function, no significant other renal diseases, and must be vaccinated against specific infections. They can't join if they have certain infections like HIV or hepatitis, previous pegcetacoplan treatment, low neutrophil count, weigh over 100 kg, or have certain cancer histories.

Inclusion Criteria

No more than 50% glomerulosclerosis or interstitial fibrosis on the screening renal allograft biopsy

Stable regimen for recurrent C3G/IC-MPGN for at least 4 weeks prior to the screening renal allograft biopsy and from the time of the screening renal allograft biopsy until randomization

Have received required vaccinations against N. meningitidis, S. pneumoniae, and H. influenzae (type B) or agree to receive vaccinations, if applicable vaccination records are not available. Vaccination is mandatory unless documented evidence exists that subjects are non-responders to vaccination.

Exclusion Criteria

You have a kidney disease caused by another condition, like an infection or cancer, that would make it difficult to understand the study results.

Evidence of rejection on the screening renal allograft biopsy that requires treatment

Diagnosis or history of HIV, hepatitis B, or hepatitis C infection or positive serology at screening indicative of infection with any of these viruses

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Randomized Controlled Phase

Participants are randomized to receive either pegcetacoplan treatment or no intervention for 12 weeks

12 weeks

Bi-weekly visits for treatment group

Open-label Treatment Phase

All participants receive pegcetacoplan treatment after the initial 12 weeks

40 weeks

Bi-weekly visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Pegcetacoplan

Trial Overview The study tests the safety and effectiveness of Pegcetacoplan in patients with post-transplant recurrence of C3G/IC-MPGN. It's an open-label Phase 2 trial where everyone knows what treatment is being given; some will receive Pegcetacoplan while others may not as part of the control group.

How Is the Trial Designed?

Group I: Group 2 Experimental Treatment 1 Intervention

No intervention given during the randomized controlled portion of the study (through week 12). After week 12, subjects will receive pegcetacoplan treatment.

Group II: Group 1 Experimental Treatment 1 Intervention

Pegcetacoplan treatment of 1080 mg (sub-cutaneous infusion) twice weekly will be given throughout the entire study.

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. 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.

2. investors.apellis.com investors.apellis.com/news-releases/news-release-details/fda-approves-apellis-empavelir-pegcetacoplan-first-c3g-and

FDA Approves Apellis' EMPAVELI® (pegcetacoplan) as the ... Proven efficacy across all three key markers of disease—68% reduction in proteinuria, stabilization of kidney function, and substantial ...

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. renalandurologynews.com renalandurologynews.com/news/pegcetacoplan-gets-priority-review-two-rare-kidney-diseases/

Pegcetacoplan Gets Priority Review for Two Rare Kidney ... Findings showed treatment with pegcetacoplan was associated with a statistically significant 68% reduction in proteinuria compared with placebo ...

5. 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 ...

6. investors.apellis.com investors.apellis.com/news-releases/news-release-details/positive-one-year-data-phase-2-study-pegcetacoplan-post

Positive One-Year Data from Phase 2 Study of ... Treatment with pegcetacoplan rapidly reduced disease activity in only 12 weeks, and the effects were sustained over the long term.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04572854

NCT04572854 | Study Assessing the Safety and Efficacy of ... This is a Phase 2, multicenter, open-label, randomized, controlled study designed to evaluate the safety and efficacy of pegcetacoplan in patients who have ...

8. 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 ...