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New drug shows promise for controlling rare kidney disorders

NCT ID NCT05809531

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests the long-term safety and effectiveness of pegcetacoplan in 100 people with two rare kidney diseases, C3 glomerulopathy and IC-MPGN. Participants previously completed a one-year trial and saw benefit from the drug. The goal is to see if pegcetacoplan can keep reducing protein in urine, a sign of kidney damage, over a longer period.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Pegcetacoplan
What this could lead to
If successful, this could provide a long-term treatment option to control kidney damage in people with C3G or IC-MPGN.
What could go wrong
This is an extension study, so it only includes people who already responded well. It may not show benefit for all patients, and long-term risks are still being evaluated.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 100 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2023

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Completed participation in Study APL2-C3G-310 through the week 52 visit requirements * Experienced clinical benefit from pegcetacoplan while participating in the previous trial, in the opinion of the investigator * Must remain on a stable regimen for C3G or IC-MPGN treatment according to the requirements of Study APL2-C3G-310 * Received vaccinations against S pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) according to the requirements of Study APL2-C3G-310 and agree to receive any additional vaccinations recommended according to ACIP recommendations for adults or children with complement deficiencies and/or immunocompromising conditions or other similar local applicable guidelines * Female participants of childbearing potential, defined as any woman who has experienced menarche and who is not permanently sterile or postmenopausal, must have a negative urine pregnancy test at visit 1 and must agree to use protocol-defined methods of contraception for the duration of the study through at least 90 days after receiving the last dose of pegcetacoplan * Male participants must agree to use protocol-defined methods of contraception and agree to refrain from donating semen for the duration of the study through at least 90 days after receiving the last dose of pegcetacoplan * Participants above the legal age of consent, in accordance with local regulations, must be willing and able to provide informed consent. The legally authorized representative of participants under the legal age of consent must be willing and able to provide informed consent; where appropriate, participants under the legal age of consent must also give their assent to participation in the study * Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration Exclusion Criteria: * Female participants who are or are planning to become pregnant or who are currently breastfeeding and are unwilling to discontinue for the duration of the study and for at least 90 days after the final dose of study drug * 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 * Evidence of ongoing drug or alcohol abuse or dependence, in the opinion of the investigator

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • AMC Apotheek Goederenontvangst (31002)

    Amsterdam, 1105 AZ, Netherlands

  • Academic Medical Research Institute (01034)

    Los Angeles, California, 90022, United States

  • CHU de Bordeaux - Hopital Pellegrin (33010)

    Bordeaux, 33076, France

  • CHUV Lausanne (41002)

    Lausanne, CH-1011, Switzerland

  • Catholic University of Leuven (32001)

    Leuven, B-3000, Belgium

  • Children's Hospital Colorado (01037)

    Aurora, Colorado, 80045, United States

  • Clinica Privada Velez Sarsfield (54007)

    Córdoba, X5000, Argentina

  • Cohen Children Hospital, Pediatric Pharmacy (01022)

    Hyde Park, New York, 11040, United States

  • Columbia Nephrology (01039)

    New York, New York, 10032, United States

  • Emory Investigational Drug Service (01021)

    Atlanta, Georgia, 30322, United States

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (39004)

    Milan, 20122, Italy

  • HC UNESP Botucatu (55010)

    Botucatu, 18618-687, Brazil

  • Hackensack Meridian Health (01036)

    Hackensack, New Jersey, 07601, United States

  • Hosp. Universit. Materno-Infantil Vall d' Hebron (34006)

    Barcelona, 8035, Spain

  • Hospital De Base (55014)

    São José do Rio Preto, 1500900-000, Brazil

  • Hospital Privado-Universitario de Cordoba (54004)

    Córdoba, CPA X5016KEH, Argentina

  • Hospital Universitario 12 de Octubre (34008)

    Madrid, 28041, Spain

  • Hospital Universitario Dr Peset (34009)

    Valencia, 46017, Spain

  • Hospital de Clinicas de Porto Alegre (55018)

    Porto Alegre, 90035-903, Brazil

  • Hospital do Rim - Fundacao Oswaldo Ramos (55005)

    São Paulo, 04038-002, Brazil

  • Imperial College Hammersmith Hospital (44013)

    London, W12 0HS, United Kingdom

  • Institut de transplantation urologie-nephrologie (33011)

    Nantes, 44093, France

  • Institute for Clinical and Experimental Medicine (42001)

    Prague, 14021, Czechia

  • Irmandade da Santa Casa Misericordia Porto Alegre (55016)

    Porto Alegre, Rio Grande do Sul, 90020-090, Brazil

  • Istituto di Ricerche Farmacologiche Mario Negri IRCCS (39003)

    Ranica, 24020, Italy

  • Kidney Trials Unit, Princess Alexandra Hospital (61007)

    Woolloongab, Queensland, 4102, Australia

  • Nagasaki University Hospital (81005)

    Nagasaki, Nagasaki, 852-8501, Japan

  • Northeast Clinical Research Center LLC (01009)

    Bethlehem, Pennsylvania, 18017, United States

  • Nottingham University Hospitals NHS Trust (44012)

    Nottingham, NG7 2UH, United Kingdom

  • Oregon Health & Science Univ., Dept. Pediatrics (01038)

    Portland, Oregon, 97239, United States

  • Ospesale Pediatrico bambino Gesu (39005)

    Rome, 165, Italy

  • Radboud University Medical Center (31003)

    Nijmegen, 6500 HB, Netherlands

  • Real Hospital Portugues de Beneficencia (55015)

    Recife, 52010-095, Brazil

  • Royal Manchester Children's Hospital (44011)

    Manchester, M13 9WL, United Kingdom

  • Ruschel Medicina E Pesquisa Clinica (55012)

    Rio de Janeiro, 22270-060, Brazil

  • Santa Casa de Misericordia de Belo Horizonte (55017)

    Belo Horizonte, Minas Gerais, 30150-221, Brazil

  • Schneider Childrens Medical Center (97204)

    Petah Tikva, 4920235, Israel

  • Seirei Hamamatsu General Hospital (81004)

    Hamamatsu, Shizuoka, 430-8558, Japan

  • Seoul National University Hospital (82003 & 82005)

    Soeul, 3080, South Korea

  • St. Vincent's Hospital Melbourne (61003)

    Fitzroy, 3065, Australia

  • Texas Children's Hospital (01027)

    Houston, Texas, 77030, United States

  • Univ Sao Paulo, Av. Dr Eneas Carvalho de Aguiar, 155 (55007)

    São Paulo, 05403-900, Brazil

  • University Medical Center Groningen (31004)

    Groningen de Brug, 9713 GZ, Netherlands

  • University of Florida, Department of Pediatric Nephrology (01010)

    Gainsville, Florida, 32610, United States

  • University of Iowa Hospital and Clinics (01030)

    Iowa City, Iowa, 52242, United States

  • University of Michigan Hospital (01008)

    Ann Arbor, Michigan, 48109, United States

  • Yonsei University College of Medicine, Sinchon Severance Hospital (82002)

    Seoul, 3722, South Korea

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