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 (VALE)

March 31, 2026 updated by: Apellis Pharmaceuticals, Inc.

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

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
100

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Córdoba, Argentina, CPA X5016KEH
        • Hospital Privado-Universitario de Cordoba (54004)
      • Córdoba, Argentina, X5000
        • Clinica Privada Velez Sarsfield (54007)
  • Australia
      • Fitzroy, Australia, 3065
        • St. Vincent's Hospital Melbourne (61003)
    • Queensland
      • Woolloongab, Queensland, Australia, 4102
        • Kidney Trials Unit, Princess Alexandra Hospital (61007)
  • Belgium
      • Leuven, Belgium, B-3000
        • Catholic University of Leuven (32001)
  • Brazil
      • Botucatu, Brazil, 18618-687
        • HC UNESP Botucatu (55010)
      • Porto Alegre, Brazil, 90035-903
        • Hospital de Clinicas de Porto Alegre (55018)
      • Recife, Brazil, 52010-095
        • Real Hospital Portugues de Beneficencia (55015)
      • Rio de Janeiro, Brazil, 22270-060
        • Ruschel Medicina E Pesquisa Clinica (55012)
      • São José do Rio Preto, Brazil, 1500900-000
        • Hospital De Base (55014)
      • São Paulo, Brazil, 04038-002
        • Hospital do Rim - Fundacao Oswaldo Ramos (55005)
      • São Paulo, Brazil, 05403-900
        • Univ Sao Paulo, Av. Dr Eneas Carvalho de Aguiar, 155 (55007)
    • Minas Gerais
      • Belo Horizonte, Minas Gerais, Brazil, 30150-221
        • Santa Casa de Misericordia de Belo Horizonte (55017)
    • Rio Grande do Sul
      • Porto Alegre, Rio Grande do Sul, Brazil, 90020-090
        • Irmandade da Santa Casa Misericordia Porto Alegre (55016)
  • Czechia
      • Prague, Czechia, 14021
        • Institute for Clinical and Experimental Medicine (42001)
  • France
      • Bordeaux, France, 33076
        • CHU de Bordeaux - Hopital Pellegrin (33010)
      • Nantes, France, 44093
        • Institut de transplantation urologie-nephrologie (33011)
  • Israel
      • Petah Tikva, Israel, 4920235
        • Schneider Childrens Medical Center (97204)
  • Italy
      • Milan, Italy, 20122
        • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (39004)
      • Ranica, Italy, 24020
        • Istituto di Ricerche Farmacologiche Mario Negri IRCCS (39003)
      • Rome, Italy, 165
        • Ospesale Pediatrico bambino Gesu (39005)
  • Japan
    • Nagasaki
      • Nagasaki, Nagasaki, Japan, 852-8501
        • Nagasaki University Hospital (81005)
    • Shizuoka
      • Hamamatsu, Shizuoka, Japan, 430-8558
        • Seirei Hamamatsu General Hospital (81004)
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • AMC Apotheek Goederenontvangst (31002)
      • Groningen de Brug, Netherlands, 9713 GZ
        • University Medical Center Groningen (31004)
      • Nijmegen, Netherlands, 6500 HB
        • Radboud University Medical Center (31003)
  • South Korea
      • Seoul, South Korea, 3722
        • Yonsei University College of Medicine, Sinchon Severance Hospital (82002)
      • Soeul, South Korea, 3080
        • Seoul National University Hospital (82003 & 82005)
  • Spain
      • Barcelona, Spain, 8035
        • Hosp. Universit. Materno-Infantil Vall d' Hebron (34006)
      • Madrid, Spain, 28041
        • Hospital Universitario 12 de Octubre (34008)
      • Valencia, Spain, 46017
        • Hospital Universitario Dr Peset (34009)
  • Switzerland
      • Lausanne, Switzerland, CH-1011
        • CHUV Lausanne (41002)
  • United Kingdom
      • London, United Kingdom, W12 0HS
        • Imperial College Hammersmith Hospital (44013)
      • Manchester, United Kingdom, M13 9WL
        • Royal Manchester Children's Hospital (44011)
      • Nottingham, United Kingdom, NG7 2UH
        • Nottingham University Hospitals NHS Trust (44012)
  • United States
    • California
      • Los Angeles, California, United States, 90022
        • Academic Medical Research Institute (01034)
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado (01037)
    • Florida
      • Gainsville, Florida, United States, 32610
        • University of Florida, Department of Pediatric Nephrology (01010)
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory Investigational Drug Service (01021)
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospital and Clinics (01030)
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Hospital (01008)
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack Meridian Health (01036)
    • New York
      • Hyde Park, New York, United States, 11040
        • Cohen Children Hospital, Pediatric Pharmacy (01022)
      • New York, New York, United States, 10032
        • Columbia Nephrology (01039)
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science Univ., Dept. Pediatrics (01038)
    • Pennsylvania
      • Bethlehem, Pennsylvania, United States, 18017
        • Northeast Clinical Research Center LLC (01009)
    • Texas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital (01027)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Pegcetacoplan administered subcutaneously
Pegcetacoplan administered subcutaneously twice weekly according to protocol defined dosing regimen
Drug: Pegcetacoplan
Complement (C3) Inhibitor
Other Names:
  • APL-2

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Proportion of participants with a reduction in urine protein-to-creatinine ratio (uPCR) of at least 50% from the pretreatment value over time.
Time Frame: 2.5 years
2.5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Apellis Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 29, 2023

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 30, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 30, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 12, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 2, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 31, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • APL2-C3G-314
  • 2023-504625-39-00 (Other Identifier: CTIS)
  • U1111-1290-7826 (Other Identifier: Universal Trial Number (UTN))
  • 2022-002833-33 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings