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An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

This study is an extension of a previous trial, looking long-term at how safe and effective a medicine called pegcetacoplan is for two rare kidney conditions: C3 Glomerulopathy (C3G) and Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN). These conditions affect how your kidneys filter waste from your blood. The research aims to see if pegcetacoplan continues to help people who have already shown some improvement on the drug in the earlier study. Participants will continue taking the medicine, and doctors will carefully monitor their health over time. This helps researchers understand the medicine's full picture for people living with these kidney problems.

At a glance

What is this study about?

This study focuses on two specific and rare kidney diseases: C3 Glomerulopathy (C3G) and Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN). These conditions are often caused by problems with a part of your immune system called the complement system, which can damage the tiny filters in your kidneys. This damage can lead to serious kidney problems over time.

The main goal of this research is to learn more about a medication called pegcetacoplan. Researchers want to understand if this medicine continues to be safe and effective for people with C3G or IC-MPGN when used over a longer period. This study is an 'extension' of a previous trial, meaning that people who were already taking pegcetacoplan and seemed to be getting better will keep receiving the medicine under close medical supervision.

By following participants for a longer time, doctors hope to gather valuable information about how pegcetacoplan impacts the progression of these kidney diseases and improves patients' overall health. This kind of long-term study is really important for developing new and better treatments for challenging conditions like C3G and IC-MPGN.

Key takeaways

  • This study is for people with C3G or IC-MPGN who were in a previous trial.
  • It aims to see the long-term safety and effectiveness of pegcetacoplan medication.
  • Participants will continue taking pegcetacoplan and have regular health checks.
  • The study helps doctors understand the medicine over a longer period.
  • Close medical monitoring will be provided throughout the study.
  • You can discuss with your doctor if this study is right for you.

Who may be eligible?

To join this study, you must have already taken part in a previous study called APL2-C3G-310 and completed all your visits up to the 52-week mark. Your study doctor would also need to feel that you benefited from pegcetacoplan in that earlier study. You'll need to be on a stable treatment plan for your kidney condition, and you must have had certain vaccinations for common infections affecting people with these kinds of health issues.

If you're a woman who could become pregnant, you'll need to have a negative pregnancy test and agree to use birth control during and for a period after the study. Men will also need to use birth control and not donate sperm. Everyone joining, or their legal guardian if they are under the age of consent, needs to agree to participate. You must also be able to give yourself the medicine or have someone who can help you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  1. Were you in the previous study APL2-C3G-310 and completed your visits up to week 52?
  2. Does your study doctor believe pegcetacoplan helped you in the previous study?
  3. Are you able to maintain a stable treatment plan for your kidney condition?
  4. Are you up-to-date with required vaccinations for people with kidney conditions?
  5. Are you able to give yourself injections or do you have someone who can help you?
  6. If you could become pregnant, are you able to use birth control during the study?
Answer every question to see your result.

What does participation involve?

If you join this study, you will continue to receive the study medication, pegcetacoplan, for an extended period. The medication is given by injection. You'll have regular hospital or clinic visits where the study team will monitor your health closely. This will involve physical examinations, blood tests, urine tests, and answering questions about how you are feeling. The study team will be looking for any changes in your kidney function and any side effects from the medication. You'll need to make sure you keep up-to-date with any recommended vaccinations. The total duration of your participation will depend on the study plan, but the main aim is to gather long-term information on the medication.

Potential risks and benefits

Participating in this study might offer potential benefits, such as continuing to receive an experimental treatment that your doctor believes has helped you. You'll also receive close medical attention and monitoring from a specialist team. However, there are potential risks, including side effects from the medication that are not yet fully known over a long period. Taking part also requires time and commitment for clinic visits and following study procedures. It's important to remember that you can decide to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (47)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Academic Medical Research Institute (01034)
    Verified postcode
    Los Angeles, United States
  • Children's Hospital Colorado (01037)
    Verified postcode
    Aurora, United States
  • University of Florida, Department of Pediatric Nephrology (01010)
    Unverified
    Gainsville, United States
  • Emory Investigational Drug Service (01021)
    Verified postcode
    Atlanta, United States
  • University of Iowa Hospital and Clinics (01030)
    Verified postcode
    Iowa City, United States
  • University of Michigan Hospital (01008)
    Verified postcode
    Ann Arbor, United States
  • Hackensack Meridian Health (01036)
    Verified postcode
    Hackensack, United States
  • Cohen Children Hospital, Pediatric Pharmacy (01022)
    Verified postcode
    Hyde Park, United States
  • Columbia Nephrology (01039)
    Verified postcode
    New York, United States
  • Oregon Health & Science Univ., Dept. Pediatrics (01038)
    Verified postcode
    Portland, United States
  • Northeast Clinical Research Center LLC (01009)
    Verified postcode
    Bethlehem, United States
  • Texas Children's Hospital (01027)
    Verified postcode
    Houston, United States

Common questions

What is C3 Glomerulopathy (C3G) or IC-MPGN?

These are rare kidney diseases where parts of your immune system cause damage to the filtering units in your kidneys, which can lead to kidney problems.

What is pegcetacoplan?

It's an investigational medicine being studied for these kidney conditions. It works by targeting a specific part of your immune system.

Why is this an 'extension' study?

It means it's a continuation of a previous study. People who were already taking pegcetacoplan and seemed to benefit from it are invited to continue.

Will I get paid to participate?

Generally, study participation isn't paid, but some studies might cover travel costs. You should ask your doctor about this.

Can I stop participating at any time?

Yes, you have the right to withdraw from the study at any point without it affecting your regular medical care.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Discussion

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