Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not yet recruitingRare disease

Efficacy and Safety of Pegcetacoplan in Adults and Adolescents with Focal Segmental Glomerulosclerosis: Phase 2/3 Open‑Label and Randomized Placebo‑Controlled Study

Verified siteRegistered drug
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

Focal segmental glomerulosclerosis is a rare kidney condition in which small parts of the filtering units become scarred, leading to leakage of protein into the urine. This leakage, called proteinuria, can be measured by the amount of protein relative to creatinine in a urine sample. The study evaluates the investigational drug pegcetacoplan, which is given by a subcutaneous injection, meaning it is administered under the skin.

The purpose of the study is to determine whether twice‑weekly dosing of the drug can lower protein loss in the urine compared with placebo. Participants will first receive the active medication in an open‑label phase, followed by a randomized, double‑blind phase where some receive the drug and others receive the placebo, with regular monitoring of kidney function and safety.

Throughout the trial, participants will have periodic clinic visits where urine samples are collected to calculate the urine protein to creatinine ratio and the urine albumin to creatinine ratio, both of which indicate the level of protein leakage. Blood tests will also be performed to assess the estimated Glomerular Filtration Rate, a standard measure of how well the kidneys are filtering waste. The study continues over several months to observe changes in these measurements and to ensure the treatment is safe.

The research process

The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Baseline assessment

    After joining the study, you will have a baseline visit where your health status is recorded. this includes urine tests, blood tests, and measurements of kidney function.

    The study staff will explain the purpose of the trial and answer any questions you may have.

  2. Step 2

    Training on subcutaneous injection

    You will receive instruction on how to give a subcutaneous injection, which means the medication is injected just under the skin.

    The training includes how to prepare the dose, how to clean the injection site, and how to dispose of used supplies safely.

  3. Step 3

    Phase 2 open‑label treatment start

    You will begin receiving pegcetacoplan at a dose of 1080 mg per injection.

    The injection is given twice weekly under the skin. the exact days are chosen by the study team to fit your schedule.

    This part of the trial is open‑label, meaning you know you are receiving the active medication.

  4. Step 4

    Regular monitoring during phase 2

    Throughout the phase 2 period you will attend scheduled visits where urine protein to creatinine ratio (uPCR) and urine albumin to creatinine ratio (uACR) are measured.

    Blood tests and assessments of estimated glomerular filtration rate (eGFR) are also performed to monitor kidney function.

    Any side effects are recorded and discussed with the study staff.

  5. Step 5

    Transition to phase 3 double‑blind period

    After completing the open‑label phase, you will enter the phase 3 portion, which is randomized, placebo‑controlled, and double‑blind.

    You will be randomly assigned to continue receiving either pegcetacoplan 1080 mg or a placebo that looks identical.

    Neither you nor the study staff will know which treatment you are receiving during this period.

  6. Step 6

    Phase 3 treatment continuation

    The same dosing schedule of a subcutaneous injection twice weekly is continued for the duration of phase 3.

    The study medication (active or placebo) is administered in the same manner as in phase 2.

  7. Step 7

    Ongoing assessments in phase 3

    Regular visits continue to measure changes in proteinuria using the urine protein to creatinine ratio (uPCR) and to evaluate other laboratory values.

    The proportion of participants achieving complete remission and the slope of estimated glomerular filtration rate (eGFR) are also tracked.

    Safety information, including any adverse events, is collected at each visit.

  8. Step 8

    Final study visit and end of treatment

    At the conclusion of the trial you will have a final visit where all study measurements are performed one last time.

    The study medication is stopped, and you will receive information about any follow‑up care that may be recommended.

Who can join the trial?

6 criteria

  • Be 18 years of age or older (some sites may allow participants who are 12‑17 years old if approved).
  • Weigh at least 30 kg (about 66 lb) and no more than 100 kg (about 220 lb) at the screening visit.
  • Have a diagnosis of FSGS (primary, genetic, or unknown cause) confirmed by a kidney biopsy (a small piece of kidney tissue taken for examination) or by a known podocyte genetic mutation (a specific gene change in kidney cells).
  • Show at least 1.5 g of protein in a 24‑hour urine collection each day (called proteinuria) and have a urine protein‑to‑creatinine ratio (uPCR) of 1.5 g/g or higher in two separate screening samples.
  • Have an estimated glomerular filtration rate (eGFR) of 25 mL/min/1.73 m² or higher, which is a test that estimates how well the kidneys are filtering blood.
  • Be on a stable treatment plan for FSGS for at least 12 weeks before the first study dose, with no expected changes to the medicines or doses during that time (stable regimen).

Who cannot join the trial?

9 criteria

  • Previously taken pegcetacoplan before the study.
  • Kidney disease has been getting better in the 8 weeks before screening.
  • Have FSGS caused by another condition such as an infection, diabetes, certain medicines, obesity, being born early, sickle‑cell disease, urinary‑tract problems, or kidney birth defects.
  • Have type 1 diabetes or type 2 diabetes that is not well controlled (blood‑sugar test called HbA1C 8 % or higher). HbA1C measures average blood sugar over the past three months.
  • Have had a kidney transplant in the past.
  • Current or past infection with HIV, hepatitis B, or hepatitis C, or test positive for any of these viruses during screening. HIV is a virus that attacks the immune system; hepatitis B and hepatitis C are viruses that affect the liver.
  • Allergy (hypersensitivity) to pegcetacoplan or any of its inactive ingredients (excipients). Hypersensitivity means an allergic reaction; excipients are substances added to a medication that are not the active drug.
  • Another serious kidney disease that doctors think would make it hard to understand the study results.
  • Used medicines such as rituximab, belimumab, or any other approved or experimental drug that affects the immune system’s complement pathway within five drug half‑lives before screening. Half‑life is the time it takes for half of a drug to leave the body; the complement pathway is part of the immune system.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not yet recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Pegcetacoplan is a medication given by injection under the skin. It works by blocking part of the immune system called complement C3, which can help reduce the amount of protein that leaks into the urine in people with focal segmental glomerulosclerosis (FSGS). In this trial, participants received pegcetacoplan twice a week to see if it can lower proteinuria and improve kidney health.

What is already known about the treatment

  • Pegcetacoplan

    Pegcetacoplan is given by subcutaneous injection as a 1080 mg solution for infusion, usually twice a week. It is an approved complement C3 inhibitor that is used for treating the rare kidney disorder focal segmental glomerulosclerosis (FSGS) and has also been studied for other complement‑mediated diseases. The drug works by attaching to the complement protein C3, stopping a chain reaction that can damage kidney cells. It is classified as a complement inhibitor and an orphan peptide drug.

  • Placebo

    The placebo is a sterile solution of acetate buffer with sorbitol, administered subcutaneously in the same way as the active drug. It contains no medicinal ingredient and is used only as a control to compare the effects of pegcetacoplan. Because it has no active components, it has no therapeutic indication. It is classified as an inert control substance.

Investigated diseases

Focal segmental glomerulosclerosis (FSGS) - It is a kidney disorder that damages the tiny filtering units called glomeruli. The damage occurs in patches, affecting only parts of some glomeruli. Over time, the scarred areas reduce the kidney’s ability to filter waste, leading to a gradual increase in protein loss in the urine. The condition often progresses slowly, with kidney function declining as more glomeruli become affected.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2025-524448-36-00Protocol codeAPL2-FSG-319Estimated enrolment238 patientsSponsorApellis Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.